title: Hematopoietic stem cell transplantation in SCD.
abstract: Hematopoietic stem cell transplantation (HSCT) is the one and only curative therapy available for patient with severe sickle cell disease (SCD). Until today, several hundreds of patients have undergone geno-identical HSCT. More than 200 patients were transplanted in France. The first indication was cerebral vasculopathy. Among both malignant and non-malignant diseases treated with HSCT, the success rate obtained in SCD patients appears as the best one. From the year 2000, more than 95% of transplanted patients survived the HSCT procedure and more than 90% are completely cured and experience a very satisfying health condition post-transplantation. However, the current standard procedure includes a myeloablative conditioning regimen for warranting engraftment. Such regime is linked to severe long-term side effects such as hypofertility. Due to the excellent obtained results, we have to think about a possible widening of indications, a decrease of conditioning intensity and toxicity, and about HSCT from alternative stem cell sources, such as mismatch family donor, unrelated volunteer donor or unrelated cord blood.
authors: Service d'hémato-immunologie pédiatrique, hôpital Robert-Debré, AP-HP, 48, boulevard Sérurier, 75935 Paris cedex 19, France. jean-hugues.dalle@rdb.aphp.fr
authors: Jean-Hugues
authors: JH
authors: Dalle
mesh_terms: D000293:Adolescent; D000755:Anemia, Sickle Cell / Q000601:surgery*; D016026:Bone Marrow Transplantation / Q000706:statistics & numerical data; D002648:Child; D002675:Child, Preschool; D036101:Cord Blood Stem Cell Transplantation / Q000706:statistics & numerical data; D059247:Fertility Preservation; D005602:France; D018380:Hematopoietic Stem Cell Transplantation / Q000379:methods / Q000706:statistics & numerical data; D006648:Histocompatibility; D006801:Humans; D007166:Immunosuppressive Agents / Q000009:adverse effects / Q000627:therapeutic use; D007223:Infant; D019520:Living Donors; D019653:Myeloablative Agonists / Q000009:adverse effects; D011788:Quality of Life; D035781:Siblings; D019172:Transplantation Conditioning / Q000009:adverse effects / Q000379:methods; D016896:Treatment Outcome
chemical_list: D007166:Immunosuppressive Agents; D019653:Myeloablative Agonists