Giving children and adolescents with cancer a future!

Transcription

1 Schweizerische Pädiatrische Onkologie Gruppe Groupe d Oncologie Pédiatrique Suisse Gruppo d Oncologia Pediatrica Svizzera Swiss Paediatric Oncology Group Giving children and adolescents with cancer a future! Annual report 08

2 Contact SPOG Coordinating Center Effingerstrasse CH-008 Bern Phone Bank accounts Postfinance: 0--8 Berner Kantonalbank BEKB 00 Bern BIC / SWIFT: KBBECHXXX Account number (IBAN): CH Auditors BDO AG Hodlerstrasse 00 Bern

3 Contents Editorial 4 Scientific reports Patient-related clinical research Swiss Childhood Cancer Registry: report on activities Translational research / Biobank SPOG Scientific Meeting 08 SPOG Scientific Advisory Board SPOG publications in 08 Working group reports Professional Development Working Group Protocol Working Group Stem Cell Therapy Working Group Germline DNA Working Group Paediatric Haematology Working Group Shared Care Working Group Reports from the SPOG Coordinating Center Clinical Project Management and Quality Management Fundraising 8 Congratulations and special thanks 0 Annual accounts Structure of SPOG People

4 Editorial Dear readers And finally, the bidding consortium comprising SPOG and Bern University, the current sponsor of the Swiss Childhood Cancer Registry (SCCR), that was mentioned in last year s Editorial was successful. The consortium was assigned the tasks associated with the future childhood cancer registry by the federal government with effect from 00. I would additionally like to highlight two other aspects that are not primarily related to research. The newly established Haematology Working Group got off to a very successful start. It has received so many membership requests that membership has had to be restricted to keep the group to a manageable size. And it withstood its baptism of fire by providing prompt, clear and clinically relevant information during a temporary medication supply shortage. I would like to take this opportunity to report on some of the aspects and decisions taken in the past year that are relevant for the future of paediatric haematology/oncology as well as for the future of SPOG. There are four aspects of research that I would like to highlight: Firstly, the spectrum of open international studies and research projects in which SPOG represents the sponsors in Switzerland was broadened. Specifically, four new studies were opened in 08, compared with three in each of 0 and 07. This meant that a total of studies, 4 of them research projects, were open for patient recruitment at the end of 08. The spectrum of ongoing research projects and studies is of course still narrower than in neighbouring countries, but SPOG has made progress here. Secondly, the basic agreements between SPOG and the individual SPOG member centres called for in the report on the Swissmedic inspection mentioned in last year s Editorial were completed. These agreements govern the rights and obligations between SPOG and the member centres in general, independently of specific study projects. Comparable agreements were established at SAKK, the Swiss Group for Clinical Cancer Research in adult patients, years ago and have proven valuable. Thirdly, patient recruitment to the internal SPOG study SPOG 0 FN Definition, an interventional, randomized, controlled multicentre study, was completed successfully in September 08. Publication of the initial findings from this study of supportive care, which involved most of the SPOG centres, is expected in the first half of 0. At the same time, the process of defining the area of research and objectives of the next one or two internal SPOG studies has already begun within SPOG. 4 At the end of 08 SPOG decided to initiate an additional specialist association. Like other specialist associations, it is intended to take over tasks from SPOG such as the organization of subspecialty exams, provision of opinions to the IVHSM (Intercantonal Agreement on Highly Specialized Medicine), the award of further training credits and management of the collection of clinical guidelines. This will once again bring the research-oriented structure of SPOG more in line with its tasks. And finally I would like to thank all the employees at the SPOG CC and the individual centres for the tremendous effort they put into reaching our shared goals and, more particularly, the patients and their families for taking part in the studies together we are helping to improve the chances of recovery for children and adolescents with cancer. Prof. Roland A. Ammann President of SPOG

5 Scientific reports Patient-related clinical research Bellinzona Bern Geneva Lausanne Lucerne St. Gallen Zurich Total Total number of study participations Number of patients (excluding multiple references) Treatment studies AIEOP-BFM ALL 00 Register ALL SCTped 0 FORUM BEACON CWS-007-HR EuroNet-PHL-C EuroNet-PHL-LP EWING 008 HR-NBL-.8 / SIOPEN IntReALL SR 00 Craniopharyngioma 007 LCH-IV LINES reecur SIOP CNS GCT II SIOP PNET MB VINILO II Supportive care studies SPOG 0 FN Definition Research projects (registry studies and biology studies) COSS CWS Register SoTiSaR EWOG-MDS 00 EWOG-SAA 00 I-HIT-MED GPOH-MET Registry INFORM MNP.0 NHL-BFM Registry 0 SIOP-LGG 004 Interim Register STEP Total non-study patients Basel SPOG station Aarau Number of patients included in studies in 08 A total of new patients were recruited to SPOG studies in of them took part in two studies and were counted in both The non-study patients comprise 4 initial reports, 8 relapse reports and secondary tumour cases. This study was managed as a clinical study until the end of 0. Only registry patients have been enrolled since 07, but the study will remain in the category of clinical studies and therapy studies until it has been completed and evaluated.

6 Overview of ongoing clinical studies and research projects as of December 08 The clinical studies and 4 research projects listed in the following tables were open for patient recruitment at the end of 08. SPOG tries to offer ongoing studies at as many of the nine member hospitals in Switzerland as possible so that the largest possible number of children and adolescents with cancer can benefit from SPOG studies. Overview of clinical studies open on December 08 Number of participating SPOG member institutions Study at the end of 08 open since Study code Condition under investigation AIEOP-BFM ALL 00 Acute lymphoblastic leukaemia 7 00 ALL SCTped 0 FORUM Allogeneic stem cell transplant in children and adolescents with acute lymphoblastic leukaemia 0 BEACON Neuroblastoma 0 CWS-007-HR High-risk soft tissue sarcoma 0 EuroNet-PHL-C Classical Hodgkin lymphoma 0 HR-NBL-.8 / SIOPEN High-risk neuroblastoma 004 IntReALL SR 00 Standard risk relapsed acute lymphoblastic leukaemia 0 Craniopharyngioma 007 Craniopharyngioma 008 LCH-IV Langerhans cell histiocytosis 04 LINES Low- and medium-risk neuroblastoma 0 PHITT Liver tumours (hepatoblastoma and hepatocellular carcinoma) 8 08 reecur Recurrent and primary refractory Ewing sarcoma 08 SIOP Ependymoma II Ependymoma 08 SIOP PNET MB Brain tumours (medulloblastoma) 0 VINILO II Recurrent or refractory low-grade glioma 07

7 Overview of research projects open on December 08 Number of participating SPOG member institutions Study at the end of 08 open since Study code Condition under investigation ALL-REZ BFM Relapsed acute lymphoblastic leukaemia 0 COSS Register Osteosarcomas and other bone sarcomas 7 0 CWS Register SoTiSaR Soft tissue sarcoma and other soft tissue tumours 7 0 EU-RHAB Rhabdoid tumours 7 0 EWOG-MDS 00 Myelodysplastic syndrome and juvenile myelomonocytic leukaemia 8 00 EWOG-SAA 00 Acquired severe aplastic anaemia 7 0 GPOH-MET Register Malignant endocrine tumours 7 0 I-HIT-MED Medulloblastoma, ependymoma, pineoblastoma and CNS primitive neuroectodermal tumours 07 INFORM Tumours that are recurrent or progressing under therapy 07 MNP.0 Diagnosis of brain tumours 08 NHL-BFM Registry 0 Non-Hodgkin lymphoma 7 0 Relapsed AML 00 Recurrent or refractory acute myeloid leukaemia 0 SIOP-LGG 004 Interim Register Low-grade gliomas 7 0 STEP Register Rare tumours in children and adolescents 7 0 7

8 Swiss Childhood Cancer Registry: report on activities Since 7 the Swiss Childhood Cancer Registry (SCCR) has collected data on cancer among children and adolescents throughout Switzerland. It was set up by the paediatric oncologists in Switzerland (SPOG) and has been based at the Institute for Social and Preventive Medicine (ISPM) at Bern University since 004. It records not only detailed information on the diagnosis but also on therapy, the course of the disease, late effects and quality of life. In this way it helps to gain a better understanding of the causes of cancers, to improve therapy and to prevent late effects. All the children s hospitals in Switzerland that treat children with cancer report newly diagnosed cases to the register and then send annual updates. An exchange of information also takes place with bodies such as the Federal Statistical Office and the cantonal and regional cancer registries. Close cooperation with doctors allows study results to be quickly incorporated into cancer treatments. Parents and patients can obtain information and support. The authorities are provided with rapid answers to urgent questions. The childhood cancer registry monitors cancer in children and adolescents throughout Switzerland and in this way contributes to public health reporting. Epidemiological research at the SCCR The SCCR is working on a number of research projects to investigate the frequency and prognosis of cancers in children and adolescents, possible causes of cancer in children and the long-term effects in cured children as part of the national Swiss Childhood Cancer Survivor Study. Our research projects are described in detail on the SCCR website ( and in the latest annual report of the SCCR. News Cancer Registration Act, CRA To date there has been no legal requirement to report cancers in Switzerland. This is now changing with the new Cancer Registration Act that will come into force on January 00. The Act foresees a childhood cancer registry managed by the federal government. The SCCR and the Swiss Paediatric Oncology Group (SPOG) submitted a joint tender for this federal task and were awarded the mandate by the federal government. The SCCR will begin preparing for this federal task in 0. The SCCR has been involved in the process of drafting the Cancer Registration Act (CRA) and the Cancer Registration Ordinance (CRO) since 0. It supports the Federal Office of Public Health (FOPH) in all questions relating to cancer in children and adolescents, such as which information should be recorded in future as basic and additional data, and how health reporting and cancer monitoring can be expanded in this area. It is a member of the implementation of the CRA Working Group set up by the FOPH. The SCCR is also involved intensively in the production of new registry software that is being programmed by the federal government. Cantonal cancer registries The aim of the SCCR is to compile a complete record of cancer in children and adolescents in Switzerland. In order to achieve this aim, the cases registered in the SCCR are compared regularly with the cantonal cancer registries. This enables any cases that may have been missed to be included in the register subsequently. In 08 data comparisons were performed with the cantonal cancer registries in Basel and Ticino and with the Paul Scherrer Institute (PSI). Oncosuisse National Strategy against Cancer (NSC) The SCCR is one of the lead bodies in the National Strategy against Cancer, and is an active participant in project cluster J Epidemiology and monitoring, especially projects 7. Registry data on treatment quality and data linkage and 7. Knowledge transfer in everyday work and politics. Federal Statistical Office (FSO) The SCCR provides the FSO with annual data on the incidence of cancer in children in Switzerland for publication on the FSO website. 8

9 Childhood Cancer Switzerland The SCCR is a founding member of Childhood Cancer Switzerland, belongs to its Board and Management Conference and, together with SPOG, covers research within Childhood Cancer Switzerland. The SCCR is also a member of the Aftercare Working Group, which aims to improve aftercare for childhood cancer survivors, and helps to organize childhood cancer conferences. National Institute for Cancer Epidemiology and Registration (NICER) Since 0 the SCCR has been an associate member of NICER and attends meetings of the NICER Registries Advisory Board and the NICER CoReDays. The SCCR is a member of the Treatment data-cra Working Group organized by NICER. Prof. Claudia Kuehni st Co-Head Swiss Childhood Cancer Registry Further information You can find more information and news on our website ( Dr. rer. nat. Verena Pfeiffer nd Co-Head Swiss Childhood Cancer Registry

