We conducted two parallel prospective, multicenter, phase II studies to evaluate the safety and efficacy of HLA-haploidentical peripheral blood stem cell transplantation using post-transplant cyclophosphamide (PTCy-haploPBSCT) following myeloablative conditioning (MAC, n = 50) and reduced-intensity conditioning (RIC, n = 77). Event-free survival (EFS) at 1-year as for primary endpoint was 64% and 43% in the MAC and RIC groups, respectively. Neutrophil engraftment was achieved in 98% and 94% in the MAC and RIC groups, respectively. The incidences of grades II–IV and III–IV acute graft-versus-host disease (GVHD) were 18% and 8% in the MAC group, and 14% and 5% in the RIC group, respectively. Those of all grade and moderate to severe chronic GVHD at 2-year were 36% and 20% in the MAC group, and 27% and 20% in the RIC group, respectively. Overall survival (OS), EFS, nonrelapse mortality, and relapse rate at 2-year were 68%, 54%, 10%, and 36% in the MAC group, and 44%, 35%, 20%, and 45% in the RIC group, respectively. Notably, 83% and 86% of patients who survived without relapse stopped immunosuppressant at 2-year in the MAC and RIC groups, respectively. Our results indicate that both MAC and RIC are valid options for PTCy-haploPBSCT for adults with hematological malignancies.

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This work was supported by the grant from Regional Medicine Research Foundation (Tochigi, Japan), North Japan Hematology Study Group (NJHSG), and Japan Agency for Medical Research and Development (AMED, JP17ek0510012).

Author information


  1. Hematology, Faculty of Medicine, Hokkaido University, Sapporo, Japan

    • Junichi Sugita
    •  & Takanori Teshima
  2. Hematology, Japanese Red Cross Nagoya First Hospital, Nagoya, Japan

    • Yusuke Kagaya
  3. Hematology/Oncology, Kyushu University Hospital, Fukuoka, Japan

    • Toshihiro Miyamoto
    •  & Koichi Akashi
  4. Stem Cell Transplantation, Niigata University Medical and Dental Hospital, Niigata, Japan

    • Yasuhiko Shibasaki
  5. Hematology and Oncology, Kurume University School of Medicine, Kurume, Japan

    • Koji Nagafuji
  6. Hematology, Sapporo Hokuyu Hospital, Sapporo, Japan

    • Shuichi Ota
  7. Hematology, Nagaoka Red Cross Hospital, Nagaoka, Japan

    • Tatsuo Furukawa
  8. Hematology/Nephrology/Rheumatology, Akita University Hospital, Akita, Japan

    • Miho Nara
  9. Hematology, Toranomon Hospital, Tokyo, Japan

    • Shuichi Taniguchi
  10. Karatsu Higashimatsuura Medical Center, Karatsu, Japan

    • Mine Harada
  11. Molecular and Clinical Epidemiology, Aichi Cancer Center Research Institute, Nagoya, Japan

    • Keitaro Matsuo


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  1. on behalf of the Japan Study Group for Cell Therapy and Transplantation (JSCT)

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    Correspondence to Takanori Teshima.

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