title: Bone marrow transplantation for Fanconi anemia using fludarabine-based conditioning.
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.
authors.affiliation: Department of Pediatric Hematology-Oncology, Hadassah Hebrew University Medical Center, Jerusalem, Israel.
authors.forename: Polina
authors.initials: P
authors.lastname: Stepensky
authors.forename: Michael Y
authors.initials: MY
authors.lastname: Shapira
authors.forename: Dmitry
authors.initials: D
authors.lastname: Balashov
authors.forename: Pavel
authors.initials: P
authors.lastname: Trakhtman
authors.forename: Elena
authors.initials: E
authors.lastname: Skorobogatova
authors.forename: Lyudmila
authors.initials: L
authors.lastname: Rheingold
authors.forename: Rebecca
authors.initials: R
authors.lastname: Brooks
authors.forename: Shoshana
authors.initials: S
authors.lastname: Revel-Vilk
authors.forename: Michael
authors.initials: M
authors.lastname: Weintraub
authors.forename: Jerry
authors.initials: J
authors.lastname: Stein
authors.forename: Alexey
authors.initials: A
authors.lastname: Maschan
authors.forename: Reuven
authors.initials: R
authors.lastname: Or
authors.forename: Igor B
authors.initials: IB
authors.lastname: Resnick
mesh_terms: D000293:Adolescent; D000328:Adult; D000961:Antilymphocyte Serum / Q000627:therapeutic use; D000971:Antineoplastic Combined Chemotherapy Protocols / Q000627:therapeutic use*; D016026:Bone Marrow Transplantation / Q000379:methods*; D002066:Busulfan / Q000627:therapeutic use; D002648:Child; D002675:Child, Preschool; D003520:Cyclophosphamide / Q000627:therapeutic use; D005199:Fanconi Anemia / Q000628:therapy*; D006085:Graft Survival; D006801:Humans; D007223:Infant; D012189:Retrospective Studies; D019172:Transplantation Conditioning / Q000379:methods*; D014740:Vidarabine / Q000031:analogs & derivatives* / Q000627:therapeutic use; D055815:Young Adult
chemical_list: D000961:Antilymphocyte Serum; D003520:Cyclophosphamide; D014740:Vidarabine; D002066:Busulfan; C024352:fludarabine