10 Translational research / Biobank Swiss Paediatric Haematology and Oncology Group (SPHO) Biobank Network The aim of the biobank is to collect samples from most of the patients who receive oncological treatment or a stem cell transplant in Switzerland and store them in a national biobank for future research projects. This is the background against which the necessary infrastructure was established at the University Children s Hospital Zurich. The following samples have been stored in recent years: Nov 08 Total Leukaemia ALL Leukaemia AML Leukaemia CML Pending Total Stored leukaemia samples Diagnosis Leukaemia ALL Leukaemia AML 0 Leukaemia CML 0 Pending 0 Total November 08 Follow-up Pending Total Leukaemia samples classified by the time at which the samples were obtained. Pending = Details of the samples still being clarified Total (October) (00 08) Bone tumours 7 CNS tumours 4 Germ cell tumours Kidney tumours Liver tumours Lymphomas 4 Neuroblastomas 0 Normal tissues Pulmonary tumours 0 Rhabdomyosarcoma 0 Other tumours + pending Total Stored solid tumour samples 0

11 A network for solid tumours was established with the institutes of pathology and the associated SPOG centres. Cooperation has already started with Zurich, Bern and Basel, and agreements have been drawn up with the centres in Geneva, Lucerne, St. Gallen and Lausanne. General consent is obtained from patients and families at the SPOG centre concerned to enable both samples and relevant biological and clinical information to be stored in encoded form. The biobank database will be linked to the Swiss Childhood Cancer Registry. Quality-controlled data from European therapy optimization studies are also available for research purposes. These data should be used, if possible, to treat all paediatric patients with an oncological diagnosis in Switzerland. Samples and information are made available to researchers in compliance with the legal requirements after the responsible working groups have evaluated the research proposals. Prof. Jean-Pierre Bourquin (Director of the SPHO Biobank Network) submitted a proposal with the title Swiss Paediatric Haematology/ Oncology Metabank a network for precision medicine research to the Swiss National Science Foundation (SNSF, BioLink call 08) in collaboration with Prof. Marc Ansari (Head of the Biobank in Haematology and Oncology Paediatric, BaHOP in Geneva) and Prof. Claudia Kuehni (Head of the SCCR). This project will be funded for two years starting in April 0. Its objective is to expand the IT platform so that the network can be operated efficiently. Within the network, the samples and data in the SPHO Biobank Network and the germ cell DNA biobank (Germline DNA Biobank Switzerland for Childhood Cancer and Haematological Diseases (BISKIDS)) will be linked to the SCCR data to enable research projects in the field of paediatric oncology and haematology to be carried out. The managers of the SPHO Biobank Network are at your disposal to provide further information: SPHO Biobank Network Dr. Irina Banzola, Biobank Manager University Children s Hospital Zurich Balgrist Campus Lengghalde 8008 Zurich [email protected] [email protected] Prof. Jean-Pierre Bourquin Senior oncologist University Children s Hospital Zurich [email protected] Dr. Irina Banzola Biobank Manger Prof. Jean-Pierre Bourquin Senior oncologist at the University Children s Hospital Zurich

12 SPOG Scientific Meeting got off to a splendid start with the SPOG Scientific Meeting in Lugano at the end of January. Almost 80 participants from all over Switzerland took part in the scientifically valuable exchange in Ticino. SPOG board member Prof. Maja Beck Popovic was responsible for the wide-ranging programme of interesting presentations of research projects focusing on basic research, clinical research, registry studies, epidemiological topics and supportive care. This provided the researchers with a platform to showcase their ideas, objectives and findings and to discuss them in a subsequent question and answer session. Particularly notable was the presentation given by guest speaker Birgit Geoerger from the Institut Gustave Roussy in Paris. She talked about Paediatric Cancer Precision Medicine: Profiling And Beyond. Special thanks are due to the following people for the outstanding organization of the Meeting: Prof. Maja Beck Popovic for putting together the interesting programme, and Dr. Pierluigi Brazzola, who mastered the logistical challenges on the ground. We are also grateful to the staff at the SPOG Coordinating Center who all made sure that everyone felt at home in Ticino during the two days of the Meeting. This scientific event was made possible in part by financial support from various pharmaceutical companies. Dr. Manuel Diezi from CHUV University Hospital Lausanne, talking to Prof. Roland A. Ammann from Inselspital Bern, President of SPOG. Dr. André von Büren from HUG Geneva University Hospitals / CANSEARCH Research Laboratory talked about brain tumours in children. Participants at the Meeting. Derya Keller and Michael Zeller both work in Clinical Project Management at the SPOG Coordinating Center.

13 SPOG Scientific Advisory Board The SPOG Scientific Advisory Board Meeting, held every two years, took place in June 08. The members of the Scientific Advisory Board provide advice to SPOG on current medical, scientific and health policy topics and challenges. The focus this time was on shared care. In 08, for the first time, the Meeting formed part of the Swiss Oncology & Haematology Congress (SOHC) in Zurich. It was attended by the three SPOG Scientific Advisory Board Members Prof. em. Günter Henze from Berlin, Prof. Rod Skinner from Newcastle and the 08 Chairman of the Advisory Board, Dr. Jean Michon from Paris, who was requested to write a recommendation report for SPOG after the Meeting. The SPOG member institutions were represented by Prof. Maja Beck Popovic and Dr. Raffaele Renella from CHUV University Hospital Lausanne, Prof. Jochen Rössler and Prof. Roland A. Ammann from Inselspital Bern, Prof. Marc Ansari from HUG Geneva University Hospitals, Dr. Freimut H. Schilling from Lucerne Cantonal Children s Hospital, and Dr. Heinz Hengartner from the Children s Hospital of Eastern Switzerland in St. Gallen. Dr. Reta Malär, a consultant in paediatric oncology and haematology at Cantonal Hospital Graubünden, took part as a Prof. em. Günter Henze Former Director of the Department of Paediatric Oncology and Haematology Charité CVK, Berlin, Germany guest. The SPOG Coordinating Center was represented by its Managing Director Isabelle Lamontagne-Müller, the Head of Clinical Operations Julia Ruckstuhl, Nicola Miglino and Patrizia Specker. A lively exchange on the subject of shared care took place between the members of the Advisory Board and the SPOG representatives; it was fuelled by the valuable experience of the Advisory Board members and contrasted the situation at SPOG with the structures in Switzerland. Prof. Roderick Skinner Consultant in Paediatric and Adolescent Oncology / BMT, Honorary Professor of Childhood Cancer Research, Newcastle University, Newcastle upon Tyne, United Kingdom Dr. Jean Michon Paediatric oncologist, President of the French Society to combat cancer and leukaemia in childhood and adolescence (SFCE) Centre SIREDO, Institut Curie, Paris, France

14 SPOG publications in 08 Below is a list of the publications that appeared in renowned scientific journals in 08 and in which people from the SPOG network were actively involved. Authors / title / journal Publications in peer-reviewed journals JIF Adamczick C, Dierig A, Welzel T, Schifferli A, Blum J, Ritz NJBID. Double trouble: visceral leishmaniasis in twins after traveling to Tuscany a case report. BMC Infectious Diseases. (08); 8(), 4. doi:0.8/s Amoon AT, Crespi CM, Ahlbom A, Bhatnagar M, Bray I, Bunch KJ, Clavel J, Feychting M, Hémon D, Johansen C, Kreis C, Malagoli C, Marquant F, Pedersen C, Raaschou-Nielsen O, Röösli M, Spycher BD, Sudan M, Swanson J, Tittarelli A, Tuck DM, Tynes T, Vergara X, Vinceti M, Wünsch-Filho V, Kheifets L. Proximity to overhead power lines and childhood leukaemia: an international pooled analysis. British Journal of Cancer. (08); (), 4-7. doi:0.08/s Arni D, Wildhaber BE, McLin V, Rimensberger PC, Ansari M, Fontana P, Karam O. Effects of plasma transfusions on antithrombin levels after paediatric liver transplantation. Vox Sang. (08). doi:0./vox Baenziger J, Roser K, Mader L, Christen S, Kuehni CE, Gumy-Pause F, Tinner EM, Michel G. Can the theory of planned behavior help explain attendance to follow-up care of childhood cancer survivors? Psychooncology. (08); 7(), doi:0.00/pon Baleydier F, Ranza E, Schappi M, Rougemont AL, Merlini L, Ansari M, Blanchard-Rohner G. Activated Phosphoinositide Kinase Delta Syndrome (APDS): A Primary Immunodeficiency Mimicking Lymphoma. J Pediatr Hematol Oncol. (08). doi:0.07/mph Bartolini B, Puccinelli F, Hajdu SD, Stathopoulos C, Beck Popovic M, Munier FL, Saliou G. Middle meningeal artery occlusion for intra-arterial chemotherapy of retinoblastoma. Interv Neuroradiol. (08), doi:0.77/ Bassan R, Bourquin JP, DeAngelo DJ, Chiaretti S. New Approaches to the Management of Adult Acute Lymphoblastic Leukemia. J Clin Oncol. (08), JCO doi:0.00/jco Belle FN, Kasteler R, Schindera C, Bochud M, Ammann RA, von der Weid NX, Kuehni CE; Swiss Paediatric Oncology Group. No evidence of overweight in long-term survivors of childhood cancer after glucocorticoid treatment. Cancer. (08); 4(7), 7-8. doi:0.00/cncr...7 Belle FN, Weiss A, Schindler M, Goutaki M, Bochud M, Zimmermann K, von der Weid NX, Ammann RA, Kuehni CE. Overweight in childhood cancer survivors: the Swiss Childhood Cancer Survivor Study. Am J Clin Nutr. (08); 07(), -. doi:0.0/ajcn/nqx Belle FN, Wenke-Zobler J, Cignacco E, Spycher BD, Ammann RA, Kuehni CE, Zimmermann K. Overweight in childhood cancer patients at diagnosis and throughout therapy: A multicentre cohort study. Clin Nutr. (08). doi:0.0/j. clnu Benadiba J, Ansari M, Krajinovic M, Vachon MF, Duval M, Teira P, Cellot S, Bittencourt H. Pharmacokinetics-adapted Busulfan-based myeloablative conditioning before unrelated umbilical cord blood transplantation for myeloid malignancies in children. PLoS One. (08); (4), e08. doi:0.7/journal.pone Bennett CM, Neunert C, Grace RF, Buchanan G, Imbach P, Vesely SK, Kühne T. Predictors of remission in children with newly diagnosed immune thrombocytopenia: Data from the Intercontinental Cooperative ITP Study Group Registry II participants. Pediatr Blood Cancer. (08); (). doi:0.00/pbc.7..4 Benzing V, Eggenberger N, Spitzhuttl J, Siegwart V, Pastore-Wapp M, Kiefer C, Slavova N, Grotzer MA, Heinks T, Schmidt M, Conzelmann A, Steinlin M, Everts R, Leibundgut K. The Brainfit study: efficacy of cognitive training and exergaming in pediatric cancer survivors - a randomized controlled trial. BMC Cancer. (08); 8(), 8. doi:0.8/s x Blesinger H, Kaulfuss S, Aung T, Schwoch S, Prantl L, Rössler J, Wilting J, Becker J. PIKCA mutations are specifically localized to lymphatic endothelial cells of lymphatic malformations. PLoS One. (08); (7), e0004. doi:0.7/journal. pone Bochtler T, Kartal-Kaess M, Granzow M, Hielscher T, Cosenza MR, Herold-Mende C, Jauch A, Kramer A. Micronucleus formation in human cancer cells is biased by chromosome size. Genes Chromosomes Cancer. (08). doi:0.00/ gcc

