TY - JOUR
T1 - Three-Year Outcomes in Recipients of Mismatched Unrelated Bone Marrow Donor Transplants Using Post-Transplantation Cyclophosphamide
T2 - Follow-Up from a National Marrow Donor Program-Sponsored Prospective Clinical Trial
AU - Shaw, Bronwen E.
AU - Jimenez-Jimenez, Antonio Martin
AU - Burns, Linda J.
AU - Logan, Brent R.
AU - Khimani, Farhad
AU - Shaffer, Brian C.
AU - Shah, Nirav N.
AU - Mussetter, Alisha
AU - Tang, Xiao Ying
AU - McCarty, John M.
AU - Alavi, Asif
AU - Farhadfar, Nosha
AU - Jamieson, Katarzyna
AU - Hardy, Nancy M.
AU - Choe, Hannah
AU - Ambinder, Richard F.
AU - Anasetti, Claudio
AU - Perales, Miguel Angel
AU - Spellman, Stephen R.
AU - Howard, Alan
AU - Komanduri, Krishna V.
AU - Luznik, Leo
AU - Norkin, Maxim
AU - Pidala, Joseph A.
AU - Ratanatharathorn, Voravit
AU - Confer, Dennis L.
AU - Devine, Steven M.
AU - Horowitz, Mary M.
AU - Bolaños-Meade, Javier
N1 - Publisher Copyright:
© 2023 The American Society for Transplantation and Cellular Therapy
PY - 2023/3
Y1 - 2023/3
N2 - The use of post-transplantation cyclophosphamide (PTCy) as graft-versus-host disease (GVHD) prophylaxis has resulted in reductions in GVHD and improved outcomes in allogeneic hematopoietic cell transplantation (HCT) using HLA-mismatched related donors. We report the 3-year outcomes of the first multicenter prospective clinical trial using PTCy in the setting of mismatched unrelated donor (MMUD) bone marrow HCT. The study enrolled 80 patients, treated with either myeloablative conditioning (MAC; n = 40) or reduced-intensity conditioning (RIC; n = 40), with the primary endpoint of 1-year overall survival (OS). The median follow-up for this study was 34 months (range, 12 to 46 months) in the RIC group and 36 months (range, 18 to 49 months) in the MAC group. Three-year OS and nonrelapse mortality were 70% and 15%, respectively, in the RIC group and 62% and 10% in the MAC group. No GVHD was reported after 1 year. The incidence of relapse was 29% in the RIC group and 51% in the MAC group. OS did not differ based on HLA match grade (63% in the 7/8 strata and 71% in the 4 to 6/8 strata). These encouraging outcomes, which were sustained for 3 years post-HCT, support the continued exploration of MMUD HCT using a PTCy platform. Important future areas to address include relapse reduction and furthering our understanding of optimal donor selection based on HLA and non-HLA factors.
AB - The use of post-transplantation cyclophosphamide (PTCy) as graft-versus-host disease (GVHD) prophylaxis has resulted in reductions in GVHD and improved outcomes in allogeneic hematopoietic cell transplantation (HCT) using HLA-mismatched related donors. We report the 3-year outcomes of the first multicenter prospective clinical trial using PTCy in the setting of mismatched unrelated donor (MMUD) bone marrow HCT. The study enrolled 80 patients, treated with either myeloablative conditioning (MAC; n = 40) or reduced-intensity conditioning (RIC; n = 40), with the primary endpoint of 1-year overall survival (OS). The median follow-up for this study was 34 months (range, 12 to 46 months) in the RIC group and 36 months (range, 18 to 49 months) in the MAC group. Three-year OS and nonrelapse mortality were 70% and 15%, respectively, in the RIC group and 62% and 10% in the MAC group. No GVHD was reported after 1 year. The incidence of relapse was 29% in the RIC group and 51% in the MAC group. OS did not differ based on HLA match grade (63% in the 7/8 strata and 71% in the 4 to 6/8 strata). These encouraging outcomes, which were sustained for 3 years post-HCT, support the continued exploration of MMUD HCT using a PTCy platform. Important future areas to address include relapse reduction and furthering our understanding of optimal donor selection based on HLA and non-HLA factors.
KW - HLA
KW - Hematopoietic cell transplant
KW - Mismatched unrelated donor
KW - Post transplant cyclophosphamide
UR - http://www.scopus.com/inward/record.url?scp=85146784394&partnerID=8YFLogxK
UR - http://www.scopus.com/inward/citedby.url?scp=85146784394&partnerID=8YFLogxK
U2 - 10.1016/j.jtct.2022.12.017
DO - 10.1016/j.jtct.2022.12.017
M3 - Article
C2 - 36584941
AN - SCOPUS:85146784394
SN - 2666-6367
VL - 29
SP - 208.e1-208.e6
JO - Transplantation and Cellular Therapy
JF - Transplantation and Cellular Therapy
IS - 3
ER -