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1 Not for publication or presentation A G E N D A CIBMTR WORKING COMMITTEE FOR GRAFT SOURCES & MANIPULATION Grapevine, TX Thursday, February 27, 2014, 2:45 4:45 pm Co Chair: Co Chair: Co Chair: Statisticians: Scientific Director: Daniel H. Fowler, MD, National Cancer Institute, Bethesda, MD Telephone: ; Fax: ; E mail: Miguel Angel Perales, MD, Memorial Sloan Kettering Cancer Center, New York, NY Telephone: ; Fax: ; E mail: Vanderson Rocha, MD, PhD; Churchill Hospital, Oxford, UK Telephone: ; Fax: ; E mail: Mei Jie Zhang, PhD, CIBMTR Statistical Center, Milwaukee, WI Telephone: ; Fax: ; E mail: Junfang Chen, MS, CIBMTR Statistical Center, Milwaukee, WI Telephone: ; Fax: ; E mail: Mary Eapen, MD, MS, Medical College of Wisconsin, Milwaukee, WI Telephone: ; Fax: ; E mail: 1. Introduction Minutes and Overview Plan from February 2013 meeting (Attachment 1) 2. Accrual summary (Attachment 2) 3. Published or submitted papers a. GS08 02 Alousi AM, Le Rademacher J, Saliba RM, Appelbaum FR, Artz A, Benjamin J, Devine SM, Kan F, Laughlin MJ, Lazarus HM, Liesveld J, Perales MA, Maziarz RT, Sabloff M, Waller EK, Eapen M, Champlin RE. Who is the better donor for older hematopoietic transplant recipients: an older aged sibling or a young, matched unrelated volunteer? Blood 121(13): , b. GS09 02 Eapen M, Klein JP, Ruggeri A, Spellman S, Lee SJ, Anasetti C, Arcese W, Barker JN, Baxter Lowe LA, Brown M, Fernandez Vina MA, Freeman J, He W, Iori AP, Horowitz MM, Locatelli F, Marino S, Maiers M, Michel G, Sanz GF, Gluckman E, Rocha V. Impact of allele level HLA matching on outcomes after myeloablative single unit umbilical cord blood transplantation for hematologic malignancy. Blood 123(1):133 40, c. GS11 01 Marks DI, Ahn KW, Xiaobo Z, Appelbaum, Bachanova V, Barker JN, Brunstein CG, Gibson J, Kebriaei P, Lazarus HM, Olsson R, Perales, Pidala J, Savani B, Rocha V, Eapen M. Unrelated umbilical cord blood transplant for adult acute lymphoblastic leukemia in first and second complete remission: a comparison with allografts from adult unrelated donors. Haematologica 99(2): ,

2 Not for publication or presentation d. GS12 03 Weisdorf D, Eapen M, Ruggeri A, Zhang M, Zhong X, Brunstein C, Ustun C, Rocha V, Gluckman E. Alternative donor transplantation for order patients with Acute Myeloid Leukemia in first complete remission: a CIBMTR Eurocord analysis. Submitted. 4. Studies in progress (Attachment 3) a. GS05 01 CB processing and thawing techniques on MA CB HCT Manuscript Preparation outcomes (K Ballen) b. GS08 01 Reassessment of impact donor age on outcome after URD Manuscript Preparation HCT (C Kollman) c. GS12 01 Stem cell dose in RIC HCT for AML and MDS (J Törlén/T Manuscript Preparation Erkers/O Ringdén) d. GS13 01 ATG vs non ATG for CB HCT (D Ponce/ M Perales/J Barker) Analysis e. GS13 02 Matching between UCB units in double UCB Protocol Development transplantation (C Brunstein) 5. New study proposals a. PROP Comparison of clinical outcomes between T cell replete haploidentical and matched related and unrelated donor hematopoietic stem cell transplantation (S Pingali/ E Fuchs/ S Ciurea) (Attachment 4) b. PROP Haplo identical transplants as an alternative to matched unrelated donor transplants (O Ringden) (Attachment 5) c. PROP Evaluation of three alternative donor strategies for patients with hematologic malignancies: Comparison between unrelated double umbilical cord blood (ducb) and HLA haploidentical related donor using either post transplant cyclophosphamide or ex vivo T cell depletion (A Mussetti/ M Perales) (Attachment 6) d. PROP Trends in HLA haploidentical allogeneic hematopoietic stem cell transplantation for hematologic malignancies: a CIBMTR analysis (G Akpek) (Attachment 7) e. PROP Association between recipient and donor sex and clinical outcome after allogeneic hematopoietic stem cell transplantation (P Armand/ H Kim) (Attachment 8) f. PROP Peripheral stem cell graft composition of female donors and transplant outcome (J Schetelig/ A Nagler/ A Shimoni/ G Ehninger) (Attachment 9) g. PROP The impact of graft T cell subset doses on the outcomes of allogeneic peripheral blood stem cell transplants after reduced intensity conditioning in patients with hematologic malignancies (R Reshef/ D Porter) (Attachment 10) h. PROP Using landmark analysis to provide updated relapse and leukemia free survival estimates to patients after umbilical cord blood transplantation (C Brunstein/ S Lee) (Attachment 11) 2