15 Authors / title / journal Publications in peer-reviewed journals JIF Bovero M, Giacomo C, Ansari M, Roulin MJ. Role of advanced nurse practitioners in the care pathway for children diagnosed with leukemia. Eur J Oncol Nurs. (08);, doi:0.0/j.ejon Bredell M, Rordorf T, Kroiss S, Rucker M, Zweifel DF, Rostetter C. Denosumab as a Treatment Alternative for Central Giant Cell Granuloma: A Long-Term Retrospective Cohort Study. J Oral Maxillofac Surg. (08); 7(4), doi:0.0/j. joms Bright CJ, Hawkins MM, Winter DL, Alessi D, Allodji RS, Bagnasco F, Bardi E, Bautz A, Byrne J, Feijen EAM, Fidler MM, Garwicz S, Grabow D, Gudmundsdottir T, Guha J, Haddy N, Jankovic M, Kaatsch P, Kaiser M, Kuehni CE, Linge H, Ofstaas H, Ronckers CM, Skinner R, Teepen JC, Terenziani M, Vu-Bezin G, Wesenberg F, Wiebe T, Sacerdote C, Jakab Z, Haupt R, Lahteenmaki P, Zaletel LZ, Kuonen R, Winther JF, de Vathaire F, Kremer LC, Hjorth L, Reulen RC, PanCareSurFup C. Risk of Soft-Tissue Sarcoma Among 40 Five-Year Survivors of Childhood Cancer in Europe. J Natl Cancer Inst. (08); 0(), 4-0. doi:0.0/jnci/djx..8 Brock PR, Maibach R, Childs M, Rajput K, Roebuck D, Sullivan MJ, Laithier V, Ronghe M, Dall'Igna P, Hiyama E, Brichard B, Skeen J, Mateos ME, Capra M, Rangaswami AA, Ansari M, Rechnitzer C, Veal GJ, Covezzoli A, Brugieres L, Perilongo G, Czauderna P, Morland B, Neuwelt EA. Sodium Thiosulfate for Protection from Cisplatin-Induced Hearing Loss. N Engl J Med. (08); 78(), 7-8. doi:0.0/nejmoa Byrne J, Alessi D, Allodji RS, Bagnasco F, Bardi E, Bautz A, Bright CJ, Brown M, Diallo I, Feijen E, Fidler MM, Frey E, Garwicz S, Grabow D, Gudmundsdottir T, Hagberg O, Harila-Saari A, Hau EM, Haupt R, Hawkins MM, Jakab Z, Jankovic M, Kaatsch P, Kaiser M, Kremer LCM, Kuehni CE, Kuonen R, Ladenstein R, Lahteenmaki PM, Levitt G, Linge H, D LL, Michel G, Morsellino V, Mulder RL, Reulen RC, Ronckers CM, Sacerdote C, Skinner R, Steliarova-Foucher E, van der Pal HJ, de Vathaire F, Vu Bezin G, Wesenberg F, Wiebe T, Winter DL, Falck Winther J, Witthoff E, Zadravec Zaletel L, Hjorth L. The PanCareSurFup consortium: research and guidelines to improve lives for survivors of childhood cancer. Eur J Cancer. (08); 0, doi:0.0/j.ejca Byrne J, Grabow D, Campbell H, O'Brien K, Bielack S, Am Zehnhoff-Dinnesen A, Calaminus G, Kremer L, Langer T, van den Heuvel-Eibrink MM, van Dulmen-den Broeder E, Baust K, Bautz A, Beck JD, Berger C, Binder H, Borgmann-Staudt A, Broer L, Cario H, Casagranda L, Clemens E, Deuster D, de Vries A, Dirksen U, Falck Winther J, Fossa S, Font-Gonzalez A, Grandage V, Haupt R, Hecker-Nolting S, Hjorth L, Kaiser M, Kenborg L, Kepak T, Kepakova K, Knudsen LE, Krawczuk-Rybak M, Kruseova J, Kuehni CE, Kunstreich M, Kuonen R, Lackner H, Leiper A, Loeffen EAH, Luks A, Modan-Moses D, Mulder R, Parfitt R, Paul NW, Ranft A, Ruud E, Schilling R, Spix C, Stefanowicz J, Straubeta G, Uitterlinden AG, van den Berg M, van der Kooi AL, van Dijk M, van Leeuwen F, Zolk O, Zoller D, Kaatsch P. PanCareLIFE: The scientific basis for a European project to improve long-term care regarding fertility, ototoxicity and health-related quality of life after cancer occurring among children and adolescents. Eur J Cancer. (08); 0, 7-7. doi:0.0/j.ejca Chatzis O, Darbre S, Pasquier J, Meylan P, Manuel O, Aubert JD, Beck Popovic M, Masouridi-Levrat S, Ansari M, Kaiser L, Posfay-Barbe KM, Asner SA. Burden of severe RSV disease among immunocompromised children and adults: a 0 year retrospective study. BMC Infect Dis. (08); 8(),. doi:0.8/s Choong E, Uppugunduri CRS, Marino D, Kuntzinger M, Doffey-Lazeyras F, Lo Piccolo R, Chalandon Y, Peters C, Daali Y, Ansari M. Therapeutic Drug Monitoring of Busulfan for the Management of Pediatric Patients: Cross-Validation of Methods and Long-Term Performance. Ther Drug Monit. (08); 40(), 84-. doi:0.07/ftd Corbacioglu S, Carreras E, Ansari M, Balduzzi A, Cesaro S, Dalle JH, Dignan F, Gibson B, Güngör T, Gruhn B, Lankester A, Locatelli F, Pagliuca A, Peters C, Richardson PG, Schulz AS, Sedlacek P, Stein J, Sykora KW, Toporski J, Trigoso E, Vetteranta K, Wachowiak J, Wallhult E, Wynn R, Yaniv I, Yesilipek A, Mohty M, Bader P. Diagnosis and severity criteria for sinusoidal obstruction syndrome/veno-occlusive disease in pediatric patients: a new classification from the European society for blood and marrow transplantation. Bone Marrow Transplant. (08); (), 8-4. doi:0.08/bmt Diorio C, Robinson PD, Ammann RA, Castagnola E, Erickson K, Esbenshade A, Fisher BT, Haeusler GM, Kuczynski S, Lehrnbecher T, Phillips R, Cabral S, Dupuis LL, Sung L. Guideline for the management of Clostridium difficile infection in children and adolescents with cancer and pediatric hematopoietic stem cell transplantation recipients. J Clin Oncol (08); : Drozdov D, Bonaventure A, Nakata K, Suttorp M, Belot A. Temporal trends in the proportion of «cure» in children, adolescents, and young adults diagnosed with chronic myeloid leukemia in England: A population-based study. Pediatr Blood Cancer. (08); (), e74. doi:0.00/pbc Dworzak MN, Buldini B, Gaipa G, Ratei R, Hrusak O, Luria D, Rosenthal E, Bourquin JP, Sartor M, Schumich A, Karawajew L, Mejstrikova E, Maglia O, Mann G, Ludwig WD, Biondi A, Schrappe M, Basso G, International BFMFn. AIEOP-BFM consensus guidelines 0 for flow cytometric immunophenotyping of Pediatric acute lymphoblastic leukemia. Cytometry B Clin Cytom. (08); 4(), 8-. doi:0.00/cyto.b El-Ayadi M, Egervari K, Merkler D, McKee TA, Gumy-Pause F, Stichel D, Capper D, Pietsch T, Ansari M, von Büren AO. Concurrent IDH and SMARCB Mutations in Pediatric Medulloblastoma: A Case Report. Front Neurol. (08);, 8. doi:0.8/fneur

16 Authors / title / journal Publications in peer reviewed journals JIF Fidler MM, Reulen RC, Winter DL, Allodji RS, Bagnasco F, Bardi E, Bautz A, Bright CJ, Byrne J, Feijen EAM, Garwicz S, Grabow D, Gudmundsdottir T, Guha J, Haddy N, Jankovic M, Kaatsch P, Kaiser M, Kuonen R, Linge H, Maule M, Merletti F, Ofstaas H, Ronckers CM, Skinner R, Teepen J, Terenziani M, Vu-Bezin G, Wesenberg F, Wiebe T, Jakab Z, Haupt R, Lahteenmaki P, Zaletel LZ, Kuehni CE, Winther JF, de Vathaire F, Kremer LC, Hjorth L, Hawkins MM. Risk of Subsequent Bone Cancers Among 40 Five-Year Survivors of Childhood and Adolescent Cancer in Europe. J Natl Cancer Inst. (08); 0(). doi:0.0/jnci/djx..8 0 Furtwangler R, Kager L, Melchior P, Rube C, Ebinger M, Nourkami-Tutdibi N, Niggli F, Warmann S, Hubertus J, Amman G, Leuschner I, Vokuhl C, Graf N, Fruhwald MC. High-dose treatment for malignant rhabdoid tumor of the kidney: No evidence for improved survival-the Gesellschaft fur Padiatrische Onkologie und Hamatologie (GPOH) experience. Pediatr Blood Cancer. (08); (). doi:0.00/pbc Gaertner K, Lüer SC, Frei-Erb M, von Ammon K. Complementary individual homeopathy in paediatric cancer care: A case series from a University Hospital, Switzerland. Complement Ther Med. (08); 4, doi:0.0/j. ctim Georgi TW, Kluge R, Kurch L, Chavdarova L, Hasenclever D, Stoevesandt D, Pelz T, Landman-Parker J, Wallace WH, Karlen J, Fernandez-Teijeiro A, Cepelova M, Fossa A, Balwierz W, Attarbaschi A, Ammann RA, Pears J, Hraskova A, Uyttebroeck A, Beishuizen A, Dieckmann K, Leblanc T, Daw S, Baumann J, Korholz D, Sabri O, Mauz-Korholz C. (8)F-FDG PET Response of Skeletal (Bone Marrow and Bone) Involvement After Induction Chemotherapy in Pediatric Hodgkin Lymphoma: Are Specific Response Criteria Required? J Nucl Med. (08); (0), 4-0. doi:0.7/jnumed Gerth-Kahlert C, Tiwari A, Hauri-Hohl MM, Hanson JVM, Bahr A, Palmowski-Wolfe A, Güngör T, Berger W. Unusual retinopathy in a child with severe combined immune deficiency. Ophthalmic Genet. (08); (), -4. doi:0.080/ Grabow D, Kaiser M, Hjorth L, Byrne J, Alessi D, Allodji RS, Bagnasco F, Bardi E, Bautz A, Bright CJ, de Vathaire F, Feijen EAM, Garwicz S, Hagberg O, Haupt R, Hawkins MM, Jakab Z, Kremer LCM, Kuehni CE, Kuonen R, Lahteenmaki PM, Reulen RC, Ronckers CM, Sacerdote C, Vu-Bezin G, Wesenberg F, Wiebe T, Winter DL, Winther JF, Zaletel LZ, Kaatsch P, PanCareSurFup C. The PanCareSurFup cohort of 8, five-year survivors of childhood cancer: a cohort from European countries. Eur J Epidemiol. (08); (), -4. doi:0.007/s Greiner J, Schrappe M, Claviez A, Zimmermann M, Niemeyer C, Kolb R, Eberl W, Berthold F, Bergsträsser E, Gnekow A, Lassay E, Vorwerk P, Lauten M, Sauerbrey A, Rischewski J, Beilken A, Henze G, Korte W, Moricke A. THROMBOTECT a randomized study comparing low molecular weight heparin, antithrombin and unfractionated heparin for thromboprophylaxis during inudction therapy of acute lymphoblastic leukemia in children and adolescents. Haematologica. (08). doi:0.4/haematol Harju E, Roser K, Dehler S, Michel G. Health-related quality of life in adolescent and young adult cancer survivors. Support Care Cancer. (08); (), 0-0. doi:0.007/s z..7 7 Heinemann M, Ranft A, Langer T, Jurgens H, Kreyer J, Vieth V, Schafers M, Weckesser M, Simon T, Hassenpflug W, Corbacioglu S, Bielack S, Mayer-Steinacker R, Kühne T, van den Berg H, Gelderblom H, Bauer S, Stegger L, Dirksen U. Recurrence of Ewing sarcoma: Is detection by imaging follow-up protocol associated with survival advantage? Pediatr Blood Cancer. (08); (7), e70. doi:0.00/pbc Hoffman LM, Veldhuijzen van Zanten SEM, Colditz N, Baugh J, Chaney B, Hoffmann M, Lane A, Fuller C, Miles L, Hawkins C, Bartels U, Bouffet E, Goldman S, Leary S, Foreman NK, Packer R, Warren KE, Broniscer A, Kieran MW, Minturn J, Comito M, Broxson E, Shih CS, Khatua S, Chintagumpala M, Carret AS, Escorza NY, Hassall T, Ziegler DS, Gottardo N, Dholaria H, Doughman R, Benesch M, Drissi R, Nazarian J, Jabado N, Boddaert N, Varlet P, Giraud G, Castel D, Puget S, Jones C, Hulleman E, Modena P, Giagnacovo M, Antonelli M, Pietsch T, Gielen GH, Jones DTW, Sturm D, Pfister SM, Gerber NU, Grotzer MA, Pfaff E, von Büren AO, Hargrave D, Solanki GA, Jadrijevic Cvrlje F, Kaspers GJL, Vandertop WP, Grill J, Bailey S, Biassoni V, Massimino M, Calmon R, Sanchez E, Bison B, Warmuth-Metz M, Leach J, Jones B, van Vuurden DG, Kramm CM, Fouladi M. Clinical, Radiologic, Pathologic, and Molecular Characteristics of Long-Term Survivors of Diffuse Intrinsic Pontine Glioma (DIPG): A Collaborative Report From the International and European Society for Pediatric Oncology DIPG Registries. J Clin Oncol. (08); (), -7. doi:0.00/jco Jankovic M, Haupt R, Spinetta JJ, Beck JD, Byrne J, Calaminus G, Lackner H, Biondi A, Oeffinger K, Hudson M, Skinner R, Reaman G, van der Pal H, Kremer L, den Hartogh J, Michel G, Frey E, Bardi E, Hawkins M, Rizvi K, Terenziani M, Valsecchi MG, Bode G, Jenney M, de Vathaire F, Garwicz S, Levitt GA, Grabow D, Kuehni CE, Schrappe M, Hjorth L, participants in P. Long-term survivors of childhood cancer: cure and care-the Erice Statement (00) revised after 0 years (0). J Cancer Surviv. (08); (), doi:0.007/s Janssens GO, Kramm CM, von Büren AO. Diffuse intrinsic pontine gliomas (DIPG) at recurrence: is there a window to test new therapies in some patients? J Neurooncol. (08); (), 0. doi:0.007/s z Johnston DL, Keene D, Strother D, Taneva M, Lafay-Cousin L, Fryer C, Scheinemann K, Carret AS, Fleming A, Afzal S, Wilson B, Bowes L, Zelcer S, Mpofu C, Silva M, Larouche V, Brossard J, Bouffet E. Survival Following Tumor Recurrence in Children With Medulloblastoma. J Pediatr Hematol Oncol. (08); 40(), e-e. doi:0.07/mph

