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International Journal of Hematology

, Volume 110, Issue 3, pp 364–369 | Cite as

Long-term outcome and chimerism in patients with Wiskott–Aldrich syndrome treated by hematopoietic cell transplantation: a retrospective nationwide survey

  • Akihiro IguchiEmail author
  • Yuko Cho
  • Hiromasa Yabe
  • Shunichi Kato
  • Koji Kato
  • Junichi Hara
  • Katsuyoshi Koh
  • Junko Takita
  • Takashi Ishihara
  • Masami Inoue
  • Kohsuke Imai
  • Hideki Nakayama
  • Yoshiko Hashii
  • Akira Morimoto
  • Yoshiko Atsuta
  • Tomohiro Morio
  • Hereditary disorder Working Group of the Japan Society for Hematopoietic Cell Transplantation
Original Article
  • 177 Downloads

Abstract

We analyzed the outcomes of allogeneic stem cell transplantation (SCT) and risk factors for chimerism in 108 patients with Wiskott–Aldrich syndrome (WAS) who were registered with The Japan Society for Hematopoietic Cell Transplantation between January 1985 and December 2016. A preparative conditioning regimen consisting of myeloablative conditioning (MAC) was provided to 76 patients, and reduced-intensity conditioning was provided to 30 patients. Fifty-one patients received prophylaxis against graft-versus-host disease (GVHD) with cyclosporine, and 51 patients received tacrolimus (Tac). Chimerism analyses had been performed in 91 patients. Neutrophil engraftment was achieved in 91 patients (84.3%). The engraftment rate was significantly higher in patients who received Tac for GVHD prophylaxis (p = 0.028). Overall survival rate (OS) was significantly higher in patients with complete chimerism than in patients with mixed chimerism (88.2 ± 6.1% and 66.7 ± 9.9%, respectively, p = 0.003). Multivariate analysis showed that the rate of complete chimerism in patients who received MAC including cyclophosphamide (CY) at a dose of 200 mg/kg was significantly higher (p = 0.021) than that in patients who received other conditioning. Thus, MAC including CY at a dose of 200 mg/kg and Tac for GVHD prophylaxis were optimal conditions of SCT for patients with WAS under existing study.

Keywords

Stem cell transplantation (SCT) Wiskott–Aldrich syndrome (WAS) Chimerism Cyclophosphamide (CY) Tacrolimus (Tac) 

Notes

Acknowledgements

We thank Mrs. Yumiko Shinohe and Mrs. Miyuki Yanagida for their excellent assistance in the preparation of this manuscript.

Compliance with ethical standards

Conflict of interest

No potential conflicts of interest were disclosed.

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Copyright information

© Japanese Society of Hematology 2019

Authors and Affiliations

  • Akihiro Iguchi
    • 1
    Email author
  • Yuko Cho
    • 1
  • Hiromasa Yabe
    • 2
  • Shunichi Kato
    • 2
  • Koji Kato
    • 3
  • Junichi Hara
    • 4
  • Katsuyoshi Koh
    • 5
  • Junko Takita
    • 6
  • Takashi Ishihara
    • 7
  • Masami Inoue
    • 8
  • Kohsuke Imai
    • 9
  • Hideki Nakayama
    • 10
  • Yoshiko Hashii
    • 11
  • Akira Morimoto
    • 12
  • Yoshiko Atsuta
    • 13
    • 14
  • Tomohiro Morio
    • 9
  • Hereditary disorder Working Group of the Japan Society for Hematopoietic Cell Transplantation
  1. 1.Department of PediatricsHokkaido University HospitalSapporoJapan
  2. 2.Department of Cell Transplantation and Regenerative MedicineTokai University School of MedicineTokyoJapan
  3. 3.Department of Hematology and OncologyChildren’s Medical Center, Japanese Red Cross Nagoya First HospitalNagoyaJapan
  4. 4.Department of Pediatric Hematology/OncologyOsaka City General HospitalOsakaJapan
  5. 5.Department of Hematology/OncologySaitama Children’s Medical CenterSaitamaJapan
  6. 6.Department of Cell Therapy and Transplantation Medicine (Pediatrics)The University of Tokyo HospitalTokyoJapan
  7. 7.Department of PediatricsNara Medical University HospitalNaraJapan
  8. 8.Department of Hematology/OncologyOsaka Medical Center and Research Institute for Maternal and Child HealthOsakaJapan
  9. 9.Department of PediatricsMedical Hospital, Tokyo Medical and Dental UniversityTokyoJapan
  10. 10.Department of PediatricsNational Kyushu Cancer CenterFukuokaJapan
  11. 11.Department of PediatricsOsaka University Graduate School of MedicineSuitaJapan
  12. 12.Children’s Medical Center TochigiJichi Medical UniversityShimotsukeJapan
  13. 13.Japanese Data Center for Hematopoietic Cell TransplantationNagoyaJapan
  14. 14.Department of Healthcare AdministrationNagoya University Graduate School of MedicineNagoyaJapan

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