title: Long-term results of related myeloablative stem-cell transplantation to cure sickle cell disease.
abstract: Allogeneic hematopoietic stem-cell transplantation (HSCT) is the only curative treatment for sickle cell disease (SCD); nevertheless, its use has been limited by the risk of transplantation-related mortality (TRM). Between November 1988 and December 2004, 87 consecutive patients with severe SCD ranging from 2 to 22 years of age received transplants in France. Cerebral vasculopathy was the principal indication for transplantation (55 patients). All the patients received grafts from a sibling donor after a myeloablative conditioning regimen (CR). The only change in the CR during the study period was the introduction of antithymocyte globulin (ATG) in March 1992. The rejection rate was 22.6% before the use of ATG but 3% thereafter. With a median follow-up of 6 years (range, 2.0 to 17.9 years), the overall and event-free survival (EFS) rates were 93.1% and 86.1%, respectively. Graft versus host disease (GVHD) was the main cause of TRM. Importantly, cord blood transplant recipients did not develop GVHD. No new ischemic lesions were detected after engraftment, and cerebral velocities were significantly reduced. The outcome improved significantly with time: the EFS rate among the 44 patients receiving transplants after January 2000 was 95.3%. These results indicate that HLA-identical sibling HSCT after myeloablative conditioning with ATG should be considered as a standard of care for SCD children who are at high risk for stroke.
authors.affiliation: Reference Center for Sickle Cell Disease, Intercommunal Hospital, Créteil, France. francoise.bernaudin@chicreteil.fr
authors.forename: Françoise
authors.initials: F
authors.lastname: Bernaudin
authors.forename: Gérard
authors.initials: G
authors.lastname: Socie
authors.forename: Mathieu
authors.initials: M
authors.lastname: Kuentz
authors.forename: Sylvie
authors.initials: S
authors.lastname: Chevret
authors.forename: Michel
authors.initials: M
authors.lastname: Duval
authors.forename: Yves
authors.initials: Y
authors.lastname: Bertrand
authors.forename: Jean-Pierre
authors.initials: JP
authors.lastname: Vannier
authors.forename: Karima
authors.initials: K
authors.lastname: Yakouben
authors.forename: Isabelle
authors.initials: I
authors.lastname: Thuret
authors.forename: Pierre
authors.initials: P
authors.lastname: Bordigoni
authors.forename: Alain
authors.initials: A
authors.lastname: Fischer
authors.forename: Patrick
authors.initials: P
authors.lastname: Lutz
authors.forename: Jean-Louis
authors.initials: JL
authors.lastname: Stephan
authors.forename: Nathalie
authors.initials: N
authors.lastname: Dhedin
authors.forename: Emmanuel
authors.initials: E
authors.lastname: Plouvier
authors.forename: Geneviève
authors.initials: G
authors.lastname: Margueritte
authors.forename: Dominique
authors.initials: D
authors.lastname: Bories
authors.forename: Suzanne
authors.initials: S
authors.lastname: Verlhac
authors.forename: Hélène
authors.initials: H
authors.lastname: Esperou
authors.forename: Lena
authors.initials: L
authors.lastname: Coic
authors.forename: Jean-Paul
authors.initials: JP
authors.lastname: Vernant
authors.forename: Eliane
authors.initials: E
authors.lastname: Gluckman
mesh_terms: D000293:Adolescent; D000328:Adult; D000755:Anemia, Sickle Cell / Q000276:immunology / Q000473:pathology* / Q000601:surgery*; D002648:Child; D002675:Child, Preschool; D046528:Chimerism; D018572:Disease-Free Survival; D005260:Female; D006084:Graft Rejection / Q000276:immunology; D006085:Graft Survival / Q000276:immunology; D006086:Graft vs Host Disease / Q000276:immunology / Q000473:pathology; D042381:Granulocyte Precursor Cells / Q000276:immunology / Q000378:metabolism / Q000637:transplantation*; D018380:Hematopoietic Stem Cell Transplantation; D006801:Humans; D008297:Male; D013997:Time Factors; D016896:Treatment Outcome