title: Hydroxyurea treatment for sickle cell disease: impact on haematopoietic stem cell transplantation's outcome.
abstract: Since 1988, 24 children have undergone haematopoietic stem cell transplantation (HSCT) for severe sickle cell disease (SCD) in our unit, 13 being grafted after having been exposed to hydroxyurea (HU) to control SCD-related complications. Different pre-transplant conditioning regimens were given over time: Bu14/Cy200 in six patients (group 1), Bu16/Cy200/antithymocyte globulin (ATG) in five (group 2) and Bu16/Cy200/ATG with HU prior to HSCT in 13 (group 3). The aim of this study is to compare the outcome after HSCT of these groups of patients, which differ according to pre-transplant drug exposure. Overall, 20 of the 24 transplanted children had stable engraftment and have remained free of SCD-related symptoms after HSCT; 19 of them are currently alive and cured of SCD. In group 1 (HU-, ATG-), we observed one unexplainable late death, one absent engraftment, one late rejection and one mixed stable chimerism. In group 2 (HU-, ATG+), we observed the absence of engraftment in two patients and one early rejection. In group 3 (HU+, ATG+), we observed no cases of either absent engraftment, mixed stable chimerism or late rejection. In our experience, pre-transplant treatment with HU seems to be associated with a lower incidence of rejection/absent engraftment in severe SCD patients. These results need to be confirmed with a larger number of patients.
authors.affiliation: 1Haemato-Oncology Unit, Hôpital Universitaire des Enfants-ULB, Brussels, Belgium.
authors.forename: C
authors.initials: C
authors.lastname: Brachet
authors.forename: N
authors.initials: N
authors.lastname: Azzi
authors.forename: A
authors.initials: A
authors.lastname: Demulder
authors.forename: C
authors.initials: C
authors.lastname: Devalck
authors.forename: A
authors.initials: A
authors.lastname: Gourdin
authors.forename: B
authors.initials: B
authors.lastname: Gulbis
authors.forename: A
authors.initials: A
authors.lastname: Klein
authors.forename: P Q
authors.initials: PQ
authors.lastname: Le
authors.forename: M
authors.initials: M
authors.lastname: Loop
authors.forename: E
authors.initials: E
authors.lastname: Sariban
authors.forename: A
authors.initials: A
authors.lastname: Ferster
mesh_terms: D000755:Anemia, Sickle Cell / Q000188:drug therapy* / Q000628:therapy*; D000986:Antisickling Agents / Q000627:therapeutic use*; D002648:Child; D002675:Child, Preschool; D005260:Female; D006085:Graft Survival; D006086:Graft vs Host Disease / Q000209:etiology; D018380:Hematopoietic Stem Cell Transplantation / Q000009:adverse effects; D006801:Humans; D006918:Hydroxyurea / Q000627:therapeutic use*; D008297:Male; D012640:Seizures / Q000209:etiology; D019172:Transplantation Conditioning
chemical_list: D000986:Antisickling Agents; D006918:Hydroxyurea