Bone marrow transplantation for fanconi anemia using fludarabine-based conditioning

Polina Stepensky, Michael Y. Shapira, Dmitry Balashov, Pavel Trakhtman, Elena Skorobogatova, Lyudmila Rheingold, Rebecca Brooks, Shoshana Revel-Vilk, Michael Weintraub, Jerry Stein, Alexey Maschan, Reuven Or, Igor B. Resnick

Research output: Contribution to journalArticlepeer-review

Abstract

In the mid-1990s, we introduced a fludarabine (Flu)-based conditioning regimen for hematopoietic stem cell transplantation (HSCT) in patients with Fanconi anemia (FA).The aim of this study is to compare Flu-based conditioning to alternative regimens in patients with FA. Forty-one patients with FA (aged 0.5-31, median, 10.3 years) who underwent allogeneic HSCT were included in this retrospective study. Hospital records were reviewed for conditioning regimens, engraftment data, and toxicity. The median (range) follow-up was 32 (0.5-149) months. Flu-based conditioning regimens were used in 24 patients: 17 patients were treated with alternative conditioning regimens including a radiation-based regimen/cyclophosphamide and busulfan regimen. The disease-free survival (DFS) after Flu-based regimens is 83% (20/24) versus 35% (6/17) for the alternative regimens (P = .002). Toxicity was significantly lower in patients who received Flu-based conditioning (modified Bearman toxicity score [. P = .001]). Seven patients received transplants from matched unrelated donors without irradiation (5 of whom are currently alive and well). All patients who survived are disease free and in good clinical condition. We conclude that a combination of fludarabine with antithymocyte globulin (ATG) and low-dose cyclophosphamide (Cy) and/or busulfan (Bu) is safe, demonstrates low rejection rates, and is well tolerated by FA patients.

Original languageEnglish
Pages (from-to)1282-1288
Number of pages7
JournalBiology of Blood and Marrow Transplantation
Volume17
Issue number9
DOIs
StatePublished - Sep 2011
Externally publishedYes

Keywords

  • Allogeneic hematopoietic stem cell transplantation
  • Fanconi anemia
  • Fludarabine

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