3 Not for publication or presentation Attachment 1 MINUTES AND OVERVIEW PLAN CIBMTR WORKING COMMITTEE FOR GRAFT SOURCES AND MANIPULATION Salt Lake City, Utah Friday, February 15, 2013, 12:15 pm 2:15 pm Co Chair: Co Chair: Co Chair: Statisticians: Scientific Director: Mary Laughlin, MD, University of Virginia, Charlottesville, VA Telephone: ; Fax: ; E mail: Daniel H. Fowler, MD, National Cancer Institute, Bethesda, MD Telephone: ; Fax: ; E mail: Miguel Angel Perales, MD, Memorial Sloan Kettering Cancer Center, New York, NY Telephone: ; Fax: ; E mail: Mei Jie Zhang, PhD, CIBMTR Statistical Center, Milwaukee, WI Telephone: ; Fax: ; E mail: Junfang Chen, MS, CIBMTR Statistical Center, Milwaukee, WI Telephone: ; Fax: ; E mail: Mary Eapen, MD, MS, Medical College of Wisconsin, Milwaukee, WI Telephone: ; Fax: ; E mail: 1. Introduction Dr. Perales called the meeting to order at 12:20pm, Drs. Laughlin, Fowler, Perales and Eapen chaired the meeting. Dr. Perales thanked Dr. Mary Laughlin for her strong leadership and guidance as Co Chair of the committee, and welcomed incoming Co Chair Vanderson Rocha, MD, PhD. Dr. Perales also clarified that the voting scale is from 1 to 9, the lowest score has the highest scientific impact. The minutes of the February 2012 meeting were approved without modifications. 2. Accrual summary Due to the full agenda, the accrual summary of registration and research cases between 1989 and 2012 were not presented to the committee but included in the Working Committee documents. 3. Presentations, published or submitted papers Publications were briefly reviewed. a. GS09 03 Brunstein CG, Eapen M, Ahn KW, Appelbaum FR, Ballen KK, Champlin RE, Cutler C, Kan F, Laughlin MJ, Soiffer RJ, Weisdorf DJ, Woolfrey A, Wagner JE. Reduced intensity conditioning transplantation in acute leukemia: the effect of source of unrelated donor stem cells on outcomes. Blood 119(23): ,

4 Not for publication or presentation Attachment 1 b. GS08 05 Scaradavou A, Brunstein C, Eapen M, Le Rademacher J, Barker J, Spellman S, Kan F, Laughlin MJ, Kurtzberg J, Wagner JE, Shpall EJ. Umbilical cord blood transplantation for adults with acute leukemia: impact of single versus double cord blood units on transplantation outcomes. Blood 121(5): , c. GS08 02 Alousi AM, Le Rademacher J, Saliba RM, Appelbaum FR, Artz A, Benjamin J, Divine SM, Kan F, Laughlin MJ, Lazarus HM, Liesveld J, Perales M, Maziarz RT, Sabloff M, Waller EK, Eapen M, Champlin RE. Who is the better donor for older hematopoietic transplant recipients: an older aged sibling or a young, matched unrelated volunteer? Blood. In Press. 4. Studies in progress a. HC03 01 Prevalence of microbially contaminated hematopoietic stem cell products (R Champlin) This study examines the effect (if any) on early survival after transplantation of microbially contaminated hematopoietic stem cell products. The manuscript for this study is being prepared will require some additional analyses prior to final draft. b. GS05 01 Effect of cord blood processing and thawing techniques on transplant outcomes after single myeloablative umbilical cord blood transplantation (K Ballen) The study will study the effect of cord blood banking practice on hematopoietic recovery and early survival after myeloablative single umbilical cord blood transplants. The study population is limited to US cord blood banks and transplants in the U.S. The final analysis has been completed; a copy of the results and its interpretation submitted to the Project Officer for the SCTOD. c. GS07 01 Peripheral blood versus bone marrow for non myeloablative stem cell transplantation (R Champlin) This study is dropped was not discussed at the meeting. A larger more recent cohort includes these patients and the findings of the more recent study (funded study SC11 02). d. GS08 01 Reassessment of impact of donor age on outcome after unrelated donor hematopoietic cell transplantation (HCT) (C Kollman) This study explored the association between donor characteristics and graft versus host disease and survival in over 6000 matched or mismatched donor recipient pairs. The final draft of the manuscript is under review writing committee and will be submitted shortly. e. GS09 02 The effect of better HLA matching on survival after umbilical cord blood transplants (M Eapen /V Rocha /S Spellman) The study examined the relative importance of donor recipient allele level matching at HLA A, B, C and DRB1 on outcomes after umbilical cord blood transplantation for acute leukemia and myelodysplastic syndrome. The results suggest better HLA matching of the cord blood unit to the recipient results in significantly lower rates of non relapse mortality. Further, an isolated mismatch at HLA A, C and DRB1 led to high rates of non relapse mortality. This effect was not seen at the HLA B locus implying a mismatch at HLA B was better tolerated than a mismatch at HLA A, C or DRB1. In addition, the effect of TNC is independent of HLA mismatching; the optimal TNC is > /Kg, increasing TNC to higher doses including in excess of /Kg does not overcome the excess non relapse mortality risks associated with HLA mismatch. The manuscript is being prepared. 4

