title: Impact of conditioning on outcome of hematopoietic stem cell transplantation for wiskott-Aldrich syndrome.
abstract: Hematopoietic stem cell transplantation (HSCT) is the treatment of choice for Wiskott-Aldrich syndrome (WAS). The aim of this retrospective study is to report the effect of the conditioning regimen and donor source on disease-free survival (DFS) in children undergoing HSCT for WAS. Fourteen children who underwent HSCT at 4 Israeli centers from 1996 to 2011 were included in this study. Five children were transplanted from matched related donors (4/5 siblings, 1/5 fully matched uncle) and other donors were used in 9 children. Six patients were conditioned with full dose busulfan/cyclophosphamide (Bu/Cy) whereas 8 patients were conditioned with other regimens. Thirteen of 14 patients (92.8%) are alive with a median follow-up of 3.4 years (range, 5 mo to 12.5 y). Nine patients (64.3%) survive with complete clinical, immunologic, and hematologic recovery. Children conditioned with full dose Bu/Cy had a 100% DFS, compared with children conditioned with other regimens, 25%±19% (P=0.022). Donor source was not associated with DFS. Graft failure was related to the use of conditioning regimens other than full dose Bu/Cy and not to the donor source. Further studies are required to determine the best conditioning regimen and optimal donor source for children with WAS.
authors: Department of Pediatric Hematology-Oncology, Hadassah Hebrew University Medical Center, Jerusalem, Israel. polina@hadassah.org.il
authors: Polina
authors: P
authors: Stepensky
authors: Aviva
authors: A
authors: Krauss
authors: Gal
authors: G
authors: Goldstein
authors: Irena
authors: I
authors: Zaidman
authors: Ronit
authors: R
authors: Elhasid
authors: Bela
authors: B
authors: Bielorai
authors: Raz
authors: R
authors: Somech
authors: Reuven
authors: R
authors: Or
authors: Jerry
authors: J
authors: Stein
authors: Shoshana
authors: S
authors: Revel-Vilk
authors: Michael
authors: M
authors: Weintraub
mesh_terms: D002648:Child; D002675:Child, Preschool; D018572:Disease-Free Survival; D006085:Graft Survival; D018380:Hematopoietic Stem Cell Transplantation / Q000379:methods*; D006801:Humans; D007223:Infant; D053208:Kaplan-Meier Estimate; D012189:Retrospective Studies; D019172:Transplantation Conditioning / Q000379:methods*; D014184:Transplantation, Homologous / Q000401:mortality; D014923:Wiskott-Aldrich Syndrome / Q000401:mortality / Q000601:surgery*