[Hematopoietic stem cell transplantation for severe sickle cell disease].
Hematopoietic stem cell transplantation is the sole curative therapy for sickle cell anemia (SCA). This treatment is restricted to severe forms of disease. Lack of HLA-identical sibling donor is the major limiting factor for delivering this therapy. Conditioning regimen should be myeloablative. Post transplantation immunosuppressive medication is necessary for both graft tolerance and graft-versus-host disease (GvHD) control. For the majority of patients, bone marrow is source of stem cells. Alternative sources including related cord blood stem cells are under evaluation and promising. Outcome for 250 grafted patients worldwide is excellent. More than 85 % survive free of SCA and have a good quality of life although GvHD remains the main complication.
Klein
A
A
Unité Hemato-Oncologie, HUDERF.
Brachet
C
C
Azzi
N
N
Ferster
A
A
D000755:Anemia, Sickle Cell / Q000601:surgery*; D018380:Hematopoietic Stem Cell Transplantation; D006801:Humans; D012720:Severity of Illness Index