5 Not for publication or presentation Attachment 1 f. GS10 03 Outcomes using a cryopreserved donor graft in unrelated allogeneic HCT (N Frey /H Lazarus) The primary objective of this study is to compare outcomes after transplantation of cryopreserved grafts (bone marrow or peripheral blood) from adult unrelated donors. There are about such transplants facilitated by the NMDP. Given the numbers of proposals the committee received, the working committee members were asked to assign a priority score. The study received an average priority score therefore, the study was dropped. g. GS11 01 The outcome of myeloablative and reduced intensity unrelated donor cord blood transplants for ALL in CR1 and CR2 in adults. A comparison with unrelated donor marrow and peripheral blood transplantation (D Marks) This study compared outcomes after single or double cord blood transplantation for ALL in first or second complete remission in adults to that after 8/8 or 7/8 HLA matched bone marrow/peripheral blood transplants. Compared to recipients of adult donor transplantation, recipient of cord blood transplants had significantly slower and lower likelihood of neutrophil recovery at day 28 and platelet recovery at day 100. The risk of graft failure was higher after cord blood transplants. Results of multivariate analysis confirmed lower risks for 2 4 acute graft versus host disease (GVHD) but without differences in chronic GVHD, non relapse and overall mortality. However, treatment failure was higher after cord blood transplants failure rates were driven by higher relapse risks, which is contrary to other publications. Review of data at the statistical center revealed inconsistencies in the definition of leukemia relapse. The study file has been reviewed / corrected as needed. The final analysis will be undertaken shortly. The manuscript for this study is being prepared. h. GS12 01 Role of stem cell dose in reduced intensity conditioning transplants for AML/MDS and ALL (J Törlén/T Erkers/O Ringdén) The aim of this study is to evaluate the role of nucleated and CD34 cell dose in the setting of reduced intensity conditioning. The study population will include patients with acute lymphoblastic, acute myeloid leukemia or myelodysplastic syndrome and received allografts (bone marrow or peripheral blood) from HLA matched siblings or matched unrelated donors, between 1998 and Outcomes of interest include: hematopoietic recovery, graft failure, acute and chronic graft versus host disease, non relapse mortality, relapse, treatment failure and overall mortality. The study is important since it is necessary to deliver an optimal cell dose for successful transplantation and whether delivering doses in excess may in fact contribute to higher risks of acute or chronic graft versus host disease. There was extensive discussion of this study; suggestions included looking at the ratio of CD34 to nucleated cells, including pediatric patients, and a sub analysis of conditioning intensity. It was pointed out to Dr. Ringden that in the analysis, the interaction between conditioning regimen and in vivo T cell depletion reported from a paper from this working committee be considered. Another important point is the predominance of the graft, peripheral blood progenitor cells in the setting of reduced intensity conditioning. The number of bone marrow transplants available may prohibit the study of this graft. Further, Dr. Ringden made no efforts to limit the study population to current practice as it pertains to selection of adult unrelated donors. The statistical center recommends that only 8/8 and 7/8 HLA matched unrelated donor transplants be included. The statistical center will also review the number of eligible bone marrow recipient to determine whether these cases should 5

6 Not for publication or presentation Attachment 1 be included. The statistical center will review the conditioning regimens used and will likely limit the study population to transplants with alkylating agents / fludarabine with or without in vivo T cell depletion, low dose TBI (200 cgy) and commonly used graft versus host disease prophylaxis package regimens. Other suggestions included separate analyses of donor types as well as graft types (if feasible) and a careful consideration be given to patient ages included in the dataset. For example, reduced intensity conditioning is infrequent in younger patients and perhaps excluded to generate a homogeneous population. Also review carefully the diseases included particularly the number of patients with acute lymphoblastic leukemia. Given the numbers of proposals the committee received, the working committee members were asked to assign a priority score. The study received a high priority score proceed to protocol development. 5. Future/ Proposed studies a. PROP Who is the better donor for older adult hematopoietic transplant recipients: an older aged sibling or unrelated double UCB? (M Laughlin/C Brunstein/J Barker) This study will compare rates of graft versus host disease, overall and non relapse mortality in older patients who received mismatched cord blood transplants to those who received grafts from their sibling. This proposal was developed in light of the findings of GS08 02, which showed lower rates of graft versus host disease after older matched sibling donor transplants compared to HLA matched transplants from younger donors and better survival in good risk patients after HLA matched sibling transplants. As with GS08 02, the study population will include patients 50 years or older who received grafts from their HLA matched sibling or mismatched umbilical cord blood (one or two units). Approximately 1200 HLA matched sibling transplants and 400 double cord blood transplants are eligible. Discussions included the relevance of such a study often when a matched sibling is available, that donor is preferred. However graft versus host disease is lower after cord blood transplants compared to unrelated donor transplants BUT unlikely to be lower than matched sibling transplants. There was discussion about co morbidities in older sibling donors rendering them ineligible to donate. There was also some discussion as to what would determine the optimal unit, i.e., differing selection criteria between transplant centers and how would one adjust for practice variation. While there were no major concerns about feasibility or scientific merit, when ranked against the approved studies and other proposals this was assigned a lower priority score. Therefore this proposal was dropped. b. PROP Comparison of cord blood transplantation outcomes according to the inclusion of anti thymocyte globulin (ATG) in pre transplant conditioning (DM Ponce/JN Barker/ MA Perales) Dr. Juliet Barker presented the proposal. The primary aim of the study is to compare diseasefree and overall survival after cord blood transplants testing the effect of in vivo T cell depletion in the setting of myeloablative and reduced intensity conditioning. Other outcomes include: hematopoietic recovery including graft failure, acute and chronic graft versus host disease, EBVassociated PTLD, immune reconstitution, recurrent disease and non relapse mortality. The hypothesis being omission of anti thymocyte globulin will result in better disease free survival. Study population will include the following hematologic malignancies (ALL, AML, CLL, MDS, NHL and HL) and transplanted with cord blood grafts between 2007 and Suggestions included: information on ATG dose, type of ATG and if possible timing (this would require supplemental 6

