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Comparable survival outcomes with haploidentical stem cell transplantation and cord blood transplantation

Bone Marrow Transplantation volume 57, pages 1681–1688 (2022)Cite this article

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Abstract

HLA-haploidentical stem cell transplantation using post-transplant cyclophosphamide (PTCy-haplo) and umbilical cord blood transplantation (UCBT) are alternative to HLA-matched stem cell transplantation. We conducted a matched-pair analysis of PTCy-haplo and UCBT using the Japanese registry data. We identified 136 patients aged between 16 and 69 years who received PTCy-haplo as their first transplantation for acute leukemia or myelodysplastic syndromes. Control group included 408 UCBT recipients selected to match the PTCy-haplo group. Overall and relapse-free survival probabilities at 2 years were comparable between the PTCy-haplo and UCBT groups: 55% vs. 53% for overall survival (p = 0.46), and 47% vs. 48% for relapse-free survival (p = 0.79), respectively. The cumulative incidence of relapse was significantly higher (43% vs. 29%, respectively, p = 0.006), while the cumulative incidence of non-relapse mortality (NRM) was significantly lower (9% vs. 23%, respectively, p < 0.001) in the PTCy-haplo group. The cumulative incidence of grade II-IV acute graft-versus-host disease (GVHD) was lower in the PTCy-haplo group compared to the UCBT group (29% vs. 41%, respectively, p = 0.016), while those of grade III-IV acute GVHD and chronic GVHD were not statistically different between the two groups. Our results suggest that both PTCy-haplo and UCBT are viable alternatives to HLA-matched stem cell transplantation.

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Fig. 1: Probabilities of overall and relapse-free survival.
Fig. 2: Cumulative incidences of relapse and non-relapse mortality.
Fig. 3: Cumulative incidences of acute and chronic GVHD.
Fig. 4: Cumulative incidences of neutrophil and platelet recovery.
Fig. 5: Probabilities of overall survival stratified by revised DRI.

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Acknowledgements

The authors are grateful for the assistance and cooperation of all the staff members at the following collaborating institutes: the Japan Society for Hematopoietic Cell Transplantation, the Japan Marrow Donor Program, and the Japanese Data Center for Hematopoietic Cell Transplantation. This work was supported in part by the Practical Research Project for Allergic Diseases and Immunology (Research Technology of Medical Transplantation) of the Japan Agency for Medical Research and Development (AMED) under Grant Numbers 19ek0510023h0002 and 19ek0510023h0002.

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Author notes

  1. These authors contributed equally: Junichi Sugita, Yoshiko Atsuta.

Authors and Affiliations

  1. Department of Hematology, Hokkaido University Hospital, Sapporo, Japan

    Junichi Sugita, Souichi Shiratori & Takanori Teshima

  2. Japanese Data Center for Hematopoietic Cell Transplantation, Nagakute, Japan

    Yoshiko Atsuta & Mio Kurata

  3. Department of Registry Science for Transplant and Cellular Therapy, Aichi Medical University School of Medicine, Nagakute, Japan

    Yoshiko Atsuta

  4. Hematology, Osaka City University Hospital, Osaka, Japan

    Hirohisa Nakamae

  5. Department of Hematology, University of Tsukuba Hospital, Tsukuba, Japan

    Yumiko Maruyama

  6. Department of Hematology, Kanazawa University Hospital, Kanazawa, Japan

    Ken Ishiyama

  7. Department of Hematopoietic Stem Cell Transplantation, National Cancer Center Hospital, Tokyo, Japan

    Takahiro Fukuda

  8. Hematology Division, Tokyo Metropolitan Cancer and Infectious Diseases Center, Komagome Hospital, Tokyo, Japan

    Naoki Shingai

  9. Department of Hematology, Japanese Red Cross Aichi Medical Center Nagoya Daiichi Hospital, Nagoya, Japan

    Yukiyasu Ozawa

  10. Department of Hematopoietic Cell Therapy, Niigata University Medical and Dental Hospital, Niigata, Japan

    Masayoshi Masuko

  11. Division of Hematology and Oncology, Department of Medicine, Kurume University Hospital, Kurume, Japan

    Koji Nagafuji

  12. Department of Hematology, Federation of National Public Service Personnel Mutual Aid Associations Toranomon Hospital, Tokyo, Japan

    Naoyuki Uchida

  13. Department of Hematology, Kanagawa Cancer Center, Kanagawa, Japan

    Masatsugu Tanaka

  14. Department of Hematology/Oncology, Tokai University School of Medicine, Isehara, Japan

    Makoto Onizuka

  15. Department of Hematology and Oncology, Graduate School of Medicine, Kyoto University, Kyoto, Japan

    Junya Kanda

  16. Preparation Department, Japanese Red Cross Kinki Block Blood Center, Ibaraki, Japan

    Takafumi Kimura

  17. Department of Hematology and Oncology, Research Institute for Radiation Biology and Medicine, Hiroshima University, Hiroshima, Japan

    Tatsuo Ichinohe

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Contributions

JS, YA, and TT designed the study and wrote the paper; MK and YA analyzed the results and created the figures; JK, TK, and TI. reviewed and cleaned the clinical data and reviewed the results; HN, YM, KI, SS, TF, NS, YO, MM, KN, NU, MT, and MO submitted and cleaned the clinical data and reviewed the results.

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Correspondence to Takanori Teshima.

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Sugita, J., Atsuta, Y., Nakamae, H. et al. Comparable survival outcomes with haploidentical stem cell transplantation and cord blood transplantation. Bone Marrow Transplant 57, 1681–1688 (2022). https://doi.org/10.1038/s41409-022-01770-y

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