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. 2024 Aug 27;8(16):4281-4293.
doi: 10.1182/bloodadvances.2024013451.

Donor types and outcomes of transplantation in myelofibrosis: a CIBMTR study

Tania Jain  1 , Noel Estrada-Merly  2 , M Queralt Salas  3 , Soyoung Kim  2   4 , Jakob DeVos  2 , Min Chen  2 , Xi Fang  4 , Rajat Kumar  5 , Marcio Andrade-Campos  6 , Hany Elmariah  7 , Vaibhav Agrawal  8 , Mahmoud Aljurf  9 , Ulrike Bacher  10 , Talha Badar  11 , Sherif M Badawy  12   13 , Karen Ballen  14 , Amer Beitinjaneh  15 , Vijaya Raj Bhatt  16 , Christopher Bredeson  17 , Zachariah DeFilipp  18 , Bhagirathbhai Dholaria  19 , Nosha Farhadfar  20 , Shatha Farhan  21 , Arpita P Gandhi  22 , Siddhartha Ganguly  23 , Usama Gergis  24 , Michael R Grunwald  25 , Nada Hamad  26   27   28 , Betty K Hamilton  29 , Yoshihiro Inamoto  30 , Madiha Iqbal  11 , Omer Jamy  31 , Mark Juckett  32 , Mohamed A Kharfan-Dabaja  11 , Maxwell M Krem  33 , Deepesh P Lad  34 , Jane Liesveld  35 , Monzr M Al Malki  36 , Adriana K Malone  37 , Hemant S Murthy  11 , Guillermo Ortí  38 , Sagar S Patel  39 , Attaphol Pawarode  40 , Miguel-Angel Perales  41   42 , Marjolein van der Poel  43 , Olle Ringden  44 , David A Rizzieri  45 , Alicia Rovó  46 , Bipin N Savani  47 , Mary Lynn Savoie  48 , Sachiko Seo  49 , Melhem Solh  50 , Celalettin Ustun  51 , Leo F Verdonck  52 , John R Wingard  53 , Baldeep Wirk  54 , Nelli Bejanyan  7 , Richard J Jones  1 , Taiga Nishihori  55   56 , Betul Oran  57 , Ryotaro Nakamura  36 , Bart Scott  58 , Wael Saber  2 , Vikas Gupta  5
Affiliations

Donor types and outcomes of transplantation in myelofibrosis: a CIBMTR study

Tania Jain et al. Blood Adv. .

Abstract

We evaluate the impact of donor types on outcomes of hematopoietic cell transplantation (HCT) in myelofibrosis, using the Center for International Blood and Marrow Transplant Research registry data for HCTs done between 2013 and 2019. In all 1597 patients, the use of haploidentical donors increased from 3% in 2013 to 19% in 2019. In study-eligible 1032 patients who received peripheral blood grafts for chronic-phase myelofibrosis, 38% of recipients of haploidentical HCT were non-White/Caucasian. Matched sibling donor (MSD)-HCTs were associated with superior overall survival (OS) in the first 3 months (haploidentical hazard ratio [HR], 5.80 [95% confidence interval (CI), 2.52-13.35]; matched unrelated (MUD) HR, 4.50 [95% CI, 2.24-9.03]; mismatched unrelated HR, 5.13 [95% CI, 1.44-18.31]; P < .001). This difference in OS aligns with lower graft failure with MSD (haploidentical HR, 6.11 [95% CI, 2.98-12.54]; matched unrelated HR, 2.33 [95% CI, 1.20-4.51]; mismatched unrelated HR, 1.82 [95% CI, 0.58-5.72]). There was no significant difference in OS among haploidentical, MUD, and mismatched unrelated donor HCTs in the first 3 months. Donor type was not associated with differences in OS beyond 3 months after HCT, relapse, disease-free survival, or OS among patients who underwent HCT within 24 months of diagnosis. Patients who experienced graft failure had more advanced disease and commonly used nonmyeloablative conditioning. Although MSD-HCTs were superior, there is no significant difference in HCT outcomes from haploidentical and MUDs. These results establish haploidentical HCT with posttransplantation cyclophosphamide as a viable option in myelofibrosis, especially for ethnic minorities underrepresented in the donor registries.