7 Not for publication or presentation Attachment 1 data collection contact centers to establish the timing of administration which is likely to be uniform at centers). Received the highest priority score approved / proceed to protocol development. c. PROP Matching between UCB units in double UCB transplantation (C Brunstein) The purpose of the study is to determine whether matching between two mismatched units affect hematopoietic recovery, acute graft versus host disease and early survival. The selection of units when considering a double unit transplant varies some match the two units to each other to no more than two mismatches and others ignore matching between two units. The study population will include transplants for acute lymphoblastic and myeloid leukemia from The outcomes of interest include hematopoietic recovery, GVHD and overall survival at day 100 and 1 year. Dr. Laughlin suggested looking at whether the mismatch was at class I or class II or both, if feasible. HLA match will consider HLA match at A and B at intermediate resolution and allele level at DRB1. There were also suggestions to extend the transplant period to include 2012 transplants as outcomes are short term: day 28 neutrophil recovery, day 100 / 1 year survival and grade 2 4 acute GVHD. This proposal received a high priority score approved / proceed to protocol development. d. PROP Cord blood unit release testing criteria and the impact on transplantation outcome (D Regan) The primary aim of the study is to determine the impact of umbilical cord blood colony forming unit (CFU) testing at the time of release on transplantation outcome. The secondary aim is to determine potential CFU growth thresholds for release of cord blood units for transplantation. Study population includes recipients with first allogeneic, myeloablative single unit cord blood transplants for AML, ALL or MDS facilitated by the NMDP. The interested outcomes are: hematopoietic recovery and day 100 overall survival. In a survey undertaken by the NMDP, 19 cord blood banks in the US are eligible. A survey was undertaken to determine which of these banks have established criteria and/or criteria used to determine which of the units are released to transplant centers. Of the 19 eligible banks, 14 responded to the survey. Twelve of 14 have established criteria for release but there is no consensus as to what these criteria should be. Of interest for this proposal is CFU testing the survey suggest only 6 of the 14 banks perform CFU testing prior to release and the results reported as any growth versus no growth. The other 8 banks test either total nucleated cell dose and/or CD34 dose. Twelve of the 14 banks perform viability but the method of testing is variable between banks and the acceptable viability range also varied between banks. This proposal indicated the primary question would be: was any CFU growth present vs. none. In the ensuing discussion there were concerns about the lack of a quantitative number for CFUs, the heterogeneous approach to testing adopted by the banks, heterogeneous handling / processing methods at transplant centers and the cumulative effect of these variations on transplant outcome. Additionally, this proposal will require collection of supplemental data from cord blood banks per unit basis (to link release criteria as established by each bank to the unit used for transplantation). In addition to feasibility, there were concerns about the scientific merit / validity of any results generated from the planned analyses in particular the lack of quantification of CFU growth at the banks that routinely perform this testing and its comparison with the other banks that do 7

8 Not for publication or presentation Attachment 1 not use this method for release. The committee assigned a low priority score the proposal was dropped. 6. Other business None. Proposals and were approved. Meeting adjourned at 2:06 PM. Working Committee Overview Plan for a. HC03 01 Prevalence of microbially contaminated hematopoietic stem cell products we will update survival, re run logistic regression model for early survival (day 100) the primary endpoint. We anticipate submitting the manuscript June 2013 Dr. Laughlin will have primary responsibility for drafting the manuscript and therefore, first author. b. GS05 01 Effect of cord blood processing and thawing techniques on transplant outcomes after single myeloablative umbilical cord blood transplantation. Completed final analyses and report submitted to the Project Officer for the SCTOD. A copy of that report will be send to Dr. Ballen as soon we have approval from HRSA / Advisory Committee to the Stem Cell Therapeutics Outcomes Database. We anticipate submitting the manuscript by June c. GS08 01 Reassessment of impact of donor age on outcome after unrelated donor hematopoietic cell transplantation. The statistical center has a copy of the manuscript from the principal investigator this is the final copy pending any revisions undertaken at the statistical center. We anticipate submitting this manuscript for peer review March d. GS09 02 The effect of better HLA matching on survival after umbilical cord blood transplants. The manuscript for this study is being written, anticipated submission by April e. GS11 01 The outcome of myeloablative and reduced intensity unrelated donor cord blood transplants for ALL in CR1 and CR2 in adults. The manuscript for this study is being written, anticipated submission by April f. GS12 01 Role of stem cell dose in reduced intensity conditioning transplants for acute myeloid leukemia, myelodyspalstic syndrome and acute lymphoblastic leukemia. We anticipate beginning to work on this project March 2013, complete analysis by June 2013 and submit an abstract to the 2013 American Society of Hematology meeting. We anticipate submitting the manuscript for peer review October/November g. GS13 01 (PROP ) Comparison of cord blood transplantation outcomes according to the inclusion of anti thymocyte globulin in pre transplant conditioning. We anticipate developing the study protocol May/June 2013; prepare study file and final analyses by December We anticipate submitting the manuscript for peer review by March/April

9 Not for publication or presentation Attachment 1 h. GS13 02 (PROP ) Matching between cord blood units in double cord blood unit transplantation is there an effect on hematopoietic recovery, acute graft versus host disease and overall survival? We anticipate developing the study protocol May/June 2013; prepare study file and final analyses by December We anticipate submitting the manuscript for peer review by March/April Work Assignments for Working Committee Leadership (February 2013) Daniel Fowler Mary Eapen HC03 01: Prevalence of microbially contaminated hematopoietic stem cell products GS05 01: Effect of cord blood processing and thawing techniques on transplant outcomes after single myeloablative umbilical cord blood transplantation GS11 01: The outcome of myeloablative and reduced intensity unrelated donor cord blood transplants for ALL in CR1 and CR2 in adults GS13 02: (PROP ) Matching between cord blood units in double cord blood unit transplantation is there an effect on hematopoietic recovery, acute graftversus host disease and overall survival? Vanderson Rocha GS09 02: The effect of better HLA matching on survival after umbilical cord blood transplants GS12 01: Role of stem cell dose in reduced intensity conditioning transplants for acute myeloid leukemia, myelodyspalstic syndrome and acute lymphoblastic leukemia Miguel Perales GS13 01: (PROP ) Comparison of cord blood transplantation outcomes according to the inclusion of anti thymocyte globulin in pre transplant conditioning 9

