Programme 07/08/2013 World Cord Blood Congress IV and Innovative Therapies for Sickle Cell Disease. Chairs: E. Gluckman (Paris), M.

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1 World Cord Blood Congress IV and Innovative Therapies for Sickle Cell Disease Chairs: E. Gluckman (Paris), M. Cavazzana (Paris) Monaco October 2013, Under the High Patronage of his Serene Highness Prince Albert II de Monaco Thursday 24 th October, 2013 Opening SESSION I: CORD BLOOD STEM CELL Chair: H.E. Broxmeyer (Indianapolis) Preliminary Programme State of the art including methods for graft facilitation H.E. Broxmeyer (Indianapolis) Cord blood subpopulations and reprogrammed cells G. Koegler (Düsseldorf) Niche regulation of T cell generation D. Scadden (Boston) Discussion Coffee break SESSION II: METHODS OF GRAFT FACILITATION Chair: E.J. Shpall (Houston) State of the art E.J. Shpall (Houston) Generation and expansion of human T-lymphoid progenitors from I. André-Schmutz (Paris) cord blood CD34+ cells in a feeder-cell-free DL-4 culture system Building myeloid bridges to enhance engraftment in cord blood C. Delaney (Seattle) transplant and beyond Nicord expanded hematopoietic progenitor cells can outcompete J. Kurtzberg (Durham) the unmanipulated cord blood unit following myeloablative dual umbilical cord blood transplantation in adults with hematological malignancies Hematopoietic stem cell expansion to overcome non-engraftment J. Wagner (Minneapolis) Opening ceremony Reception: Congress Center Friday 25 th October, 2013 SESSION III: NEW INNOVATIVE THERAPIES Chair: M. Cavazzana (Paris) Gene therapy for hemoglobinopathies: obstacles, expectancy, M. Cavazzana (Paris) availability

2 The present and future challenges of gene therapy for sickle cell F. Mavilio (Evry) disease Biomarker - based personalized medicine S. Paczesny (Indianapolis) Engineering cord blood derived T cells to prevent infection C. Bollard (Houston) after cord blood transplantation Cytokine environment analysis can modify the outcome F. Touzot (Paris) of hematopoietic stem cell transplantation Tregs for immune modulation J. Wagner (Minneapolis) Coffee break Discussion and selected abstract presentations: Gene expression of hematopoietic stem cells (CD34+) in post-transplant hematopoiesis Assesment of HLA diversity in Marseille cord blood bank F. Frassoni (Genoa) C. Chabannon (Marseille) Lunch and poster viewing SATELLITE WORKSHOP ROOM C SESSION IV: ROLE OF HLA AND OTHER GENETIC MARKERS Chair: E. Petersdorf (Seattle) Overview: State of the art E. Petersdorf (Seattle) Role of high resolution HLA typing for cord blood transplantation M. Eapen (Milwaukee) Increasing use of hematopoietic stem cell transplantation for M. Oudshoorn (Leiden) ethnic minorities Role of non HLA markers for donor selection R. Tamouza (Paris) Criteria of donor choice for cord blood transplantation A. Ruggeri (Paris) Discussion Coffee break SESSION V: CORD BLOOD BANKING Chair: E. Baudoux (Liège) State of the art E. Baudoux (Liège) Assessment of parameters affecting the quality of cord blood units G. Koegler (Düsseldorf) Updating the cord blood unit inventory to meet current needs S. Querol (Barcelona) Improving outcomes of cord blood transplantation by screening J Kurtzberg (Durham) potency of cord blood units before release from the cord blood bank Role of registries to analyze outcomes data - Eurocord - Netcord V. Rocha (Oxford)

