TY - JOUR
T1 - A Phase I/II Multicenter Trial of HLA-Haploidentical PBSCT with PTCy for Aggressive Adult T Cell Leukemia/Lymphoma
AU - Tanaka, Takashi
AU - Nakamae, Hirohisa
AU - Ito, Ayumu
AU - Fuji, Shigeo
AU - Hirose, Asao
AU - Eto, Tetsuya
AU - Henzan, Hideho
AU - Takase, Ken
AU - Yamasaki, Satoshi
AU - Makiyama, Junya
AU - Moriuchi, Yukiyoshi
AU - Choi, Ilseung
AU - Nakano, Nobuaki
AU - Hiramoto, Nobuhiro
AU - Kato, Koji
AU - Sato, Takahiko
AU - Sawayama, Yasushi
AU - Kim, Sung Won
AU - Inoue, Yoshitaka
AU - Inamoto, Yoshihiro
AU - Fukuda, Takahiro
N1 - Publisher Copyright:
© 2021 The American Society for Transplantation and Cellular Therapy
PY - 2021/11
Y1 - 2021/11
N2 - Adult T cell leukemia/lymphoma (ATL) is a highly aggressive hematologic malignancy with a very poor prognosis, and most patients with ATL are elderly. Although post-transplantation cyclophosphamide (PTCy) has yielded promising results in various diseases, available data are limited regarding its outcomes in ATL. The aim of this study was to determine the safety and efficacy of reduced-intensity peripheral blood stem cell transplantation (PBSCT) from a human leukocyte antigen (HLA)-haploidentical donor using PTCy as graft-versus-host disease (GVHD) prophylaxis. This was a prospective, multicenter phase I/II study (UMIN000021783) conducted at 16 hospitals in Japan. The primary endpoint was the probability of survival with engraftment and without grade III/IV acute GVHD at day 60 after PBSCT. The expected probability of the primary endpoint was estimated to be 60%, and the threshold probability was set at 30% on the basis of previous studies. The conditioning regimen consisted of fludarabine (30 mg/m2/d from day –7 to –2), melphalan (40 mg/m2/d on days –3 and –2), and total body irradiation (2 Gy on day –1). GVHD prophylaxis consisted of tacrolimus starting at 0.02 mg/kg/d on day –1, PTCy (50 mg/kg/d on days +3 and +5), and mycophenolate mofetil 2000 mg/d starting on day +6. Eighteen ATL patients underwent PBSCT. The probability of patients who met the primary endpoint was 89% (95% confidence interval, 65% to 99%). The cumulative incidences of grade II to IV acute GVHD, III/IV acute GVHD, and moderate-to-severe chronic GVHD were 39%, 11%, and 17%, respectively. The probabilities of overall survival were 83% at 1 year and 73% at 2 years. The cumulative incidences of non-relapse mortality and disease progression at 1 year were 11% and 28%, respectively. HLA-haploidentical PBSCT with PTCy as GVHD prophylaxis is a valid option for patients with aggressive ATL.
AB - Adult T cell leukemia/lymphoma (ATL) is a highly aggressive hematologic malignancy with a very poor prognosis, and most patients with ATL are elderly. Although post-transplantation cyclophosphamide (PTCy) has yielded promising results in various diseases, available data are limited regarding its outcomes in ATL. The aim of this study was to determine the safety and efficacy of reduced-intensity peripheral blood stem cell transplantation (PBSCT) from a human leukocyte antigen (HLA)-haploidentical donor using PTCy as graft-versus-host disease (GVHD) prophylaxis. This was a prospective, multicenter phase I/II study (UMIN000021783) conducted at 16 hospitals in Japan. The primary endpoint was the probability of survival with engraftment and without grade III/IV acute GVHD at day 60 after PBSCT. The expected probability of the primary endpoint was estimated to be 60%, and the threshold probability was set at 30% on the basis of previous studies. The conditioning regimen consisted of fludarabine (30 mg/m2/d from day –7 to –2), melphalan (40 mg/m2/d on days –3 and –2), and total body irradiation (2 Gy on day –1). GVHD prophylaxis consisted of tacrolimus starting at 0.02 mg/kg/d on day –1, PTCy (50 mg/kg/d on days +3 and +5), and mycophenolate mofetil 2000 mg/d starting on day +6. Eighteen ATL patients underwent PBSCT. The probability of patients who met the primary endpoint was 89% (95% confidence interval, 65% to 99%). The cumulative incidences of grade II to IV acute GVHD, III/IV acute GVHD, and moderate-to-severe chronic GVHD were 39%, 11%, and 17%, respectively. The probabilities of overall survival were 83% at 1 year and 73% at 2 years. The cumulative incidences of non-relapse mortality and disease progression at 1 year were 11% and 28%, respectively. HLA-haploidentical PBSCT with PTCy as GVHD prophylaxis is a valid option for patients with aggressive ATL.
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U2 - 10.1016/j.jtct.2021.07.010
DO - 10.1016/j.jtct.2021.07.010
M3 - Article
C2 - 34274491
AN - SCOPUS:85121157429
SN - 2666-6375
VL - 27
SP - 928.e1-928.e7
JO - Transplantation and Cellular Therapy
JF - Transplantation and Cellular Therapy
IS - 11
ER -