10 Not for publication or presentation Attachment 2 Accrual Summary for Graft Sources and Manipulation Working Committee Characteristics of patients reported to the CIBMTR between 1989 and 2013 Characteristics TED N (%) Research N (%) Number of cases Donor type HLA identical sibling donor HCT Bone marrow (53) (61) Peripheral blood (46) (37) Peripheral blood + bone marrow 634 (<1) 202 (<1) Umbilical cord blood 549 ( <1) 234 (<1) Identical twin donor HCT Bone marrow 579 (41) 310 (45) Peripheral blood 834 (59) 369 (54) Peripheral blood + bone marrow 8 (<1) 2 (<1) Umbilical cord blood 4 (<1) 3 (<1) Other related donor HCT Bone marrow 8791 (60) 5709 (78) Peripheral blood 5457 (37) 1449 (20) Peripheral blood + bone marrow 228 ( 2) 66 (<1) Umbilical cord blood 113 (<1) 49 (<1) Unrelated donor HCT Bone marrow (43) (51) Peripheral blood (41) (31) Peripheral blood + bone marrow 147 (<1) 124 (<1) Umbilical cord blood (15) 6209(17) 10

11 Not for publication or presentation Attachment 3 TO: FROM: The Members, Graft Sources and Manipulation Working Committee Mary Eapen, MD, MS RE: Summary of the Committee s Activities for 02/ /2014 Completed Studies: 02/ /2014 GS05 01: Effect of cord blood processing and thawing techniques on transplant outcomes after single myeloablative umbilical cord blood transplantation (K Ballen): A variety of processing and freezing practices at the cord blood banks (CBB) and thawing techniques at transplantation centers are employed, but optimal practices have not been determined. Processing and banking practices at 16 public CBB in the United States were collected and their effect on transplant outcomes assessed. The study population included 530 single umbilical cord blood (UCB) myeloablative transplantations for hematologic malignancies. UCB banking practices were separated into four mutually exclusive groups based on whether processing was automated or manual; units were plasma / red blood cell reduced or buffy coat method or plasma reduced; and hespan or dimethyl sulfoxide (DMSO) added to the bag. Neutrophil recovery at day 28 was significantly lower after transplantation of units that were manually processed and plasma reduced with DMSO added to the bag. Day 100 survival did not differ by CBB of origin. However, day 100 survival was marginally better with units thawed with the dextranalbumin wash method at transplant centers as opposed to the no wash or a dilution only technique. These data suggest an effect of banking practices on neutrophil recovery and an early survival advantage with thawing practices at the transplant center. GS08 01: The effect of donor characteristics on survival after unrelated donor transplantation for hematologic malignancy (C Kollman): Over 6000 unrelated donor recipient transplantations between 1988 and 2006 were examined to identify donor characteristics associated with survival. All patients had a hematologic malignancy and donor recipient pairs were HLA typed at the allele level. In multivariate analysis, three donor characteristics were identified: survival was higher with donors aged 32 years or younger, HLA matched at the allele level at HLA A, B, C and DRB1 and blood group ABO matched to the recipient. Each of these donor characteristic had an independent prognostic value. Donor recipient gender match was not associated with survival. However, chronic GVHD rates were lower with transplantation of grafts from male or nulliparous female donors. Donor CMV serostatus was not associated with survival. In conclusion, donor age, donor recipient HLA match and ABO match are equally important when selecting adult unrelated donors. GS12 01: Low CD34 cell dose is associated with higher non relapse and overall mortality after reduced intensity conditioning hematopoietic cell transplantation for acute myeloid leukemia and myelodysplastic syndrome (J Thörlén/O Ringden): The effect of CD34 dose on 1057 patients aged years with AML or MDS who received RIC 11

12 Not for publication or presentation Attachment 3 regimen HCT between 2000 and 2011 were studied. Patients received grafts from HLA matched siblings (n = 370) or from volunteer adult unrelated donors matched at the allele level at HLA A, B, C and DRB1 (8/8; n = 522) or mismatched at 1 HLA locus (7/8; n = 178). All patients received peripheral blood progenitor cells (PBPC), low dose TBI regimens (200 cgy) or alkylating agent plus fludarabine containing regimens. Separate analyses were conducted for HLA matched and unrelated donor transplants. For HLA matched sibling transplants, exploratory analysis identified differences in survival for CD34 dose less than 4 x 10 6 /kg. Multivariate modeling, adjusting for performance score, disease status, cytogenetic risk and transplant period, showed transplantation of PBPC with CD34 dose < 4 x 10 6 /kg was associated with higher risks of overall mortality and non relapse mortality. The effect of CD34 dose was independent of the other factors associated with mortality. In contrast, after unrelated donor transplantation, the effect of CD34 cell dose on mortality was marginal. Exploratory analysis identified differences in survival for CD34 dose less than 6 x 10 6 /kg. After adjusting for age, performance score, disease status, interval from diagnosis to HCT, and HLA match, transplantation of PBPC with CD34 dose < 6 x 10 6 /kg was associated with marginally higher risks for overall mortality and non relapse mortality. For both donor sources, CD34 dose was not associated with acute or chronic GVHD or disease recurrence. Further, we did not identify a CD34 dose for either donor source above which acute or chronic GVHD risks were higher. In the setting of RIC transplants for AML and MDS, the data support optimizing PBPC collections such that the CD34 dose delivered is in excess of 4 x 10 6 /kg for HLA matched sibling and CD34 dose in excess of 6 x 10 6 /kg for unrelated donor transplants. GS12 03: Alternative Donor Transplantation for Older Patients with Acute Myeloid Leukemia in First Complete Remission: a CIBMTR Eurocord Analysis (D Wiesdorf): Transplant outcomes for AML in patients over age 50 years in CR1 were compared after adult unrelated donor (URD; n = 441, 8/8 and n = 94 7/8 HLA matched) and umbilical cord blood (UCB; n = 205) transplantations to determine the relative efficacy of one graft source over the other. UCB recipients less often achieved CR1 within 8 weeks, more often received reduced intensity conditioning, and cyclosporin based GVHD prophylaxis. Three year transplant related mortality was higher and leukemiafree survival lower with UCB compared to 8/8 URD transplantation. Compared to 8/8 URD transplants, transplant related mortality was higher after 7/8 URD, but leukemia free survival similar. Chronic GVHD was lowest after in UCB transplantation. Three year survival was highest after 8/8 URD 43%. Survival was lower after UCB 30% and 7/8 URD transplants. Although a fully matched URD is preferred when available, transplantation with UCB or mismatched URD extends survival for over a third of older patients. Less frequent chronic GVHD with UCB transplantation may be of particular value for older patients. Studies: Active GS13 01: Comparison of cord blood transplantation outcomes according to the inclusion of antithymocyte globulin in pre transplant conditioning (D Ponce/ M Perales/J Barker): This study compares outcomes after cord blood transplantation testing the effect of in vivo T cell depletion. Given differences in patient characteristics, in particular age, by transplant conditioning intensity, two separate analyses will be conducted. The first will focus on myeloablative transplant conditioning regimens in a pediatric population and second, on adults who received reduced intensity conditioning. GS13 02: Matching between UCB units in double UCB transplantation (C Brunstein): This study will explore whether better matching between two mismatched units in the setting of double umbilical cord blood transplantation affects early transplant outcomes. 12