17 Authors / title / journal Publications in peer reviewed journals JIF 4 Jyotsana N, Sharma A, Chaturvedi A, Scherr M, Kuchenbauer F, Sajti L, Barchanski A, Lindner R, Noyan F, Suhs KW, Grote-Koska D, Brand K, Vornlocher HP, Stanulla M, Bornhauser B, Bourquin JP, Eder M, Thol F, Ganser A, Humphries RK, Ramsay E, Cullis P, Heuser M. RNA interference efficiently targets human leukemia driven by a fusion oncogene in vivo. Leukemia. (08); (), 4-. doi:0.08/leu Karow A, Wilhelm A, Ammann RA, Baerlocher GM, Pabst T, Mansouri Taleghani B, Rössler J, Leibundgut K. Peripheral blood progenitor cell collection in pediatric patients optimized by high pre-apheresis count of circulating CD4+ cells and high blood flow. Bone Marrow Transplant. (08). doi:0.08/s Karremann M, Gielen GH, Hoffmann M, Wiese M, Colditz N, Warmuth-Metz M, Bison B, Claviez A, van Vuurden DG, von Büren AO, Gessi M, Kuhnle I, Hans VH, Benesch M, Sturm D, Kortmann RD, Waha A, Pietsch T, Kramm CM. Diffuse high-grade gliomas with H K7M mutations carry a dismal prognosis independent of tumor location. Neuro Oncol. (08); 0(), -. doi:0.0/neuonc/nox Kasteler R, Kam LMH, Weiss A, Waespe N, Sommer G, Singer F, von der Weid NX, Ansari M, Kuehni CE; Swiss Paediatric Oncology Group. Monitoring pulmonary health in Swiss childhood cancer survivors. Pediatr Blood Cancer. (08); (0), e7. doi:0.00/pbc Kasteler R, Weiss A, Schindler M, Sommer G, Latzin P, von der Weid NX, Ammann RA, Kuehni CE; Swiss Paediatric Oncology Group. Long-term pulmonary disease among Swiss childhood cancer survivors. Pediatr Blood Cancer. (08); (). doi:0.00/pbc Kaufmann A, Gerber NU, Kandels D, Azizi AA, Schmidt R, Warmuth-Metz M, Pietsch T, Kortmann RD, Gnekow AK, Grotzer MA. Management of Primary Tectal Plate Low-Grade Glioma in Pediatric Patients: Results of the Multicenter Treatment Study SIOP-LGG 004. Neuropediatrics. (08); 4(), 4-. doi:0.0/s Konstantinoudis G, Kreis C, Ammann RA, Niggli F, Kuehni CE, Spycher BD; Swiss Paediatric Oncology Group, Swiss National Cohort Study G. Spatial clustering of childhood cancers in Switzerland: a nationwide study. Cancer Causes Control. (08); (), -. doi:0.007/s Kube S, Vokuhl C, Dantonello T, Scheer M, Hallmen E, Feuchtgruber S, Escherich G, Niggli F, Kuehnle I, von Kalle T, Bielack S, Klingebiel T, Koscielniak E. Inflammatory myofibroblastic tumors-a retrospective analysis of the Cooperative Weichteilsarkom Studiengruppe. Pediatr Blood Cancer. (08); (), e70. doi:0.00/pbc Kuhlen M, Willasch AM, Dalle JH, Wachowiak J, Yaniv I, Ifversen M, Sedlacek P, Güngör T, Lang P, Bader P, Sufliarska S, Balduzzi A, Strahm B, von Luettichau I, Hoell JI, Borkhardt A, Klingebiel T, Schrappe M, von Stackelberg A, Glogova E, Poetschger U, Meisel R, Peters C. Outcome of relapse after allogeneic HSCT in children with ALL enrolled in the ALL-SCT 00/007 trial. Br J Haematol. (08); 80(), 8-8. doi:0./bjh.4..8 Kühne T. Advances in chemical pharmacotherapy for the treatment of pediatric immune thrombocytopenia. Expert Opin Pharmacother. (08); (7), 7-7. doi:0.080/ Ladenstein R, Potschger U, Valteau-Couanet D, Luksch R, Castel V, Yaniv I, Laureys G, Brock P, Michon JM, Owens C, Trahair T, Chan GCF, Ruud E, Schroeder H, Beck Popovic M, Schreier G, Loibner H, Ambros P, Holmes K, Castellani MR, Gaze MN, Garaventa A, Pearson ADJ, Lode HN. Interleukin with anti-gd antibody ch4.8/cho (dinutuximab beta) in patients with high-risk neuroblastoma (HR-NBL/SIOPEN): a multicentre, randomised, phase trial. Lancet Oncol. (08); (), 7-. doi:0.0/s470-04(8) Lagreze WA, Joachimsen L, Gross N, Taschner C, Rössler J. Sirolimus-induced regression of a large orbital lymphangioma. Orbit. (08), -. doi:0.080/ Lehrnbecher T, Groll A, Agyeman P, Ammann RA, Attarbaschi A, Behrends U, Berger C, Hamprecht A, Hufnagel M, Laws H-J, Scheler M, Temme C, Vieth S, Simon A. Empfehlungen zur Diagnostik und Therapie bei krebskranken Kindern mit Fieber und Neutropenie Vergleich zweier aktueller Leitlinien. [Recommendations for Diagnostics and Therapy of Children with Cancer Presenting with Fever and Neutropenia Comparison of Two Current Guidelines]. Klin Padiatr. (08); 0(0), -. doi:0.0/s Mader L, Roser K, Baenziger J, Vetsch J, Winther JF, Scheinemann K, Michel G. Relationship status and quality of the partner relationship in parents of long-term childhood cancer survivors: The Swiss Childhood Cancer Survivor StudyParents. Psychooncology. (08). doi:0.00/pon Makarova O, Oschlies I, Muller S, Ruf S, Zimmermann M, Niggli F, Attarbaschi A, Kabickova E, Klapper W, Woessmann W, Burkhardt B. Excellent outcome with limited treatment in paediatric patients with marginal zone lymphoma. Br J Haematol. (08); 8(), 7-7. doi:0./bjh Matzdorff A, Meyer O, Ostermann H, Kiefel V, Eberl W, Kühne T, Pabinger I, Rummel M. Immunthrombozytopenie aktuelle Diagnostik und Therapie: Empfehlungen einer gemeinsamen Arbeitsgruppe der DGHO, OGHO, SGH, GPOH und DGTI. Oncol Res Treat. (08); 4 Suppl, -. doi:0./ Moreno L, Casanova M, Chisholm JC, Berlanga P, Chastagner PB, Baruchel S, Amoroso L, Gallego Melcon S, Gerber NU, Bisogno G, Fagioli F, Geoerger B, Glade Bender JL, Aerts I, Bergeron C, Hingorani P, Elias I, Simcock M, Ferrara S, Le Bruchec Y, Slepetis R, Chen N, Vassal G. Phase I results of a phase I/II study of weekly nab-paclitaxel in paediatric patients with recurrent/refractory solid tumours: A collaboration with innovative therapies for children with cancer. Eur J Cancer. (08); 00, 7-4. doi:0.0/j.ejca

