TY - JOUR
T1 - Results of a 2-arm, phase 2 clinical trial using post-transplantation cyclophosphamide for the prevention of graft-versus-host disease in haploidentical donor and mismatched unrelated donor hematopoietic stem cell transplantation
AU - Gaballa, Sameh
AU - Ge, Isabell
AU - El Fakih, Riad
AU - Brammer, Jonathan E.
AU - Kongtim, Piyanuch
AU - Tomuleasa, Ciprian
AU - Wang, Sa A.
AU - Lee, Dean
AU - Petropoulos, Demetrios
AU - Cao, Kai
AU - Rondon, Gabriela
AU - Chen, Julianne
AU - Hammerstrom, Aimee
AU - Lombardi, Lindsey
AU - Alatrash, Gheath
AU - Korbling, Martin
AU - Oran, Betul
AU - Kebriaei, Partow
AU - Ahmed, Sairah
AU - Shah, Nina
AU - Rezvani, Katayoun
AU - Marin, David
AU - Bashir, Qaiser
AU - Alousi, Amin
AU - Nieto, Yago
AU - Qazilbash, Muzaffar
AU - Hosing, Chitra
AU - Popat, Uday
AU - Shpall, Elizabeth J.
AU - Khouri, Issa
AU - Champlin, Richard E.
AU - Ciurea, Stefan O.
N1 - Publisher Copyright:
© 2016 American Cancer Society
PY - 2016/11/15
Y1 - 2016/11/15
N2 - BACKGROUND: High-dose, post-transplantation cyclophosphamide (PTCy) to prevent graft-versus-host disease (GVHD) has improved outcomes in haploidentical (HAPLO) stem cell transplantation (SCT). However, it remains unclear whether this strategy is effective in SCT from 1-antigen human leukocyte antigen (HLA)-mismatched unrelated donors (9/10 MUD) and how the outcomes of these patients compare with those of haploidentical transplantation recipients. METHODS: A parallel, 2-arm, nonrandomized phase 2 clinical trial was conducted of melphalan-based reduced-intensity conditioning with PTCy, tacrolimus, and mycophenolate mofetil to prevent GVHD in patients with high-risk hematologic malignancies who underwent HAPLO (n = 60) or 9/10 MUD (n = 46) SCT. RESULTS: The 1-year overall and progression-free survival rates were 70% and 60%, respectively, in the HAPLO arm and 60% and 47%, respectively, in the 9/10 MUD arm. The day +100 cumulative incidence of grade II to IV acute GVHD and grade III to IV acute GVHD was 28% and 3%, respectively, in the HAPLO arm and 33% and 13%, respectively, in the 9/10 MUD arm. The 2-year cumulative incidence of chronic GVHD was 24% in the HAPLO arm and 19% in the 9/10 MUD arm. The 1-year cumulative incidence of nonrelapse mortality was 21% in the HAPLO arm and 31% in the 9/10 MUD arm, and the 1-year relapse rate was 19% in the HAPLO arm and 25% in the 9/10 MUD arm. CONCLUSIONS: Although this was a nonrandomized study and could not serve as a direct comparison between the 2 groups, the authors conclude that PTCy-based GVHD prophylaxis is effective for both HAPLO and 9/10 MUD SCTs. Prospective randomized trials will be required to compare the efficacies of alternative donor options for patients lacking HLA-matched donors. Cancer 2016;122:3316–3326.
AB - BACKGROUND: High-dose, post-transplantation cyclophosphamide (PTCy) to prevent graft-versus-host disease (GVHD) has improved outcomes in haploidentical (HAPLO) stem cell transplantation (SCT). However, it remains unclear whether this strategy is effective in SCT from 1-antigen human leukocyte antigen (HLA)-mismatched unrelated donors (9/10 MUD) and how the outcomes of these patients compare with those of haploidentical transplantation recipients. METHODS: A parallel, 2-arm, nonrandomized phase 2 clinical trial was conducted of melphalan-based reduced-intensity conditioning with PTCy, tacrolimus, and mycophenolate mofetil to prevent GVHD in patients with high-risk hematologic malignancies who underwent HAPLO (n = 60) or 9/10 MUD (n = 46) SCT. RESULTS: The 1-year overall and progression-free survival rates were 70% and 60%, respectively, in the HAPLO arm and 60% and 47%, respectively, in the 9/10 MUD arm. The day +100 cumulative incidence of grade II to IV acute GVHD and grade III to IV acute GVHD was 28% and 3%, respectively, in the HAPLO arm and 33% and 13%, respectively, in the 9/10 MUD arm. The 2-year cumulative incidence of chronic GVHD was 24% in the HAPLO arm and 19% in the 9/10 MUD arm. The 1-year cumulative incidence of nonrelapse mortality was 21% in the HAPLO arm and 31% in the 9/10 MUD arm, and the 1-year relapse rate was 19% in the HAPLO arm and 25% in the 9/10 MUD arm. CONCLUSIONS: Although this was a nonrandomized study and could not serve as a direct comparison between the 2 groups, the authors conclude that PTCy-based GVHD prophylaxis is effective for both HAPLO and 9/10 MUD SCTs. Prospective randomized trials will be required to compare the efficacies of alternative donor options for patients lacking HLA-matched donors. Cancer 2016;122:3316–3326.
KW - 9/10 matched unrelated donors
KW - graft-versus-host disease
KW - haploidentical transplantation
KW - hematologic malignancies
KW - post-transplantation cyclophosphamide
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U2 - 10.1002/cncr.30180
DO - 10.1002/cncr.30180
M3 - Article
C2 - 27404668
AN - SCOPUS:84991738599
SN - 0008-543X
VL - 122
SP - 3316
EP - 3326
JO - Cancer
JF - Cancer
IS - 21
ER -