13 Not for publication or presentation Attachment 4 Study Proposal Study Title: Comparison of Clinical Outcomes between T cell Replete Haploidentical and Matched Related and Unrelated Donor Hematopoietic Stem Cell Transplantation Sai R. Pingali, MD 1, Ephraim Fuchs 2, Stefan O. Ciurea, MD 1 1 The University of Texas, M. D. Anderson Cancer Center, TX 2 Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins, MD Hypothesis: Outcomes of haploidentical stem cell transplantation performed with post transplantation cyclophosphamide, tacrolimus and mycophenolate for GVHD prevention are similar to matched transplantation. Specific Aims: To compare outcomes of patients with hematologic malignancies (myeloid and lymphoid) treated with haploidentical stem cell transplantation ( 2 antigen mismatch) with matched (related and unrelated) donor transplants for same disease indications. Outcomes to be evaluated include: engraftment, nonrelapse mortality (NRM), relapse rate (RR), overall survival (OS) and disease free survival (DFS). Background: Allogeneic Hematopoietic stem cell transplantation is an effective treatment for advanced hematologic myeloid and lymphoid malignancies 1. An HLA identical donor transplant is preferred; however, a matched sibling is available for less than 25% of the recipients. Using international registries, an unrelated donor is identified in the US for 50% 60% of the Caucasian population, while the chance to find an unrelated donor for non Caucasian population is less than that (approximately 30% for Hispanics, 10% for African Americans and Asians). In addition, it takes an average of 3 4 months to identify and procure the stem cells from an unrelated donor 2. These numbers are expected to be even smaller with increase in the minority population and interethnic/interracial marriages 3, 4. Mismatched relatives represent a potential donor source of stem cells for such patients, >90% will have an available haploidentical donor for transplantation 5,6. Historically, haploidentical stem cell transplantation (HaploSCT) was associated with high rates of graft rejection and acute graft versus host disease (agvhd) 7. T cell depletion decreased the rate of GVHD at the expense of a higher risk of rejection and severe infections 8. T cell replete (TCR) HaploSCT with the use of post transplant high dose cyclophosphamide(ptcy), tacrolimus and mycophenolate mofetil (MMF) for GVHD prophylaxis pioneered by the Hopkins group, was shown to have similar rates of agvhd with matched transplantation and low non relapse mortality without significantly compromising engraftment In addition, the risk of fatal infections was also lower with this strategy because of more effective immune reconstitution presumably related to maintaining the memory T cells in the graft 12. Recent studies comparing TCR HaploSCT with matched transplants, showed similar outcomes 12,13. We reviewed data from our institution for patients treated with a haploidentical, matched sibling and matched unrelated donor transplant in an uniform cohort of patients with AML/MDS in remission who received the same conditioning, melphalan based. There were no significant differences in engraftment, NRM, relapse rate and PFS between recipients of haploidentical and matched related or 13

14 Not for publication or presentation Attachment 4 unrelated donor transplantations, while T cell depleted HaploSCT recipients did significantly worse (ASH abstract #61013) (Figure 1). The objectives of this study are to compare outcomes of patients who underwent TCR HaploSCT (treated at MD Anderson Cancer Center and Johns Hopkins) with a matched cohort of HLA matched donor transplants reported to Center for International Blood and Marrow Transplant registry (CIBMTR) between years 2000 and Other patients who received a TCR HaploSCT with PTCY and reported to the CIBMTR database may be added to the investigational arm, if available. Methodology: Patients who underwent TCR HaploSCT with myeloablative, RIC and non myeloablative conditioning as previously defined 14 who received post transplant cyclophosphamide, tacrolimus and MMF for GVHD prophylaxis at The University of Texas M. D. Anderson Cancer Center (UTMDACC) and Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins (SKCCCJH) from January 2000 to December 2012 will be included in the experimental arm of the study. We estimate the TCR HaploSCT cohort would consist of approximately 400 patients. Patients who had matched donor transplantation during the same time period with similar patient and disease related characteristics from CIBMTR will be included in the control group 15. Control patients will be identified using a matched pair analysis with patient and disease characteristics. Comparison will be performed using Cox proportional hazards model. Overall survival (OS) is defined as time from SCT to last follow up. Similarly, non relapse mortality (NRM) will be computed from time of SCT to last known vital sign. Time to progression is defined time of SCT and date of disease progression. Patients who are alive and did not experience progression of disease at the last follow up date will be censored. The Kaplan Meier method will be used to estimate OS and DFS and the log rank test was used to assess differences between specific groups 16. NRM and time toprogression will be determined by the cumulative incidence function using the competing risks method. The cumulative incidence of grade II IV acute graft vs. host disease (GVHD) and chronic GVHD (cgvhd) (limited and extensive) will also be determined using the competing risks method 17. The competing risks include disease progression and death, while those patients who do not experience GVHD or progression of disease and alive at the last follow up will be censored. All statistical analyses will be performed using SAS 9.3 for Windows. Endpoints: The primary endpoints of these analyses are: Hematopoietic recovery: Time to neutrophil engraftment ANC > 0.5 x 10 9 /L for three consecutive days will be the primary measure for comparisons of hematopoietic recovery. Time to platelet engraftment platelet count 20 x 10 9 /L Time to platelet count 100 x 10 9 /L. Graft Failure: Primary graft failure defined as no evidence of transplanted marrow function after day 28 post transplant Secondary graft failure Development of inadequate marrow function (fall of granulocytes to <0.5/mcl for 3 or more consecutive days) any time after initial engraftment has been achieved. Incidence of acute and chronic GVHD: grade II IV acute GVHD and limited and extensive chronic GVHD. 14