18 Authors / title / journal Publications in peer-reviewed journals JIF Munier FL, Moulin A, Gaillard MC, Bongiovanni M, Decembrini S, Houghton S, Beck Popovic M, Stathopoulos C. Intracameral Chemotherapy for Globe Salvage in Retinoblastoma with Secondary Anterior Chamber Invasion. Ophthalmology. (08); (4), -7. doi:0.0/j.ophtha Nava T, Kassir N, Rezgui MA, Uppugunduri CRS, Huezo-Diaz Curtis P, Duval M, Theoret Y, Daudt LE, Litalien C, Ansari M, Krajinovic M, Bittencourt H. Incorporation of GSTA genetic variations into a population pharmacokinetic model for IV busulfan in paediatric hematopoietic stem cell transplantation. Br J Clin Pharmacol. (08); 84(7), doi:0./ bcp...88 Nava T, Rivard GE, Bonnefoy A. Challenges on the diagnostic approach of inherited platelet function disorders: Is a paradigm change necessary? Platelets. (08); (), 48-. doi:0.080/ Nikkila A, Kendall G, Raitanen J, Spycher BD, Lohi O, Auvinen A. Effects of incomplete residential histories on studies of environmental exposure with application to childhood leukaemia and background radiation. Environ Res. (08);, doi:0.0/j.envres Otth M, Scheinemann K. Surveillance imaging for high-grade childhood brain tumors: What to do 0 years after completion of treatment? Pediatr Blood Cancer. (08); (), e7. doi:0.00/pbc Passweg JR, Baldomero H, Ansari M, Baerlocher GM, Bargetzi M, Chalandon Y, Duchosal MA, Gerull S, Güngör T, Halter JP, Heim D, Hess U, Leibundgut K, Masouridi-Levrat S, Muller A, Nair G, Pabst T, Renner C, Schmidt A, Stussi G, Nicoloso de Faveri G, Schanz U. For The Swiss Blood Stem Cell Transplantation Group S. Haematopoietic cell transplantation in Switzerland, changes and results over 0 years: a report from the Swiss Blood Stem Cell Transplantation Working Group for Blood and Marrow Transplantation registry 7-0. Swiss Med Wkly. (08); 48, w48. doi:0.444/ smw Pfeifer U, Gubler D, Bergsträsser E, Bassler D. Congenital malformations, palliative care and postnatal redirection to more intensive treatment a review at a Swiss tertiary center. J Matern Fetal Neonatal Med. (08); (), doi:0.0 80/ Place AE, Goldsmith K, Bourquin JP, Loh ML, Gore L, Morgenstern DA, Sanzgiri Y, Hoffman D, Zhou Y, Ross JA, Prine B, Shebley M, McNamee M, Farazi T, Kim SY, Verdugo M, Lash-Fleming L, Zwaan CM, Vormoor J. Accelerating drug development in pediatric cancer: a novel Phase I study design of venetoclax in relapsed/refractory malignancies. Future Oncol. (08); 4(), -. doi:0.7/fon Rasche M, Zimmermann M, Borschel L, Bourquin JP, Dworzak M, Klingebiel T, Lehrnbecher T, Creutzig U, Klusmann JH, Reinhardt D. Successes and challenges in the treatment of pediatric acute myeloid leukemia: a retrospective analysis of the AML-BFM trials from 87 to 0. Leukemia. (08); (0), doi:0.08/s Richter-Pechanska P, Kunz JB, Bornhauser B, von Knebel Doeberitz C, Rausch T, Erarslan-Uysal B, Assenov Y, Frismantas V, Marovca B, Waszak SM, Zimmermann M, Seemann J, Happich M, Stanulla M, Schrappe M, Cario G, Escherich G, Bakharevich K, Kirschner-Schwabe R, Eckert C, Muckenthaler MU, Korbel JO, Bourquin JP, Kulozik AE. PD models recapitulate the genetic and epigenetic landscape of pediatric T-cell leukemia. EMBO Mol Med. (08); 0(). doi:0./ emmm Rock N, Ansari M, Villard J, Ferrari-Lacraz S, Waldvogel S, McLin VA. Factors associated with immune hemolytic anemia after pediatric liver transplantation. Pediatr Transplant. (08); (), e0. doi:0./petr Roser K, Baenziger J, Mader L, Christen S, Dehler S, Michel G. Attendance to Follow-Up Care in Survivors of Adolescent and Young Adult Cancer: Application of the Theory of Planned Behavior. J Adolesc Young Adult Oncol. (08); 7(), 84-. doi:0.08/jayao Rost M, Acheson E, Kühne T, Ansari M, Pacurari N, Brazzola P, Niggli F, Elger BS, Wangmo T. Palliative care in Swiss pediatric oncology settings: a retrospective analysis of medical records. Support Care Cancer. (08); (8), doi:0.007/s x..7 7 Rost M, Wangmo T, Rakic M, Acheson E, Rischewski J, Hengartner H, Kühne T, Elger BS. Burden of treatment in the face of childhood cancer: A quantitative study using medical records of deceased children. Eur J Cancer Care (Engl). (08); 7(), e87. doi:0./ecc Santhana Kumar K, Neve A, Guerreiro Stücklin AS, Kuzan-Fischer CM, Rushing EJ, Taylor MD, Tripolitsioti D, Behrmann L, Kirschenbaum D, Grotzer MA, Baumgartner M. TGF- Determines the Pro-migratory Potential of bfgf Signaling in Medulloblastoma. Cell Reports. (08); (), 78-8.e78. doi:0.0/j.celrep Schifferli A, Holbro A, Chitlur M, Coslovsky M, Imbach P, Donato H, Elalfy M, Graciela E, Grainger J, Holzhauer S, Riccheri C, Rodeghiero F, Ruggeri M, Tamary H, Uglova T, Wu R, Kühne T, Intercontinental Cooperative ITPSG. A comparative prospective observational study of children and adults with immune thrombocytopenia: -year follow-up. Am J Hematol. (08); (), 7-7. doi:0.00/ajh

19 Authors / title / journal Publications in peer-reviewed journals JIF 7 Schrappe M, Bleckmann K, Zimmermann M, Biondi A, Moricke A, Locatelli F, Cario G, Rizzari C, Attarbaschi A, Valsecchi MG, Bartram CR, Barisone E, Niggli F, Niemeyer C, Testi AM, Mann G, Ziino O, Schafer B, Panzer-Grumayer R, Beier R, Parasole R, Gohring G, Ludwig WD, Casale F, Schlegel PG, Basso G, Conter V. Reduced-Intensity Delayed Intensification in Standard-Risk Pediatric Acute Lymphoblastic Leukemia Defined by Undetectable Minimal Residual Disease: Results of an International Randomized Trial (AIEOP-BFM ALL 000). J Clin Oncol. (08); (), 44-. doi:0.00/ JCO Seidel C, von Büren AO, Bojko S, Hoffmann M, Pietsch T, Gielen GH, Warmuth-Metz M, Bison B, Kortmann RD, Kramm CM. Concurrent radiotherapy with temozolomide vs. concurrent radiotherapy with a cisplatinum-based polychemotherapy regimen: Acute toxicity in pediatric high-grade glioma patients. Strahlenther Onkol. (08); 4(), -4. doi:0.007/ s Simons L, Ma K, de Chappedelaine C, Moiranghtem RD, Elkaim E, Olivre J, Susini S, Appourchaux K, Reimann C, Sadek H, Pelle O, Cagnard N, Magrin E, Lagresle-Peyrou C, Taghon T, Rausell A, Cavazzana M, Andre-Schmutz I. Generation of adult human T-cell progenitors for immunotherapeutic applications. J Allergy Clin Immunol. (08); 4(4), 4-44 e44. doi:0.0/j.jaci Slack J, Albert MH, Balashov D, Belohradsky BH, Bertaina A, Bleesing J, Booth C, Buechner J, Buckley RH, Ouachee-Chardin M, Deripapa E, Drabko K, Eapen M, Feuchtinger T, Finocchi A, Gaspar HB, Ghosh S, Gillio A, Gonzalez-Granado LI, Grunebaum E, Güngör T, Heilmann C, Helminen M, Higuchi K, Imai K, Kalwak K, Kanazawa N, Karasu G, Kucuk ZY, Laberko A, Lange A, Mahlaoui N, Meisel R, Moshous D, Muramatsu H, Parikh S, Pasic S, Schmid I, Schuetz C, Schulz A, Schultz KR, Shaw PJ, Slatter MA, Sykora KW, Tamura S, Taskinen M, Wawer A, Wolska-Kusnierz B, Cowan MJ, Fischer A, Gennery AR, Inborn Errors Working Party of the European Society for B, Marrow T, the European Society for I, Stem Cell Transplant for Immunodeficiencies in E, Center for International B, Marrow Transplant R, Primary Immunodeficiency Treatment C. Outcome of hematopoietic cell transplantation for DNA double-strand break repair disorders. J Allergy Clin Immunol. (08); 4(), -8 e0. doi:0.0/j.jaci Sommer C, Gumy Pause F, Diezi M, Rougemont AL, Wildhaber BE. A National Long-Term Study of Neuroendocrine Tumors of the Appendix in Children: Are We Too Aggressive? Eur J Pediatr Surg. (08). doi:0.0/s Sparber-Sauer M, Seitz G, von Kalle T, Vokuhl C, Leuschner I, Scheer M, Munter M, Ljungman G, Bielack SS, Niggli F, Ladenstein R, Klingebiel T, Fuchs J, Koscielniak E, Group CWSS. Systemic therapy of aggressive fibromatosis in children and adolescents: Report of the Cooperative Weichteilsarkom Studiengruppe (CWS). Pediatr Blood Cancer. (08); (), e4. doi:0.00/pbc Sparber-Sauer M, Seitz G, von Kalle T, Vokuhl C, Scheer M, Munter M, Bielack SS, Kazanowska B, Ladenstein R, Niggli F, Klingebiel T, Fuchs J, Koscielniak E, Group CWSS. Alveolar soft-part sarcoma: Primary metastatic disease and metastatic relapse occurring during long-term follow-up: Treatment results of four Cooperative Weichteilsarkom Studiengruppe (CWS) trials and one registry. Pediatr Blood Cancer. (08); (), e740. doi:0.00/pbc Stanulla M, Dagdan E, Zaliova M, Moricke A, Palmi C, Cazzaniga G, Eckert C, Te Kronnie G, Bourquin JP, Bornhauser B, Koehler R, Bartram CR, Ludwig WD, Bleckmann K, Groeneveld-Krentz S, Schewe D, Junk SV, Hinze L, Klein N, Kratz CP, Biondi A, Borkhardt A, Kulozik A, Muckenthaler MU, Basso G, Valsecchi MG, Izraeli S, Petersen BS, Franke A, Dorge P, Steinemann D, Haas OA, Panzer-Grumayer R, Cave H, Houlston RS, Cario G, Schrappe M, Zimmermann M, Consortium T, International BFMSG. IKZF(plus) Defines a New Minimal Residual Disease-Dependent Very-Poor Prognostic Profile in Pediatric B-Cell Precursor Acute Lymphoblastic Leukemia. J Clin Oncol. (08); (), doi:0.00/ JCO Stathopoulos C, Gaillard MC, Moulin A, Puccinelli F, Beck Popovic M, Munier FL. Intravitreal anti-vascular endothelial growth factor for the management of neovascularization in retinoblastoma after intravenous and/or intraarterial chemotherapy: Long-Term Outcomes in a Series of Eyes. Retina. (08). doi:0.07/iae Stathopoulos C, Gaillard MC, Puccinelli F, Maeder P, Hadjistilianou D, Beck Popovic M, Munier F. Response to Khetan and Maitray's «Comment: Conservative management of massive choroidal relapse in retinoblastoma patients?». Ophthalmic Genet. (08); (), 47. doi:0.080/ Stathopoulos C, Gaillard MC, Puccinelli F, Maeder P, Hadjistilianou D, Beck Popovic M, Munier FL. Successful conservative treatment of massive choroidal relapse in retinoblastoma patients monitored by ultrasound biomicroscopy and/or spectral domain optic coherence tomography. Ophthalmic Genet. (08); (), 4-4. doi:0.080/ Tirado-Gonzalez I, Czlonka E, Nevmerzhitskaya A, Soetopo D, Bergonzani E, Mahmoud A, Contreras A, Jeremias I, Platzbecker U, Bourquin JP, Kloz U, Van der Hoeven F, Medyouf H. CRISPR/Cas-edited NSG mice as PD models of human leukemia to address the role of niche-derived SPARC. Leukemia. (08); (4), doi:0.08/leu Tonorezos ES, Barnea D, Cohn RJ, Cypriano MS, Fresneau BC, Haupt R, Hjorth L, Ishida Y, Kruseova J, Kuehni CE, Kurkure PA, Langer T, Nathan PC, Skeen JE, Skinner R, Tacyildiz N, van den Heuvel-Eibrink MM, Winther JF, Hudson MM, Oeffinger KC. Models of Care for Survivors of Childhood Cancer From Across the Globe: Advancing Survivorship Care in the Next Decade. J Clin Oncol. (08); (), -0. doi:0.00/jco

