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Posttransplant cyclophosphamide in unrelated and related peripheral blood stem cell transplantation from HLA-matched and 1 allele mismatched donor

Bone Marrow Transplantation volume 59, pages 344–349 (2024)Cite this article

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Abstract

Posttransplant cyclophosphamide (PTCy)-based graft-versus-host disease (GVHD) prophylaxis has been increasingly used in HLA-haploidentical transplantation and recent studies also demonstrated the efficacy of PTCy in HLA-matched transplantation. We conducted a prospective multicenter phase II study to evaluate the safety and efficacy of PTCy with tacrolimus and mycophenolate mofetil in 43 patients who underwent HLA-matched (n = 21), 1 allele mismatched (n = 20), or 2 allele mismatched (n = 2) peripheral blood stem cell transplantation (PBSCT) following myeloablative (n = 28) or reduced-intensity (n = 15) conditioning. The incidence of grade III–IV acute GVHD at 100 days was 2.3%. The incidences of grades II–IV acute GVHD, all grade chronic GVHD, and moderate to severe chronic GVHD at 2 years were 16.3%, 14.0%, and 4.7%, respectively. Overall survival, disease-free survival, and non-relapse mortality at 2 years were 75.3%, 74.0%, and 7.0%, respectively. GVHD-free, relapse-free survival at 2 years was 67.0%. The rate of off-immunosuppressants in patients who survived without relapse at 2 years was 85.4%. These results indicate that PTCy is a valid option for GVHD prophylaxis in both HLA-matched and HLA 1–2 allele mismatched PBSCT.

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Fig. 1: Clinical outcomes.

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Data availability

The dataset supporting the conclusions of this article is available upon request to the corresponding author.

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Acknowledgements

This work was supported by Japan Agency for Medical Research and Development (AMED, 20ek0510025h0003).

Author information

Authors and Affiliations

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

    Junichi Sugita & Takanori Teshima

  2. Department of Hematology, Sapporo Hokuyu Hospital, Sapporo, Japan

    Junichi Sugita

  3. Department of Hematology, Nagaoka Red Cross Hospital, Nagaoka, Japan

    Takashi Kuroha

  4. Department of Hematology, Osaka International Cancer Institute, Osaka, Japan

    Jun Ishikawa

  5. Department of Hematology, Hamanomachi Hospital, Fukuoka, Japan

    Tetsuya Eto & Shuichi Taniguchi

  6. Department of Hematology and Oncology, Osaka University Hospital, Suita, Japan

    Kentaro Fukushima

  7. Department of Biostatistics, Graduate School of Medicine, Hokkaido University, Sapporo, Japan

    Isao Yokota

  8. Department of Hematology/Oncology, Kyushu University Hospital, Fukuoka, Japan

    Koichi Akashi

  9. Karatsu Higashimatsuura Medical Center, Karatsu, Japan

    Mine Harada

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  1. Junichi Sugita
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Contributions

JS designed the study, recruited patients, collected, assembled, and analyzed data, performed the statistical analysis, and wrote the manuscript. TT designed the study, supervised research, analyzed data, and commented on the manuscript. KA, ST, and MH helped design the study, and commented on the manuscript. IY analyzed data, performed the statistical analysis, and commented on the manuscript. TK, JI, TE, and KF recruited patients and commented on the manuscript. All authors reviewed the manuscript and approved its submission for publication purposes.

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Correspondence to Takanori Teshima.

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Sugita, J., Kuroha, T., Ishikawa, J. et al. Posttransplant cyclophosphamide in unrelated and related peripheral blood stem cell transplantation from HLA-matched and 1 allele mismatched donor. Bone Marrow Transplant 59, 344–349 (2024). https://doi.org/10.1038/s41409-023-02162-6

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