15 Not for publication or presentation Attachment 4 Treatment related mortality: time to death without evidence of disease recurrence. Patients are censored at time of relapse or at last follow up. Disease recurrence: time to onset of disease relapse. Patients will be censored at death in continuous CR or, for patients surviving in continuous complete remission, at the last contact. Disease free survival: time to treatment failure (death or relapse). Patients are censored at time of last follow up. Overall survival: time to death. Patients are censored at time of last follow up. Variables to be analyzed: A Continuous variables B Categorical variables Patient related: Age at transplant (A) Gender (female vs male) (B) Karnofsky performance score at transplant (<90% vs 90%), when available (B) Disease related (at initial diagnosis): Cytogenetics (AML, MDS) (good/intermediate vs. poor prognosis) (B) AML: Prior hematological disorder (MDS or secondary/therapy related) (yes/no) (B) Extramedullary disease (yes/no) (B) CNS disease? (yes/no) (B) Disease related (at the time of transplant): Disease status transplant (B) Time from diagnosis to transplant (A) Ever achieved a first remission? (yes/no) (B) Time to achieve complete remission (A) Duration of complete remission (B) Number of cycles of induction therapy to achieve first complete remission (1 vs >1) for patients with acute leukemia (B) Treatment related: Donor age (A) Donor recipient gender match (F M vs M F vs M M vs F F) (B) Donor type (child/parent/sibling/other) (B) Donor recipient HLA match (# of antigen mismatches) (A) Donor recipient CMV status: ( / vs others) (B) Conditioning regimen (MA vs. RIC vs. NMA) (B) Conditioning regimen type (A) Infused number of nucleated and CD34+ cells: n/kg recipient weight (A) Infused number of CD3+ cells: n/kg recipient weight (A) Source of stem cells: (BM vs PBSC vs both) (B) Growth factors post transplant: G CSF or GM CSF: (yes/no) (B) Treatment related Second transplant (if applicable): Second transplant for graft failure? (y/n) (B) Second transplant same donor? (y/n) (B) Disease status at transplant (remission vs not) (B) Relapse after transplant? (y/n) (B) GVHD present at time of second BMT? (y/n) (B) 15

16 Not for publication or presentation Attachment 4 Time from relapse post transplant (A) Time from transplant (A) Significance: There are no large studies comparing outcomes of HaploSCT versus matched related donor transplantations. This study would allow us to effectively compare the outcomes while adjusting for the patient and disease related factors, thereby giving us a better understanding of HaploSCT outcomes. Figure 1. References: 1. Copelan, E. A. (2006). "Hematopoietic stem cell transplantation." N Engl J Med 354(17): Washington, D. C. G. A. O. (2002). Bone marrow transplant: despite recruitment successes, national programs may be underutilized. 3. J.S. Passel, W. W., P. Taylor (2011). "Marrying Out: One in Seven New U.S. Marriages is Interracial or Interethnic." Pew Research Center Publications, Washington, DC. 4. Bayraktar, U. D., et al. (2012). "Progress in haploidentical stem cell transplantation." Biol Blood Marrow Transplant 18(3): Falk, P. M., et al. (1978). "Bone marrow transplantation between a histocompatible parent and child for acute leukemia." Transplantation 25(2): Powles, R. L., et al. (1983). "Mismatched family donors for bone marrow transplantation as treatment for acute leukaemia." Lancet 1(8325): Szydlo, R., et al. (1997). "Results of allogeneic bone marrow transplants for leukemia using donors other than HLA identical siblings." J Clin Oncol 15(5):

17 Not for publication or presentation Attachment 4 8. Marmont, A. M., et al. (1991). "T cell depletion of HLA identical transplants in leukemia." Blood 78(8): Luznik, L., et al. (2008). "HLA haploidentical bone marrow transplantation for hematologic malignancies using nonmyeloablative conditioning and high dose, posttransplantation cyclophosphamide." Biol Blood Marrow Transplant 14(6): O'Donnell, P. V., et al. (2002). "Nonmyeloablative bone marrow transplantation from partially HLA mismatched related donors using posttransplantation cyclophosphamide." Biol Blood Marrow Transplant 8(7): Brunstein, C. G., et al. (2011). "Alternative donor transplantation after reduced intensity conditioning: results of parallel phase 2 trials using partially HLA mismatched related bone marrow or unrelated double umbilical cord blood grafts." Blood 118(2): Ciurea, S. O., et al. (2012). "Improved early outcomes using a T cell replete graft compared with T cell depleted haploidentical hematopoietic stem cell transplantation." Biol Blood Marrow Transplant 18(12): Bashey, A., et al. (2013). "T cell replete HLA haploidentical hematopoietic transplantation for hematologic malignancies using post transplantation cyclophosphamide results in outcomes equivalent to those of contemporaneous HLA matched related and unrelated donor transplantation." J Clin Oncol 31(10): Bacigalupo A., et al.(2009). Defining the intensity of conditioning regimens: working definitions. Biol Blood Marrow Transplant 15(12): Cox DR, (1972). " Regression models and life tables [with discussion]." J R Stat Soc B. 1972(34): Kaplan EL, M. P. " Nonparametric estimation for incomplete observations." J Am Stat Assoc 1958( 53): Prentice RL, K. J., Peterson AV "The analysis of failure times in the presence of competing risks." Jr. Biometrics 1978(34(4)):

