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Outcome of allogeneic hematopoietic stem cell transplantation in adult patients with paroxysmal nocturnal hemoglobinuria

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Abstract

The safety and efficacy of allogeneic hematopoietic stem cell transplantation (HSCT) for paroxysmal nocturnal hemoglobinuria (PNH) remain unclear. Therefore, we retrospectively analyzed the outcomes of 42 adult patients with PNH who underwent allogeneic HSCT using the registry database of the Japan Society for Hematopoietic Cell Transplantation. The median patient age was 32.5 years. The number of packed red cell (PRC) transfusions was < 20 times in 19 patients and ≥ 20 times in 16; 7 patients had missing data. Stem cell sources were bone marrow (N = 15) or peripheral blood (N = 13) from a related donor or bone marrow (N = 11) and cord blood (N = 3) from an unrelated donor. The cumulative incidence of neutrophil engraftment at day 40 was 81%. Six patients died before engraftment, and the 6-year overall survival (OS) was 74%. The OS of patients with < 20 pretransplant PRC transfusions was significantly higher than that of patients with ≥ 20 pretransplant PRC transfusions (95% vs. 63%; P < 0.05). Moreover, the OS of patients aged < 30 years was significantly higher than that of patients aged ≥ 30 years (90% vs. 59%; P < 0.05). Allogeneic HSCT for PNH could provide favorable survival; however, pretransplant transfusion burden and patient age should be considered when deciding the timing of allogeneic HSCT.

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Acknowledgements

The authors would like to thank all the physicians and data managers of each transplant center and the staff members of 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) from the Japan Agency for Medical Research and Development (AMED) under grant number 19ek0510023h0002.

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Authors and Affiliations

  1. Third Department of Internal Medicine, Yamaguchi University School of Medicine, 1-1-1 Minamikogushi, Ube, Yamaguchi, 755-8505, Japan

    Yukinori Nakamura

  2. Department of Hematology, Clinical Immunology and Infectious Diseases, Ehime University Graduate School of Medicine, Ehime, Japan

    Katsuto Takenaka

  3. Division of Transfusion Medicine, Kanazawa University Hospital, Kanazawa, Japan

    Hirohito Yamazaki

  4. Department of Hematology and Rheumatology, Tohoku University Hospital, Miyagi, Japan

    Yasushi Onishi

  5. Department of Hematology, Japanese Red Cross Nagoya First Hospital, Aichi, Japan

    Yukiyasu Ozawa

  6. Department of Hematology, Hyogo College of Medicine Hospital, Hyogo, Japan

    Kazuhiro Ikegame

  7. Department of Hematology and Oncology, Okayama University Hospital, Okayama, Japan

    Ken-ichi Matsuoka

  8. Department of Internal Medicine III, Division of Hematology and Cell Therapy, Yamagata University Faculty of Medicine, Yamagata, Japan

    Tomomi Toubai

  9. Department of Hematology/Oncology and Transfusion and Hemapheresis Center, Kurashiki Central Hospital, Okayama, Japan

    Yasunori Ueda

  10. Division of Hematology, Jichi Medical University, Tochigi, Japan

    Yoshinobu Kanda

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

    Tatsuo Ichinohe

  12. Japanese Data Center for Hematopoietic Cell Transplantation, Aichi, Japan

    Yoshiko Atsuta

  13. Department of Healthcare Administration, Nagoya University Graduate School of Medicine, Aichi, Japan

    Yoshiko Atsuta

  14. Division of Hematology, Department of Medicine, Keio University School of Medicine, Tokyo, Japan

    Takehiko Mori

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  1. Yukinori Nakamura
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  2. Katsuto Takenaka
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  3. Hirohito Yamazaki
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  6. Kazuhiro Ikegame
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  7. Ken-ichi Matsuoka
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  8. Tomomi Toubai
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  9. Yasunori Ueda
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  10. Yoshinobu Kanda
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  11. Tatsuo Ichinohe
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  12. Yoshiko Atsuta
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  13. Takehiko Mori
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Correspondence to Yukinori Nakamura.

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Yoshinobu Kanda receives personal fees from Alexion Pharma. The remaining authors declare that they have no conflict of interest.

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Nakamura, Y., Takenaka, K., Yamazaki, H. et al. Outcome of allogeneic hematopoietic stem cell transplantation in adult patients with paroxysmal nocturnal hemoglobinuria. Int J Hematol 113, 122–127 (2021). https://doi.org/10.1007/s12185-020-02982-y

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  • DOI: https://doi.org/10.1007/s12185-020-02982-y

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