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International Journal of Hematology

, Volume 95, Issue 6, pp 675–679 | Cite as

Excellent outcome of allogeneic bone marrow transplantation for Fanconi anemia using fludarabine-based reduced-intensity conditioning regimen

  • Akira Shimada
  • Yoshiyuki Takahashi
  • Hideki Muramatsu
  • Asahito Hama
  • Olfat Ismael
  • Atsushi Narita
  • Hiroshi Sakaguchi
  • Sayoko Doisaki
  • Nobuhiro Nishio
  • Makito Tanaka
  • Nao Yoshida
  • Kimikazu Matsumoto
  • Koji Kato
  • Nobuhiro Watanabe
  • Seiji KojimaEmail author
Original Article

Abstract

Fanconi anemia (FA) is a disorder characterized by developmental anomalies, bone marrow failure and a predisposition to malignancy. It has recently been shown that hematopoietic stem cell transplantation using fludarabine (FLU)-based reduced-intensity conditioning is an efficient and quite safe therapeutic modality. We retrospectively analyzed the outcome of bone marrow transplantation (BMT) in eight patients with FA performed in two institutes between 2001 and 2011. There were seven females and one male with a median age at diagnosis = 4.5 years (range 2–12 years). The constitutional characteristics associated with FA, such as developmental anomalies, short stature and skin pigmentation, were absent in three of the patients. One patient showed myelodysplastic features at the time of BMT. All patients received BMT using FLU, cyclophosphamide (CY) and rabbit anti-thymocyte globulin (ATG) either from a related donor (n = 4) or an unrelated donor (n = 4). Acute graft-versus-host disease (GVHD) of grade I developed in one patient, while chronic GVHD was not observed in any patient. All patients are alive and achieved hematopoietic recovery at a median follow-up of 72 months (range 4–117 months). BMT using FLU/low-dose CY/ATG -based regimens regardless to the donor is a beneficial therapeutic approach for FA patients.

Keywords

Fanconi anemia Hematopoietic stem cell transplantation Fludarabine 

Notes

Acknowledgments

This work was supported in part by grant for scientific research by the Ministry of Education, Culture, Sports, Science and Technology, Japan.

Conflict of interest

The authors declare no conflict of interest.

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Copyright information

© The Japanese Society of Hematology 2012

Authors and Affiliations

  • Akira Shimada
    • 1
  • Yoshiyuki Takahashi
    • 1
  • Hideki Muramatsu
    • 1
  • Asahito Hama
    • 1
  • Olfat Ismael
    • 1
  • Atsushi Narita
    • 1
  • Hiroshi Sakaguchi
    • 1
  • Sayoko Doisaki
    • 1
  • Nobuhiro Nishio
    • 1
  • Makito Tanaka
    • 1
  • Nao Yoshida
    • 2
  • Kimikazu Matsumoto
    • 2
  • Koji Kato
    • 2
  • Nobuhiro Watanabe
    • 3
  • Seiji Kojima
    • 1
    Email author
  1. 1.Department of PediatricsNagoya University Graduate School of MedicineNagoyaJapan
  2. 2.Department of Pediatric Hematology/OncologyNagoya First Red Cross HospitalNagoyaJapan
  3. 3.Department of PediatricsCyukyo HospitalNagoyaJapan

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