title: Reduction of intramedullary apoptosis after stem cell transplantation in black african variant of pediatric sickle cell anemia.
abstract: OBJECTIVE
  Allogeneic hematopoietic stem cell transplantation (HSCT) is the only curative treatment for sickle cell anemia (SCA). We report our experience with transplantation in children with the Black African variant of SCA and the effects of transplant on erythroid compartment in bone marrow (BM).
  
  
  METHODS
  Twenty-seven consecutive patients who underwent BM transplantation from HLA-identical donors following a myeloablative conditioning regimen were included. Using both CD71 and FSC parameters, we obtained three erythroid populations: EryA-C. Ery A (CD71(high) FSC(high)) are basophilic; Ery B (CD71(high) FSC(low)) are late basophilic and polychromatic; and Ery C (CD71(low) FSC(low)) are orthochromatic erythroblasts and reticulocytes. To analyze the effect of transplantation on intramedullary apoptosis, we studied Fas (CD95+) and caspase-3 expression in erythroblast subpopulations.
  
  
  RESULTS
  All patients experienced sustained engraftment, and all surviving patients remained free of SCA-related events after transplantation. The erythroid population showed expansion in the BM at baseline. After transplant, levels decreased, especially of Ery C, in parallel to reduced Fas expression and an initial caspase 3 increase in erythroid population, similar to reported later steps of "normal" erythroid maturation.
  
  
  CONCLUSIONS
  The results suggest a good chance of cure for children with SCA, with an excellent survival rate. We also observed "normalization" of erythroid populations in parallel with a decreased intramedullary apoptosis rate, suggesting normal erythroid maturation in ex-SCA patients after HSCT.
authors.affiliation: International Center for Transplantation in Thalassemia and Sickle Cell Anemia, Mediterranean Institute of Hematology, Policlinic of the University of Roma Tor Vergata., Rome, Italy.
authors.forename: Antonella
authors.initials: A
authors.lastname: Isgrò
authors.affiliation: International Center for Transplantation in Thalassemia and Sickle Cell Anemia, Mediterranean Institute of Hematology, Policlinic of the University of Roma Tor Vergata., Rome, Italy.
authors.forename: Pietro
authors.initials: P
authors.lastname: Sodani
authors.affiliation: International Center for Transplantation in Thalassemia and Sickle Cell Anemia, Mediterranean Institute of Hematology, Policlinic of the University of Roma Tor Vergata., Rome, Italy.
authors.forename: Marco
authors.initials: M
authors.lastname: Marziali
authors.affiliation: International Center for Transplantation in Thalassemia and Sickle Cell Anemia, Mediterranean Institute of Hematology, Policlinic of the University of Roma Tor Vergata., Rome, Italy.
authors.forename: Javid
authors.initials: J
authors.lastname: Gaziev
authors.affiliation: Laboratory of Oncohematology, Department of Laboratory Medicine, Policlinic of the University of Roma Tor Vergata, Rome, Italy.
authors.forename: Daniela
authors.initials: D
authors.lastname: Fraboni
authors.affiliation: International Center for Transplantation in Thalassemia and Sickle Cell Anemia, Mediterranean Institute of Hematology, Policlinic of the University of Roma Tor Vergata., Rome, Italy.
authors.forename: Katia
authors.initials: K
authors.lastname: Paciaroni
authors.affiliation: International Center for Transplantation in Thalassemia and Sickle Cell Anemia, Mediterranean Institute of Hematology, Policlinic of the University of Roma Tor Vergata., Rome, Italy.
authors.forename: Cristiano
authors.initials: C
authors.lastname: Gallucci
authors.affiliation: International Center for Transplantation in Thalassemia and Sickle Cell Anemia, Mediterranean Institute of Hematology, Policlinic of the University of Roma Tor Vergata., Rome, Italy.
authors.forename: Gioia
authors.initials: G
authors.lastname: De Angelis
authors.affiliation: International Center for Transplantation in Thalassemia and Sickle Cell Anemia, Mediterranean Institute of Hematology, Policlinic of the University of Roma Tor Vergata., Rome, Italy.
authors.forename: Cecilia
authors.initials: C
authors.lastname: Alfieri
authors.affiliation: International Center for Transplantation in Thalassemia and Sickle Cell Anemia, Mediterranean Institute of Hematology, Policlinic of the University of Roma Tor Vergata., Rome, Italy.
authors.forename: Michela
authors.initials: M
authors.lastname: Ribersani
authors.affiliation: International Center for Transplantation in Thalassemia and Sickle Cell Anemia, Mediterranean Institute of Hematology, Policlinic of the University of Roma Tor Vergata., Rome, Italy.
authors.forename: Daniele
authors.initials: D
authors.lastname: Armiento
authors.affiliation: International Center for Transplantation in Thalassemia and Sickle Cell Anemia, Mediterranean Institute of Hematology, Policlinic of the University of Roma Tor Vergata., Rome, Italy.
authors.forename: Andrea
authors.initials: A
authors.lastname: Roveda
authors.affiliation: International Center for Transplantation in Thalassemia and Sickle Cell Anemia, Mediterranean Institute of Hematology, Policlinic of the University of Roma Tor Vergata., Rome, Italy.
authors.forename: Marco
authors.initials: M
authors.lastname: Andreani
authors.affiliation: International Center for Transplantation in Thalassemia and Sickle Cell Anemia, Mediterranean Institute of Hematology, Policlinic of the University of Roma Tor Vergata., Rome, Italy.
authors.forename: Manuela
authors.initials: M
authors.lastname: Testi
authors.affiliation: International Center for Transplantation in Thalassemia and Sickle Cell Anemia, Mediterranean Institute of Hematology, Policlinic of the University of Roma Tor Vergata., Rome, Italy.
authors.forename: Guido
authors.initials: G
authors.lastname: Lucarelli