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Conflict of interest statement

Conflict-of-interest disclosure: T.J. reports receiving institutional research support from CTI BioPharma, Kartos Therapeutics, Incyte, and Bristol Myers Squibb (BMS); and advisory board participation with CareDx, BMS, Incyte, AbbVie, CTI, Kite, Cogent Biosciences, Blueprint Medicine, Telios Pharma, Protagonist Therapeutics, TScan Therapeutics, Karyopharm, and MorphoSys. R.K. reports advisory board participation for Vertex Pharmaceuticals. M.A.C. reports becoming an AstraZeneca employee while on Hospital del Mar; and advisory board or consulting fees from Novartis, Pfizer, BMS, Takeda, and Sanofi. H.E. reports advisory board for Shoreline Biosciences; and research funding from BMS. T.B. reports advisory board fees from Pfizer, Takeda, and MorphoSys. K.B. reports receiving research funding to her institution from Stemline Therapeutics. V.R.B. reports participating in safety monitoring committee for Protagonist, serving as an associate editor for the journal Current Problems in Cancer and as a contributor for BMJ Best Practice; consulting fees from Taiho, Sanofi, Imugene, Genentech, Incyte, Servier Pharmaceuticals LLC, and AbbVie; research funding (institutional) from MEI Pharma, Actinium Pharmaceutical, Sanofi US Services, AbbVie, Pfizer, Incyte, Jazz, and National Marrow Donor Program; and drug support (institutional) from Chimerix for a trial. Z.D. reports research support from Incyte, Corp, REGiMMUNE, Corp, and Taiho Oncology, Inc; and consulting fees from Sanofi, Incyte, Corp, MorphoSys AG, Inhibrx, PharmaBiome AG, and Ono Pharmaceutical. N.F. has been an advisory board member for Incyte; received speaker fees from Incyte; is on the data safety monitoring committee for Chronic Graft-versus-Host Disease Consortium; and is the medical monitor for Blood and Marrow Transplant Clinical Trial Network. A.P.G. reports advisory board/research funding from Orca Bio and Jasper Therapeutics. U.G. reports consulting and speaker bureau fees from Incyte. M.R.G. reports consulting fees from AbbVie, Amgen, Astellas, Blueprint Medicines, BMS, Cardinal Health, Sobi/CTI BioPharma, Daiichi Sankyo, Gamida Cell, Genentech, Gilead Sciences, GlaxoSmithKline (GSK)/Sierra Oncology, Incyte, Invitae, Jazz, Novartis, Ono Pharmaceutical, Pfizer, Pharmacosmos, Premier, Servier/Agios, and Stemline Therapeutics; research support from Incyte and Janssen; and stock ownership in Medtronic. N.H. reports advisory board participation with Novartis, Gilead, Janssen, AbbVie, Pfizer, Incyte, Roche, Takeda, Jazz Pharmaceuticals, Link Pharmaceuticals, and Mallinckrodt. B.K.H. reports advisory committees/consultancy fees from Nkarta, Sanofi, Incyte, and Equilium; DSMB fees from Angiocrine Bioscience; and adjudication committee fees from CSL Behring. O.J. reports advisory board participation for Ascentage Pharma. M.A.K.-D. reports grant/research support from BMS, Novartis, and Pharmacyclics; and lecture/speaking engagement fees from Kite Pharma. C.U. reports honoraria for being in speaker bureau for Takeda and Blueprint. J.R.W. reports consultant fees from Ansun, Celgene, Cidara Therapeutics, F2G, Orca Bio, and Takeda. T.N. reports clinical trial support by Novartis to the institution; and clinical trial support (drug only supply) by Karyopharm to the institution. M.A.P. reports honoraria from Adicet Bio, Allogene, Allovir, Caribou Biosciences, Celgene, BMS, Equilibrium, ExeVir, ImmPACT Bio, Incyte, Karyopharm, Kite/Gilead, Merck, Miltenyi Biotec, MorphoSys, Nektar Therapeutics, Novartis, Omeros, Orca Bio, Sanofi, Syncopation, VectivBio AG, and Vor Biopharma; serves on DSMBs for Cidara Therapeutics and Sellas Life Sciences; and the scientific advisory board of NexImmune; has ownership interests in NexImmune, Omeros, and Orca Bio; received institutional research support for clinical trials from Allogene, Incyte, Kite/Gilead, Miltenyi Biotec, Nektar Therapeutics, and Novartis; serves in a volunteer capacity as a member of the board of directors of the American Society for Transplantation and Cellular Therapy and on the Center for International Blood and Marrow Transplant Research Cellular Immunotherapy Data Resource Executive Committee; and previously served on the board of directors of Be The Match (National Marrow Donor Program). V.G. reports consulting fees from AbbVie, BMS/Celgene, GSK, Novartis, Incyte, CTI BioPharma, and Pfizer; and participation in the data safety monitoring board or advisory board for AbbVie, Incyte, GSK, BMS/Celgene, Pfizer, and Roche. The remaining authors declare no competing financial interests.

Figures

None
Graphical abstract
Figure 1.
Figure 1.
Trends of donor utilization between 2013 and 2019.
Figure 2.
Figure 2.
Outcomes of HCT in myelofibrosis by donor type. (A) Adjusted OS. (B) Adjusted graft failure. (C) Adjusted NRM.
Figure 3.
Figure 3.
OS Time to HCT ≤24 months.
See this image and copyright information in PMC

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