20 Authors / title / journal Publications in peer-reviewed journals JIF 88 Tripolitsioti D, Kumar KS, Neve A, Migliavacca J, Capdeville C, Rushing EJ, Ma M, Kijima N, Sharma A, Pruschy M, McComb S, Taylor MD, Grotzer MA, Baumgartner M. MAP4K4 controlled integrin beta activation and c-met endocytosis are associated with invasive behavior of medulloblastoma cells. Oncotarget. (08); (), 0-. doi:0.8/ oncotarget Tyagi AK, Khoshbeen MB, Curtis PH-D, Uppugunduri CRS, Ansari M. Development and validation of an allele-specific PCR assay for genotyping a promoter and exonic single nucleotide polymorphisms of MGMT gene. J Biol Methods. (08); (): e. doi:0.4440/jbm Vaezipour N, Leibundgut K. Nonalimental Scurvy With Relapse Symptoms After Stopping Oral Vitamin C Supplementation. Pediatrics. (08); 4(). doi:0.4/peds van den Berg M, van Dijk M, Byrne J, Campbell H, Berger C, Borgmann-Staudt A, Calaminus G, Dirksen U, Winther JF, Fossa SD, Grabow D, Grandage VL, van den Heuvel-Eibrink MM, Kaiser M, Kepak T, Kremer LC, Kruseova J, Kuehni CE, Lambalk CB, van Leeuwen FE, Leiper A, Modan-Moses D, Morsellino V, Spix C, Kaatsch P, van Dulmen-den Broeder E, PanCare LC. Fertility Among Female Survivors of Childhood, Adolescent, and Young Adult Cancer: Protocol for Two Pan-European Studies (PanCareLIFE). JMIR Res Protoc. (08); 7(), e084. doi:0./ van Straaten S, Bierings M, Bianchi P, Akiyoshi K, Kanno H, Serra IB, Chen J, Huang X, van Beers E, Ekwattanakit S, Güngör T, Kors WA, Smiers F, Raymakers R, Yanez L, Sevilla J, van Solinge W, Segovia JC, van Wijk R. Worldwide study of hematopoietic allogeneic stem cell transplantation in pyruvate kinase deficiency. Haematologica. (08); 0(), e8-e8. doi:0.4/haematol Vanden Bempt M, Demeyer S, Broux M, De Bie J, Bornschein S, Mentens N, Vandepoel R, Geerdens E, Radaelli E, Bornhauser B, Kulozik AE, Meijerink JP, Bourquin JP, de Bock CE, Cools J. Cooperative Enhancer Activation by TLX and STAT Drives Development of NUP4-ABL/TLX-Positive T Cell Acute Lymphoblastic Leukemia. Cancer Cell. (08); 4(), 78 e77. doi:0.0/j.ccell Vetsch J, Rueegg CS, Mader L, Bergsträsser E, Diezi M, Kuehni CE, Michel G; Swiss Paediatric Oncology Group. Parents' preferences for the organisation of long-term follow-up of childhood cancer survivors. Eur J Cancer Care (Engl). (08); 7(), e4. doi:0./ecc von Allmen AN, Zermatten MG, Leibundgut K, Agyeman P, Ammann RA. Pediatric patients at risk for fever in chemotherapy-induced neutropenia in Bern, Switzerland, -0. Sci Data. (08);, doi:0.08/sdata von Büren AO, Karremann M, Gielen GH, Benesch M, Fouladi M, van Vuurden DG, van Zanten S, Hoffman LM, Kramm CM. A suggestion to introduce the diagnosis of «diffuse midline glioma of the pons, H K7 wildtype (WHO grade IV)». Acta Neuropathol. (08); (), 7-7. doi:0.007/s Wagner S, Brack EK, Stutz-Grunder E, Agyeman P, Leibundgut K, Teuffel O, Ammann RA. The influence of different fever definitions on diagnostics and treatment after diagnosis of fever in chemotherapy-induced neutropenia in children with cancer. PLoS One. (08); (), e07. doi:0.7/journal.pone Waszak SM, Northcott PA, Buchhalter I, Robinson GW, Sutter C, Groebner S, Grund KB, Brugieres L, Jones DTW, Pajtler KW, Morrissy AS, Kool M, Sturm D, Chavez L, Ernst A, Brabetz S, Hain M, Zichner T, Segura-Wang M, Weischenfeldt J, Rausch T, Mardin BR, Zhou, Baciu C, Lawerenz C, Chan JA, Varlet P, Guerrini-Rousseau L, Fults DW, Grajkowska W, Hauser P, Jabado N, Ra YS, Zitterbart K, Shringarpure SS, De La Vega FM, Bustamante CD, Ng HK, Perry A, MacDonald TJ, Hernaiz Driever P, Bendel AE, Bowers DC, McCowage G, Chintagumpala MM, Cohn R, Hassall T, Fleischhack G, Eggen T, Wesenberg F, Feychting M, Lannering B, Schuz J, Johansen C, Andersen TV, Roosli M, Kuehni CE, Grotzer MA, Kjaerheim K, Monoranu CM, Archer TC, Duke E, Pomeroy SL, Shelagh R, Frank S, Sumerauer D, Scheurlen W, Ryzhova MV, Milde T, Kratz CP, Samuel D, Zhang J, Solomon DA, Marra M, Eils R, Bartram CR, von Hoff K, Rutkowski S, Ramaswamy V, Gilbertson RJ, Korshunov A, Taylor MD, Lichter P, Malkin D, Gajjar A, Korbel JO, Pfister SM. Spectrum and prevalence of genetic predisposition in medulloblastoma: a retrospective genetic study and prospective validation in a clinical trial cohort. Lancet Oncol. (08); (), doi:0.0/s470-04(8) Weiss A, Kuonen R, Brockmeier H, Grotzer MA, Candreia C, Maire R, Senn P, Stieger C, Rosenfeld J, Veraguth D, Kompis M, Scheinemann K, Kuehni CE; Swiss Paediatric Oncology Group. Audiological monitoring in Swiss childhood cancer patients. Pediatr Blood Cancer. (08); (). doi:0.00/pbc Weiss A, Sommer G, Schindera C, Wengenroth L, Karow A, Diezi M, Michel G, Kuehni CE; Swiss Paediatric Oncology Group. Hearing loss and quality of life in survivors of paediatric CNS tumours and other cancers. Qual Life Res. (0); 8(), -. doi:0.007/s Wray-Dutra MN, Chawla R, Thomas KR, Seymour BJ, Arkatkar T, Sommer KM, Khim S, Trapnell C, James RG, Rawlings DJ. Activated CARD accelerates germinal center kinetics, promoting mtorc and terminal differentiation. J Exp Med. (08); (), doi:0.084/jem

21 Authors / title / journal Publications in peer-reviewed journals JIF 0 Zimmermann K, Cignacco E, Engberg S, Ramelet AS, von der Weid NX, Eskola K, Bergsträsser E, Consortium P, Ansari M, Aebi C, Baer R, Beck Popovic M, Bernet V, Brazzola P, Bucher HU, Buder R, Cagnazzo S, Dinten B, Dorsaz A, Elmer F, Enriquez R, Fahrni-Nater P, Finkbeiner G, Frey B, Frey U, Greiner J, Hassink RI, Keller S, Kretschmar O, Kroell J, Laubscher B, Leibundgut K, Malär R, Meyer A, Stuessi C, Nelle M, Neuhaus T, Niggli F, Perrenoud G, Pfammatter JP, Plecko B, Rupf D, Sennhauser F, Stade C, Steinlin M, Stoffel L, Thomas K, Vonarburg C, von Vigier R, Wagner B, Wieland J, Wernz B. Patterns of paediatric end-of-life care: a chart review across different care settings in Switzerland. BMC Pediatr. (08); 8(), 7. doi:0.8/s Total JIF The Journal Impact Factor (JIF) of a specialist journal measures how often other journals cite an article from it in relation to the total number of articles published in it. It is a measure of the recognition of the quality of published research results. Non-peer-reviewed journals Meier JH, Ansari M, Beck Popovic M, Bergsträsser E, Brazzola P, Eisenreich B, Janz I, Hengartner H, Tinner EM, von der Weid NX, Redmond S, Scheinemann K: Aftercare in Pediatric Oncology in Switzerland Current State, Challenges and Future Directions. Schweizer Krebsbulletin 08;, Jg. 8: 7-7. Diesch T: Fertilitätserhaltung. Ein zunehmend wichtiger Aspekt bei krebskranken Kindern und Jugendlichen. Pädiatrie 0/08. Reimann C, Szavay P, Hartmann K, Eisenreich B, Rosenblatt L, Schilling FH: Three case reports on organ preserving surgery and brachytherapy in children with genitourinary rhabdomyosarcomas. Schweizer Krebsbulletin 08;, Jg. 8: Scheinemann K: Pediatric primary CNS tumors a quick update. Schweizer Krebsbulletin (): -. Scheinemann K: Spätfolgen nach einer Krebserkrankung im Kindesalter. Pädiatrie 08; : -4. Scheinemann K: Spätfolgen nach einer Krebserkrankung im Kindesalter: Heilung mit Nebenwirkungen. VSAO Journal 08; : von der Weid NX. Akute myeloische Leukämie im Kindes- und Jugendalter. Info@Onkologie_04_08; 0-. von der Weid NX. Immuntherapien in der pädiatrischen Onkologie. Neue Strategien. SZO 08; 4: 7-0. Book chapters and others Ansari M, Gordon E : Le cancer de l enfant et de l adolescent. J ai envie de comprendre. Éditions Planète santé 08. Guerreiro Stücklin AS, Grotzer MA. (08). Chapter Cerebellar tumors. In: Manto M, Huisman TAGM (Eds.), Handbook of Clinical Neurology (Vol., pp. 8-): Elsevier. Kühne T. Primäre Immunthrombozytopenie. In: Pädiatrische Hämatologie und Onkologie (Niemeyer C und Eggert A., Hrsg.). Springer-Verlag Deutschland 08;7-0. Kühne T: Registries in Immune Thrombocytopenia: The History of the Intercontinental Cooperative ITP Study Group. In: Imbach P. Antibody Therapy. Substitution Immunomodulation Monoclonal Immunotherapy. Springer Verlag, Berlin, Heidelberg 08; Lehrnbecher T, Ammann RA, Berger C, Groll AH, Kontny U, Laws HJ, Simon A, Strenger V. Infektionen bei pädiatrisch-onkologischen Patienten. In DGPI. DGPI Handbuch Infektionen bei Kindern und Jugendlichen.7. Auflage. Stuttgart, New York: Thieme 08; 7-7. Simon A, Furtwängler R, Laws HJ, Greiner J, Lehrnbecher T, Ammann RA, Schilling FH, Graf N; im Auftrag der GPOH. Evidenzbasierte Empfehlungen zur Anwendung dauerhaft implantierter, zentralvenöser Zugänge in der pädiatrischen Onkologie.. Überarbeitete Auflage. Wiesbaden: mhp Verlag GmbH 08; 84 pp.

22 Working group reports Professional Development Working Group (PDWG) Protocol Working Group (PWG) Professional development subspecialty examination The PWG met three times in 08, ahead of each of the research council meetings. Seven candidates registered for the subspecialty examination in 08, three of whom subsequently withdrew their registration, two of thoseat very short notice. The examination was held in German and English at the children's hospital in Aarau, with Dr. Katrin Scheinemann and Dr. Jeanette Greiner as chief examiner and co-examiner for two candidates each. Candidates are expected to demonstrate in-depth and comprehensive knowledge of all aspects of paediatric haematology and oncology in order to pass this examination. Two candidates fulfilled this requirement, which is described comprehensively and bindingly in Annex 8 of the Continuing Education Rules of the Swiss Medical Association FMH (in force since July 004, dated January 00), without difficulty, one candidate was adequate and one did not pass the examination. In contrast to the trend in previous years, the candidates in the year under review were better prepared and presented for examination after sufficient training in paediatric haematology and oncology. Yet it was once again clear that almost no candidates have a solid enough foundation in all the major areas of oncology and haematology. Both this situation and the increasing complexity of both fields is taken into account in the Continuing Education Rules of the FMH for paediatric haematology and oncology, which contain revised and updated requirements and newly defined admission criteria for the subspecialty examination. Continuing education credits Five applications for credit were submitted in the year under review and evaluated by the members of the PDWG. A total of.7 credits were awarded to the 4 haematology/oncology postgraduate training events. Dr. Jeanette Greiner Chair of the PDWG. Meeting on 7..08: The proposed protocol for craniopharyngioma has not been concluded yet and the Working Group decided to postpone the discussion until further notice. New members were admitted to the PWG at this meeting: Dr. Nicolas Gerber from Zurich, Dr. Christian Reimann from Lucerne, Prof. Jochen Rössler from Bern and Dr. Katrin Scheinemann from Aarau and Basel.. Meeting on..08: Two protocols were discussed at this meeting: a) The LOGGIC study for low-grade gliomas, which defines for the first time a standardized approach to the diagnosis, biological investigation and therapy of this common type of brain tumour. It was decided to recommend this protocol to SPOG for approval, with Dr. Katrin Scheinemann (Aarau/Basel) as National Study Chair (principal investigator for Switzerland) and Dr. Ana Guerreiro Stücklin (Zurich) as Vice-Chair (deputy principal investigator for Switzerland). b) EsPhALL 07 is a protocol for Ph+ ALL in children, a rare category of high-risk leukaemias in children in which imatinib is administered in addition to chemotherapy. The protocol compares chemotherapy with two recognized chemotherapy regimens. It was decided to recommend this protocol to SPOG with Nicole Bodmer (Zurich) as National Study Chair.. Meeting on..08: One protocol was discussed at this meeting. The LOGGIC Core BioClinical Database is the first joint European protocol for the biological aspects of low-grade gliomas which, in addition, was developed in conjunction with the Children s Oncology Group (COG). Since this protocol is linked to the LOGGIC study, which has already been accepted as a SPOG protocol, it was decided to recommend this protocol to SPOG. It was also announced at this meeting that Dr. Axel Karow, National Study Chair of the Umbrella protocol, will be leaving Bern in 0. The proposal that Prof. Jochen Rössler should take over as Chair and Dr. Sabine Kroiss as Vice-Chair of Umbrella was accepted and recommended for adoption by SPOG.

