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 Kojima

Research output: Contribution to journalArticle

17 Citations (Scopus)

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 antithymocyte globulin (ATG) either from a related donor (n = 4) or an unrelated donor (n = 4). Acute graft-versushost 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.

Original languageEnglish
Pages (from-to)675-679
Number of pages5
JournalInternational journal of hematology
Volume95
Issue number6
DOIs
Publication statusPublished - Jun 1 2012
Externally publishedYes

Keywords

  • Fanconi anemia
  • Fludarabine
  • Hematopoietic stem cell transplantation

ASJC Scopus subject areas

  • Hematology

Fingerprint Dive into the research topics of 'Excellent outcome of allogeneic bone marrow transplantation for Fanconi anemia using fludarabine-based reduced-intensity conditioning regimen'. Together they form a unique fingerprint.

  • Cite this

    Shimada, A., Takahashi, Y., Muramatsu, H., Hama, A., Ismael, O., Narita, A., Sakaguchi, H., Doisaki, S., Nishio, N., Tanaka, M., Yoshida, N., Matsumoto, K., Kato, K., Watanabe, N., & Kojima, S. (2012). Excellent outcome of allogeneic bone marrow transplantation for Fanconi anemia using fludarabine-based reduced-intensity conditioning regimen. International journal of hematology, 95(6), 675-679. https://doi.org/10.1007/s12185-012-1079-9