18 Not for publication or presentation Attachment 4 Characteristic of AML and ALL patients who underwent first allogeneic haploidentical or 8/8 HLA matched unrelated transplant and reported to CIBMTR, Ex vivo T cell depletion N (%) CD34 selection N (%) post Transplant Cy N (%) 8/8 HLA matched unrelated N (%) Variable Number of patients Number of centers Age at transplant, years <10 27 (22) 31 (19) 11 (4) 41 (5) (23) 30 (19) 17 (6) 46 (5) (12) 17 (11) 34 (12) 104 (12) (8) 13 (8) 38 (14) 123 (15) (10) 23 (14) 45 (16) 164 (20) (10) 23 (14) 68 (25) 199 (24) (15) 23 (14) 61 (22) 161 (19) Disease AML 74 (60) 103 (64) 216 (79) 684 (82) ALL 50 (40) 57 (36) 58 (21) 154 (18) Conditioning regimen Myeloablative 82 (66) 105 (66) 109 (40) 603 (72) Reduced intensity 42 (34) 55 (35) 161 (60) 235 (28) 18

19 Not for publication or presentation Attachment 5 Study Proposal Study Title: Haplo identical transplants as an alternative to matched unrelated donor transplants Olle Ringdén, MD, PhD, Professor of Transplantation Immunology Karolinska Institutet Dept. of Laboratory Medicine Division of Therapeutic Immunology, F79 Karolinska University Hospital Huddinge SE Stockholm, Sweden Phone: Fax: Specific Aims: To compare haplo identical transplants using post transplant cyclophosphamide and evaluate if this can be a valid alternative to matched unrelated donor transplants. Evaluate the cumulative probability of acute and chronic GVHD and non relapse mortality (NRM) in the two groups Compare relapse probability, overall survival and disease free survival (DFS) in the two groups Scientific Justification: Haplo identical transplants are increasingly used when there is no HLA identical sibling or HLA matched unrelated donor available 1 3. These protocols used T cell depletion with or without a megadose of CD34 stem cells from the donor to modulate GVHD. In more recent years, reduced intensity conditioning (RIC) and a high dose post transplant cyclophosphamide to eliminate alloreactive T cells from the donor has emerged as a simple way to perform haplo identical bone marrow transplantation 4 6. A promising outcome with this approach has also been confirmed by others 7, 8. In MUD transplants, immunosuppression including ATG, cyclosporine or tacrolimus combined with methotrexate or mycophenolate mofetil are used with subsequent impaired immune reconstitution and with a high incidence of infectious complications. With post transplant cyclophosphamide used in haplo identical transplants, immune reconstitution and T cell immunity against virus and fungi may be preserved 9. Using haplo identical transplants, there are several HLA antigen mismatches. Recipient mismatched HLA antigens on leukemic cells may be targets for an allo reactive donor graft versus leukemia effect. If such an effect will disappear or will be preserved by allo depletion is unknown. Therefore, it is of interest to compare haplo identical transplants using post transplant cyclophosphamide with MUD transplants. Since most patients have access to an haplo identical donor, a sibling, parent or a child, such a graft may be availabe for almost all patients. The aim of the investigation is to find out if haploidentical transplants using post transplant cyclophosphamide may be an alternative to MUD transplants. Haplo identical transplants may also be a cheaper alternative. 19

20 Not for publication or presentation Attachment 5 Patient Eligibility Population: Patients with leukemia (AML, ALL, CML, CLL), MDS and lymphomas reported to CIBMTR and transplanted between 2000 and today (with a minimum follow up of three months), using various conditioning regimens, RIC or MAC. Haplo identical transplants receiving post transplant cyclophosphamide will be compared to MUD transplants (HLA match 8/8 or 7/8). Data Requirements: The CIBMTR Data Base will be used. No additional data will be requested. Sample Requirements: No samples are needed. Variables to be analyzed: Patient related: Age at transplant Gender: male vs. female Karnofsky score pre transplant; <90% vs. 90%. Disease related: AML vs. ALL vs. CML vs. MDS vs. CLL vs. Lymphoma Lymphoma: small lymphocytic, follicular, diffuse large cell, mantel cell, other Disease status at the time of transplant: 1 st CR vs. 2 nd 3 rd CR, vs. relapse AML FAB subtype ALL: immunophenotype; CML: 1 st chronic phase, accelerated phase, blast crisis MDS: RA/RARS vs. RAEB/RAEBt Lymphoma: CR, PR, relapse sensitive, relapse resistant WBC at diagnosis: <25x10 9 /L, 25 50x10 9 /L, x10 9 /L, >100x10 9 /L Duration of CR1 (for patients beyond CR1): <6 months, 6 12 months, >12months. Time from remission to transplant (for patients in CR1): <3 months, 3 6 months, >6 months Time from remission to transplant for CML: <12 months, >12 months Cytogenetics: AML: good vs. intermediate vs. poor vs. no abnormalities vs. missing data ALL: No abnormalities vs. hyperdiploid vs. hypodiploid/ t(9;22)/ t(4;11)/ t(8;14) vs. other abnormalities vs. missing data Transplant related: Source of stem cells: bone marrow vs. peripheral stem cells Donor age: group by 10 year increments Donor recipient gender match: F M vs. M F vs. M M vs. F F Donor recipient CMV status ( / vs. /+ vs. +/ vs. +/+) Conditioning regimen: myeloablative vs. RIC vs. NMC. GVHD prophylaxis: CsA + MTX ± other, CsA ± MMF, FK506 + MTX ± other, FK506 ± other, other. Year of transplant before or after the median HLA match: MUD, HLA compatible 8/8, HLA one antigen mismatched 7/8 20

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