23 All the proposals were adopted by the SPOG research council. Dr. Tamara Diesch from UKBB (Basel) presented a study by the Paediatric Diseases Working Party of the EBMT on Pregnancy Rates and Pregnancy Outcomes after Haematopoietic Stem Cell Transplantation in Childhood: A Cross-Sectional Survey of the EBMT Paediatric WP at the ASH Congress. This is a large-scale study being carried out by the EBMT Paediatric Diseases Working Party. Prof. Marc Ansari and Prof. Tayfun Güngör are members of the Board of this EBMT Paediatric Diseases Working Party. Prof. Maja Beck Popovic Chair of the PWG Stem Cell Therapy Working Group (SCTWG) The data and results of paediatric haematopoietic stem cell transplantations performed in the past 0 years were compiled by the Geneva team (F. Bernard, HUG) and presented at the SBST meeting in Bern in January 08. Depending on the completeness of the data, publication is planned in a national or international journal on behalf of the SPOG SCTWG. Prof. Tayfun Güngör Chair of the SCTW Germline DNA Working Group (GDWG) The randomization arm of the stem cell transplantation study in acute lymphoblastic leukaemia (ALL SCTped 0 FORUM) was closed prematurely because the chemotherapy arm with busulfan/ thiotepa/fludarabine and treosulfane/thiotepa/fludarabine in children aged 4 and above showed significantly lower overall survival and higher rates of relapse and was thus inferior to standard total body irradiation. A total of n= patients were enrolled in the ALL-Forum study in Switzerland (Zurich n=, Basel n=, Geneva n=). The partial study analyses of the pharmacokinetics of busulfan and the other chemotherapeutic agents used and antithymocyte globulin (ATG), the antibody administered, have not yet been completed. The intention is to recruit further patients to the ALL-Forum study. The GDWG was set up in July 0 with the intention of establishing the collection of germline DNA from children with cancer throughout Switzerland. The working group comprises members of the SPOG centres in Basel, Zurich (where the Swiss Paediatric Haematology / Oncology Biobank (SPHO) is also headquartered), Basel, Lausanne and Geneva (where the Biobank in Hematology and Oncology Pediatric (BaHOP) is domiciled which includes the Swiss Germline DNA Biobank (BISKIDS) will reside) and representatives of the Swiss Childhood Cancer Registry (SCCR). Meetings are held roughly every three months in different locations. The Inborn Errors Working Party of the EBMT (Principal Investigators: R. Chiesa, M. Slatter and T. Güngör) carried out a retrospective analysis of more than 00 children and adults with CGD ( Allogeneic Haematopoietic Stem Cell Transplantation in Children and Adults with Chronic Granulomatous Disease (CGD): a study of the Inborn Errors Working Party (IEWP) of the EBMT ) that was presented at the 08 ASH Meeting in December 08. The SPOG centres in Geneva, Lausanne, Bern, Lucerne, Aarau, St. Gallen, Basel, Zurich and Bellinzona are collaborating closely on these patients. With the mandate of the GDWG and support of all nine SPOG member institutions, Prof. Marc Ansari (head of the SPOG member institution in Geneva, and the biobank (BaHOP)) established a new germline DNA biobank for childhood cancer and haematological diseases inside BaHOP in Switzerland: The Germline DNA Biobank Switzerland for Childhood Cancer and Haematological Diseases (BISKIDS). BISKIDS is financed by the CANSEARCH foundation and is open to accept germline DNA samples from all Switzerland. The following objectives were achieved during the past year: The GDWG has agreed to mandate the BISKIDS Biobank to be the unique repository for germline DNA samples and data for all

24 Switzerland. It was agreed that the SPOG member institutions will send genetic samples directly to BISKIDS without intermediate storage in a local biobank. concepts in the field of precision medicine, biobanks, as well as its advanced clinical applications in personalised health. The GDWG emphasized the need to foster the collaboration between the newly established BISKIDS Biobank (the resource for germline DNA samples and data), the Swiss Childhood Cancer Registry (for clinical data), and the Swiss Paediatric Haematology Oncology Biobanking Network (SPHO, for tumor samples). On this basis, Prof. Marc Ansari, Prof. Claudia Kühni (Head of the SCCR), and Prof. Jean-Pierre Bourquin (Head of the SPHO) drafted and submitted a proposal to the BioLink call 08 by the Swiss National Science Foundation (SNF). The BioLink grant is specifically intended for the networking of biobanks established for research purposes. The submitted project is entitled «Swiss Pediatric Hematology/ Oncology Metabank a network for precision medicine research». It describes the collaboration, data linkage, and privacy aspects of a Swiss data and biobanking platform for childhood cancer and haematological diseases. We are happy to announce that it was accepted by the SNF. In a collaboration of the SCCR and the CASEARCH research laboratory of the Geneva University Medical School, a proposal to use future genetic and clinical data on long-term complications after childhood cancers with the name GECCOS (Genetic risks for complications in children after oncological treatment in Switzerland) has been made. It is currently being revised in order to go through ethical comity in 0. This genotype-phenotype association study will enrol approximately 00 childhood cancer survivors. The project will be the first involving the new biobank and has been made possible by funding from the CANSEARCH Foundation. Dr Nicolas Waespe has been enrolled in 08 thanks to this grant to coordinate and move forward the GECCOS project. All SPOG member centres are supporting BISKIDS and GECCOS and have agreed to collaborate. Support will also be provided by the Swiss Biobanking Platform, the newly established Genome Centre at the Biotech Campus in Geneva and the Swiss Institute of Bioinformatics. Prof Ansari organised on behalf of the Swiss Group of pharmacogenomics and individualised therapy (SPT), together with the SPHN, CANSEARCH and the support of the SPOG at the Biotech Campus, last September, the ESPT summer school on «Precision Medecine and Personalised Health». The school s comprehensive and innovative educational programme aimed to address the fundamentals of pharmacogenomics, the latest knowledge on established and new 4 Prof. Marc Ansari Chair of the GDWG Paediatric Haematology Working Group (PHWG) In 08, the SPOG Board has officially confirmed the PHWG s membership, consisting of representatives from all SPOG centers, and finalized the group s bylaws. An important and specific aspect of PHWG activity will be that the group s work will be centered on meetings and tasks open to all interested SPOG station staff, with formal group members retaining voting rights in decisional matters. This organizational particularity reflects the PHWG s intention to remain inclusive, and ultimately act as the specialized forum for pediatric hematology in Switzerland. In this first year, the group has focused on several priorities. Of note, we collaboratively surveyed aspects of clinical practice in the area of myelodysplasia and bone marrow failure associated to a risk of oncologic transformation in Switzerland. The aim is to produce a list of PHWG-recommended sites for the laboratory and genetic investigation of these very rare, and sometimes inherited disorders in 0. In fact, all SPOG stations currently face difficulties in the reimbursement by healthcare insurers of clinically indicated tests. This is particularly the case for those investigations which are performed in reference laboratories abroad or involve constitutional DNA sequencing. The PHWG also gave space for discussion on the initiatives of individual SPOG stations in the development of next-generation sequencing platforms in pediatric hematology. Furthermore, and in the context of SPOG s participation in the EWOG consortium clinical trials for SAA/MDS/JMML, the PHWG has, in collaboration with the national study chair, initiated the establishment of a reference hematopathology center (in Lausanne), which shall be implemented in 0.

25 From a scientific standpoint, the PHWG has supported and assisted in the submission of a research study on the epidemiology of children with Diamond-Blackfan Anemia in Switzerland, which will take place in 0 in all participating SPOG stations. This will be the first scientific initiative to be led in the context of the PHWG. In addition, delegates of the PHWG participated in an expert panel focusing on iron deficiency in children, specifically its current evidence-based clinical diagnosis and therapy. The aim is to generate a consensus guideline on this condition and its management to be put to discussion and vote in the PHWG in 0, with a subsequent publication in Paediatrica, the Swiss Society of Paediatrics journal. Dr. Raffaele Renella Chair of the PHWG Shared Care Working Group (SCWG) The Shared Care Working Group (SCWG), set up by the SPOG Board in January 08 as a temporary working group, was mandated to develop proposals on the following topics for submission to the research council: Definition of structures/procedures/tools for shared care (therapy of a patient at different sites) between SPOG centres and with non-spog centres, for both research and clinical aspects Corresponding modifications of Article 4A and 4B of the current SPOG statutes Coordination of these tasks with a separately organized site selection process (selection of centres that can take part in specific studies) Positioning of Chur (not currently a SPOG member) within these shared care structures Additional tasks selected by the SCWG as required Shared care was also the subject of the SPOG Scientific Advisory Board Meeting, held every two years. The Board members presented a number of models that are operating abroad based on reports of direct experience. These range from structures without shared care (in which all diagnostic and therapeutic measures are carried out at a single centre) to two-stage and three-stage structures. All these models are working to the satisfaction of those involved. In structures with shared care it is important and indispensable for the rules governing collaboration to be defined clearly in an agreement between the participating centres so that they can be implemented in everyday clinical practice. The SCWG currently has seven members. It met four times in 08 and submitted proposals on some of the above tasks to the research council. Prof. Roland A. Ammann Chair of the SCWG

26 Reports from the SPOG Coordinating Center Clinical Project Management and Quality Management SPOG has undertaken to have the SPOG CC and the SPOG centres audited by an external service provider every three years. The last audits took place in 04-0, so the organization of the next round was started in early 08. The first SPOG centre and the SPOG CC were audited in autumn 08. The outstanding audits of the remaining SPOG centres will take place in 0. Dr. Samantha Chan was appointed Assistant Quality Management in March 08. This important function ensures that the internal Standard Operating Procedures (SOP) are revised in good time, that their content is correct and that they comply with the latest regulations. Opening of new studies The clinical studies PHITT, reecur and SIOP Ependymoma II were opened in 08. All the regulatory approvals for the Paediatric Relapsed AML 00/0 study had been obtained by the end of the year, but the study will not be initiated until 0 owing to the unavailability of a study medication which had to be replaced. The research project MNP.0 was opened at all German-speaking SPOG member hospitals. At the end of 08 the SPOG hospitals in the western and southern parts of Switzerland were still preparing to open the project. Preparations to open the ALL-REZ BFM research project were made at five SPOG centres in German-speaking Switzerland and the member hospital in Ticino and submitted to the authorities for approval at the end of 08. Treatment studies Thanks to two additional positions the Clinical Project Management (CPM) team was able to archive a large number of old studies and process without delay the new studies approved by the research council. A total of four proposals were discussed at three meetings of the Protocol Working Group (PWG), which are also attended by employees from the CC who contribute to the study evaluations their knowledge of regulatory and legal aspects and the management of human research. The decision on one research proposal was postponed due to missing information, the two other clinical studies and one research proposal were recommended by the PWG for approval by the research council. The SPOG research council accordingly assessed three study proposals positively in 08. These were the LOGGIC Europe trial, EsPhALL 07 and LOGGIC Core Bioclinical Data Bank. Twenty-nine participants from eight SPOG centres, the SCCR and the SPOG CC attended the 08 SPOG CRC (Clinical Research Coordinators) Meeting in Bern at the end of November. The main focus of the meeting was quality management. There was detailed discussion of the correct documentation and reporting of Serious Adverse Events (SAEs). Verena Pfeiffer from the SCCR also gave a preview of the new Cancer Registration Act that will come into force on January 00. reecur (International Randomized Controlled Trial of Chemotherapy for the Treatment of Recurrent and Primary Refractory Ewing Sarcoma) The clinical study was opened at all SPOG member hospitals in 08. PHITT (the Paediatric Hepatic International Tumour Trial) The clinical study was opened at all French- and German-speaking SPOG member hospitals in 08. The study is also expected to open at the SPOG centre in Bellinzona in January 0. SIOP Ependymoma II (an international clinical treatment-optimization study programme for the diagnosis and therapy of children, adolescents and young adults with ependymoma). The clinical study was opened in 08 in the SPOG member hospitals in Zurich, Basel and Aarau. Opening at the other SPOG member hospitals is in preparation.