3 Saturday 26 th October, 2013 SESSION VI: INTERNATIONAL OBSERVATORY ON SICKLE CELL DISEASE Chair: E. Gluckman (Centre Scientifique de Monaco CSM and Paris) Sickle cell disease global perspective E. Gluckman (Paris) Hydroxycarbamide in the treatment of sickle cell disease M. de Montalembert (Paris) Is sickle cell disease an autonomic nervous system disorder: T. Coates (Los Angeles) what triggers the transition from steady state to crisis? Sickle cell disease: genetic etiologies for phenotypic diversity J. Elion (Paris) Registry of pregnant women with sickle cell disease M. Cavazzana (Paris) Coffee break Hematopoietic stem cell transplant for sickle cell disease F. Bernaudin (Paris) Haplo-identical related hematopoietic stem cell transplant R. Jones (Baltimore) for sickle cell disease New directions in allogeneic stem and cellular therapy in patients M. Cairo (New York) with sickle cell disease Nicord expanded hematopoietic progenitor cells bridge J. Kurtzberg (Durham) hematopoiesis and facilitate engraftment after myeloablative chemotherapy for patients sickle cell disease Hematopoietic stem cell transplant for sickle cell disease in Brazil B. Simoes (Ribeirao Preto) Haemoglobinopathies and malaria in Africa D. Diallo (Bamako) Epidemiology and treatment of sickle cell in Senegal including hydroxycarbamide, transfusions and chelation in children I. Diagne (Saint Louis - Senegal) in adults S. Diop (Dakar) Long term outcome of paediatric patients undergoing haploidentical M. Dallas (Memphis) and matched related haematopoietic cell transplantation for sickle cell disease Discussion Lunch and poster viewing SESSION VII: HOW TO IMPROVE OUTCOME OF UNRELATED CORD BLOOD TRANSPLANT Chair: J. Wagner State of the art J. Wagner (Minneapolis) Optimization of cord blood transplant for hematologic malignancies: J. Barker (New York) graft, conditioning, graft versus host disease Results of single and double cord blood in adult malignancies: A. Ruggeri (Paris) cost efficiency study Coffee break Conditioning for cord blood transplant G. Sanz (Valencia) Cord blood transplant in children with non malignant disease E. Gluckman (Paris)

4 Cord blood transplantation for adults: what have we learned D.Weisdorf (Minneapolis) Selected abstract presentations Sunday 27 th October, 2013 SESSION VIII: COMPARISON OF CORD BLOOD TRANSPLANTS WITH OTHER SOURCES OF STEM CELLS Chair: V. Rocha State of the art V. Rocha (Oxford) The RTN Policy: a comprehensive algorythm of the allogeneic W. Arcese (Rome) hematopoietic stem cell transplant for adults with hematological malignancies Comparison between related haploidentical transplants in children F. Locatelli (Rome) Coffee break Haplo versus cord blood: pros and cons R. Jones (Baltimore) & J. Wagner (Minneapolis) Algorythm of donor choice K. Ballen (Boston) Round table: summary and future directions Chairs : H.E. Broxmeyer, E.J. Shpall, E. Petersdorf, E. Baudoux, E. Gluckman, V. Rocha, J. Wagner End of meeting SATELLITE WORKSHOP ON SICKLE CELL DISEASE IN EMERGING COUNTRIES : 14:00-17:30 Sessions will be in French and English with simultaneous translation SESSION I: SICKLE CELL DISEASE IN EMERGING COUNTRIES LA DREPANOCYTOSE DANS LES PAYS EMERGENTS Chairs: Belinda Simoes (Ribeirao Preto), Jacques Elion (Paris Guadeloupe) Sickle cell disease in India La drépanocytose en Inde D. Jain (Nagpur) Networks, research tools and Les réseaux, outils de cooperation: the example of recherche et de coopération : L. Tshilolo (Kinshasa) network research on sickle cell disease in Central Africa exemple du réseau de recherche sur la drépanocytose Treatment with hydroxycarbamide, transfusion and iron chelation: issues specific to Africa Problems related to the availability of opioids in Africa en Afrique centrale Le traitement par hydroxycarbamide, la transfusion et la chélation du fer: des problèmes spécifiques à l'afrique Problèmes liés à la disponibilité des morphiniques en Afrique J. Makani (Dar-es-Salaam) E. Kafando (Ouagadougou) Coffee Break

5 SESSION II: PAROLE AUX ASSOCIATIONS / WORDS FROM THE FAMILY SUPPORT GROUPS Chairs: Dipty Jain (Nagpur), Danielle Léna (Marseille) : : : Introduction : Santé Sud Introduction : Santé Sud N. Hanssen (Marseille) Association for the struggle against sickle cell disease in Association de Lutte contre la drépanocytose du Niger F. Hima (Niamey) Niger Struggle against sickle cell Lutte contre la Drépanocytose P. Jeannot (Chatillon) disease Madagascar-France Madagascar-France Sickle Cell Foundation of Ghana La fondation de la Drépanocytose du Ghana P. Mensah (Kumasi) Support association for SCD in Association de Soutien aux M. Wane (Nouakchott) Mauritania Senegalese Association for the struggle against Sickle Cell Disease Sickle Cell Society (Royaume Uni) Drépanocytaires en Mauritanie Association Sénégalaise de Lutte contre la Drépanocytose Sickle Cell Society (United Kingdom) M. Ndiaye (Dakar) J. James (London) GENERAL DISCUSSION Chairs: Julie Makani (Dar-es-Salaam), Léon Tshilolo (Kinshasa)

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