title: HLA-Haploidentical Peripheral Blood Stem Cell Transplantation with Post-Transplant Cyclophosphamide after Busulfan-Containing Reduced-Intensity Conditioning.
abstract: Allogeneic hematopoietic stem cell transplantation (allo-SCT) using post-transplant cyclophosphamide (PTCy) is increasingly performed. We conducted a multicenter phase II study to evaluate the safety and efficacy of PTCy-based HLA-haploidentical peripheral blood stem cell transplantation (PTCy-haploPBSCT) after busulfan-containing reduced-intensity conditioning. Thirty-one patients were enrolled; 61% patients were not in remission and 42% patients had a history of prior allo-SCT. Neutrophil engraftment was achieved in 87% patients with a median of 19 days. The cumulative incidence of grades II to IV and III to IV acute graft-versus-host disease (GVHD) and chronic GVHD at 1 year were 23%, 3%, and 15%, respectively. No patients developed severe chronic GVHD. Day 100 nonrelapse mortality (NRM) rate was 19.4%. Overall survival, relapse, and disease-free survival rates were 45%, 45%, and 34%, respectively, at 1 year. Subgroup analysis showed that patients who had a history of prior allo-SCT had lower engraftment, higher NRM, and lower overall survival than those not receiving a prior allo-SCT. Our results suggest that PTCy-haploPBSCT after busulfan-containing reduced-intensity conditioning achieved low incidences of acute and chronic GVHD and NRM and stable donor engraftment and low NRM, particularly in patients without a history of prior allo-SCT.
authors.affiliation: Department of Hematology, Hokkaido University Graduate School of Medicine, Sapporo, Japan.
authors.forename: Junichi
authors.initials: J
authors.lastname: Sugita
authors.affiliation: Department of Hematology, Japanese Red Cross Nagoya First Hospital, Nagoya, Japan.
authors.forename: Naomi
authors.initials: N
authors.lastname: Kawashima
authors.affiliation: Department of Hematology, Matsuyama Red Cross Hospital, Matsuyama, Japan.
authors.forename: Tomoaki
authors.initials: T
authors.lastname: Fujisaki
authors.affiliation: Hematology Division, Tokyo Metropolitan Cancer and Infectious diseases Center, Komagome Hospital, Tokyo, Japan.
authors.forename: Kazuhiko
authors.initials: K
authors.lastname: Kakihana
authors.affiliation: Department of Hematology, Sapporo Hokuyu Hospital, Sapporo, Japan.
authors.forename: Shuichi
authors.initials: S
authors.lastname: Ota
authors.affiliation: Department of Preventive Medicine, Kyushu University Faculty of Medical Sciences, Fukuoka, Japan.
authors.forename: Keitaro
authors.initials: K
authors.lastname: Matsuo
authors.affiliation: Department of Hematology/Oncology, Kyushu University Hospital, Fukuoka, Japan.
authors.forename: Toshihiro
authors.initials: T
authors.lastname: Miyamoto
authors.affiliation: Department of Hematology/Oncology, Kyushu University Hospital, Fukuoka, Japan.
authors.forename: Koichi
authors.initials: K
authors.lastname: Akashi
authors.affiliation: Department of Hematology, Toranomon Hospital, Tokyo, Japan.
authors.forename: Shuichi
authors.initials: S
authors.lastname: Taniguchi
authors.affiliation: Karatsu Higashimatsuura Medical Center, Karatsu, Japan.
authors.forename: Mine
authors.initials: M
authors.lastname: Harada
authors.affiliation: Department of Hematology, Hokkaido University Graduate School of Medicine, Sapporo, Japan. Electronic address: teshima@med.hokudai.ac.jp.
authors.forename: Takanori
authors.initials: T
authors.lastname: Teshima
mesh_terms: D000293:Adolescent; D000328:Adult; D000368:Aged; D002066:Busulfan / Q000008:administration & dosage*; D003520:Cyclophosphamide / Q000008:administration & dosage*; D018572:Disease-Free Survival; D005260:Female; D006680:HLA Antigens; D019337:Hematologic Neoplasms / Q000401:mortality / Q000628:therapy; D006801:Humans; D008297:Male; D008875:Middle Aged; D036102:Peripheral Blood Stem Cell Transplantation; D011446:Prospective Studies; D015996:Survival Rate; D019172:Transplantation Conditioning
chemical_list: D006680:HLA Antigens; D003520:Cyclophosphamide; D002066:Busulfan
keywords: Busulfan; HLA-haploidentical transplantation; Post-transplant cyclophosphamide; Reduced-intensity conditioning