27 Research projects MNP.0 (molecular neuropathology.0 to increase diagnostic accuracy of childhood brain tumours) The research project was opened at all German-speaking SPOG member hospitals in 08. The SPOG member hospitals in the western and southern parts of Switzerland are preparing to open the study. AML 00/0, IntReALL HR 00 and EsPhALL07). The research projects PTT.0, Umbrella and LOGGIC Core Bioclinical Data Bank are also being prepared for submission to the authorities. The SPOG research council takes decisions on the opening of further studies on an ongoing basis, in response to study proposals following assessment by the PWG. Looking ahead to new studies In 0 further clinical studies will be prepared for opening at the SPOG member institutions: studies on brain tumours (HITHGG-0, LOGGIC Europe trial), non-hodgkin lymphoma (B-BFM 0) and leukaemias (AIEOP-BFM ALL 07, Paediatric Relapsed Overview of submissions to the authorities The following table shows all relevant submissions to the authorities in 08. Ethics committees Swissmedic Federal Office of Public Health Total Centre openings 8 NA 0 8 Non-substantial amendments 8 4 Substantial amendments Annual safety reports (ASR and DSUR) Initial submissions 4 0 Total submissions 4 4 ASR = Annual Safety Report DSUR = Drug Safety Update Report One amendment = submission for one amendment (change) for all participating centres (maximum of nine) Julia Marina Ruckstuhl Head Clinical Operations, Associate Managing Director Dr. Michael Zeller Team Leader Clinical Project Management ad interim 7

28 Fundraising Funding partners State Secretariat for Education, Research and Innovation (SERI) In 08 SERI was once again the major funding partner of SPOG. Following a dispatch from the Federal Council to Parliament on the promotion of education, research and innovation, SERI mandated SPOG (cancer research for children) and SAKK (cancer research for adults). The current service level agreement covers the period from 07 to 00. Art. of the Federal Act on the Promotion of Research and Innovation (RIPA) forms the legal basis of this promotion of research by the federal government. SPOG is considered to be a research institute of national importance. Under the terms of the RIPA, the federal contribution may not exceed 0% of requirements. Swiss Cancer Research (KFS) KFS has been a reliable funding partner of SPOG for many years. Since the current research agreement expired at the end of 08, SPOG submitted a renewed application for 0 and is extremely grateful for the increased level of funding granted in the new agreement. Swiss Cancer Research and the federal government are the most important sources of funding for SPOG. Zoé4life Zoé4life particularly supports research done by SPOG into the especially difficult therapy of relapses in children and adolescents with cancer. This exceptionally active and successful organization is driven by the initiative of affected families and individuals keen to show their solidarity. Zoé4life was born in response to the story and medical history of Zoé, a little girl who died just before her th birthday after it was no longer possible to treat a recurrence of her cancer successfully. Zoé4life has been providing a high level of funding to SPOG for several years and will maintain this reliable support in 0 too. 8 Childhood Cancer Support Switzerland Childhood Cancer Support Switzerland again provided SPOG with generous support in 08. This commitment is of both financial and major non-material significance for SPOG, illustrating as it does the high value placed on SPOG s research activities by those who are directly affected. Swiss Clinical Cancer Research Foundation (SCCRF) This Foundation is another partner which has been providing SPOG with reliable support for many years. Substantial funding is awarded to selected SPOG projects every year. Childhood Cancer Switzerland The umbrella organization Childhood Cancer Switzerland also provided SPOG with financial support in 08, largely by arranging very welcome contributions from grant-making foundations. Other grant-making foundations In 08 SPOG received generous support from a total of ten other foundations. It currently has agreements for a period of several years with two foundations. One of them is the Gebauer Foundation, which provides SPOG with a high level of funding. Private donations Every year SPOG receives support from some very loyal and some new private donors. Every donation makes a difference and helps to improve the therapy options and quality of life of affected children and adolescents. This includes the fantastic support given by the students at the International School Basel. Pharmaceutical companies The Scientific Meeting in Lugano was supported by the following pharmaceutical companies in 08: Gilead, Jazz Pharmaceuticals, Lipomed, Novartis, Novo Nordisk, Pfizer and Takeda.

29 CHF '.00 CHF '80'00.0 Sources of funding in 08 SERI performance agreement Various SBFIFoundaQons Leistungsvereinbarung FördersRSungen diverse Swiss Cancer Research Krebsforschung Schweiz Kinderkrebshilfe KinderkrebshilfeSchweiz Schweiz Zoé4life Zoé4life SBFI Horizon 00 SERI Horizon 00 Kinderkrebs Schweiz, SRSungsbeiträge AndereCancer Switzerland, foundaqon contribuqon Childhood Schweizerische SRSung für Klinische Krebsforschung Other Private Spenden Schweizerische SQRung für Klinische Krebsforschung Pharmaunternehmen Kinderkrebs Schweiz Private donaqons PharmaceuQcal companies Childhood Cancer Switzerland Special thanks We would like to thank all the institutions and individuals who support our work for the generous funds they provide. It is only with their tremendous help that SPOG is able to work towards giving children and adolescents with cancer a future.

30 Congratulations and special thanks Congratulations to Prof. Maja Beck Popovic She was elected President of the SIOP Europe Neuroblastoma Group (SIOPEN) and will take up this post in 0. Congratulations to Prof. Michael Grotzer He was appointed Director of the Medical Clinical and Medical Director of the University Children s Hospital Zurich in August 08. Special thanks to Prof. Jean-Marc Joseph He stepped down from his role as paediatric surgery representative in the SPOG network in September 08. Our warmest thanks go to Jean-Marc Joseph for his services. Congratulations to Isabelle Lamontagne-Müller As Managing Director she has been running the SPOG s operative business since September 008, and celebrated ten years in this function in 08. 0

31 Annual accounts Swiss Paediatric Oncology Group SPOG Bern Operating account January to December Operating income Research contributions, federal government Research contributions, third parties Research contributions, KFS Miscellaneous income Total operating income Operating costs Miscellaneous study-related expenses Research contributions, centres Other operating expenses Total operating expenses CHF CHF '04'8.00 '0.00 0' '.0 '7' % 88' ' ' ' '0' % -87'7.7-7'.7-7' '4. -7.% -' '.8-4' '.8-4.8% Interim result '4'.04.8% '7'.8 7.% Coordination expenses Personnel expenses Other coordination expenses Total coordination expenses -'00'. -'.4 -'4' % -'0.0 -'0. -'' % Interim result 4'8.0.8 % -0' % Financial result Financial expenses Total financial result -'8.8 -' % -4'07.0-4' % Interim result 8'.7. % -4' % Extraordinary expenses & out-of-period result Extraordinary expenses Out-of-period income Total extraordinary expenses & out-of-period result -0' '4. -7' % % Annual result '4.8-4' %. %

32 Structure of SPOG Position as of December 08 SPOG MEMBER INSTITUTIONS RESEARCH COUNCIL SWISS CHILDMEDICAL ASSOCIATIONS HOOD CANCER REGISTRY HOSPITAL FACILITIES (SPOG STATIONS) Aarau Basel Bellinzona Bern Geneva Bern Pathology Paediatric Surgery Dr. K. Scheinemann Prof. N. von der Weid Dr. P. Brazzola Prof. J. Rössler Prof. M. Ansari Prof. C. Kuehni M. Trippel Dr. S. Tharakan Lausanne Lucerne St. Gallen Zurich Prof. M. Beck Popovic Dr. F. Schilling Dr. J. Greiner Prof. F. Niggli CLINICAL RESEARCH COORDINATOR Radiooncology One or more Clinical Research Coordinators at each SPOG station Prof. D. Weber Board of Directors Collaborative groups Scientific Advisory Board Prof. Roland Ammann (President) Berlin-Frankfurt-Münster BFM Prof. Michael Grotzer (Vice-President) Gesellschaft für Pädiatrische Onkologie und Hämatologie GPOH Prof. Felix Niggli (Past-President) SPOG COORDINATING CENTER Dr. Jean Michon, France Prof. Maja Beck Popovic (Assessor) Société Internationale d Oncologie Pédiatrique SIOP Prof. Roderick Skinner, United Kingdom Dr. Heinz Hengartner (Assessor) Managing Director Assistant to Managing Director Isabelle Lamontagne-Müller Marlise Rohrer Head Clinical Operations Partner Relations Julia Ruckstuhl Patrizia Specker Clinical Project Management Assistant Quality Management Interim Team Leader: Dr. Michael Zeller Dr. Chun Wai Samantha Chan Dr. Tu-My Diep Lai Lara Fux Derya Keller Dr. Moritz Saxenhofer Dr. Silvia Wirth Administration: Eliane Briggen Prof. Günter Henze, Germany

33 People President Prof. Roland A. Ammann, Bern Vice-President Prof. Michael Grotzer, Zurich Past-President Prof. Felix Niggli, Zurich Assessor Prof. Maja Beck Popovic, Lausanne Assessor Dr. Heinz Hengartner, St. Gallen Heads of the sites Dr. Katrin Scheinemann, Aarau Prof. Nicolas von der Weid, Basel Dr. Pierluigi Brazzola, Bellinzona Prof. Jochen Rössler, Bern Prof. Marc Ansari, Geneva Prof. Maja Beck Popovic, Lausanne Dr. Freimut H. Schilling, Lucerne Dr. Jeanette Greiner, St. Gallen Prof. Felix Niggli, Zurich Paediatric Surgery Dr. Sasha Tharakan, Zurich Paediatric Pathology Mafalda Trippel, Bern Radiology Prof. Damien Weber, Paul Scherrer Institute Villigen SPOG Coordinating Center Isabelle Lamontagne-Müller, Managing Director Julia Ruckstuhl, Head Clinical Operations, Associate Managing Director Dr. Michael Zeller, Team Leader Clinical Project Management ad interim Marlise Rohrer, Assistant to Managing Director Patrizia Specker, Partner Relations Dr. Samantha Chan, Assistant Quality Management Dr. Tu-My Diep Lai, Clinical Project Management Lara Fux, Clinical Project Management Derya Keller, Clinical Project Management Dr. Moritz Saxenhofer, Clinical Project Management Dr. Silvia Wirth, Clinical Project Management Eliane Briggen, Administration Clinical Project Management Study coordination Susann Drerup, Aarau Monika Imbach and Verena Stahel, Basel Pamela Balestra and Dr. Pierluigi Brazzola, Bellinzona Nadine Assbichler, Nadine Beusch and Friedgard Julmy, Bern Rodolfo Lo Piccolo and Dr. Veneranda Mattiello, Geneva Sarah Blanc, Dr. Marjorie Flahaut and Eléna Lemmel, Lausanne Janine Garibay, Lucerne Franziska Hochreutener and Astrid Schiltknecht, St. Gallen Dr. Claudia Althaus, Sabine Holzapfel, Heike Markiewicz, Annette Reinberg, Beate Schwenke and Renate Siegenthaler, Zurich Swiss Childhood Cancer Registry SCCR Prof. Claudia Kuehni, st Co-Head Dr. Verena Pfeiffer, nd Co-Head Position as at: December 08

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36 Schweizerische Pädiatrische Onkologie Gruppe Groupe d Oncologie Pédiatrique Suisse Gruppo d Oncologia Pediatrica Svizzera Swiss Paediatric Oncology Group Effingerstrasse CH-008 Bern Phone