title: Hematopoietic SCT for the Black African and non-Black African variants of sickle cell anemia.
abstract: Sickle cell anemia (SCA) remains associated with high risks of morbidity and early death. Allogeneic hematopoietic SCT (HSCT) is the only curative treatment for SCA. We report our experience with transplantation in a group of patients with the non-Black African variant and the Black African variant of SCA. This study included 40 consecutive SCA patients (13 patients with the non-Black African variant and 27 with the Black African variant) who underwent BM transplantation from HLA-identical sibling donors between June 2004 and May 2013, following a myeloablative-conditioning regimen. All patients obtained sustained engraftment. One patient (non-Black African variant) became a stable mixed chimera with 25% donor cells more than 6 years after transplantation. The probabilities of survival, SCA-free survival and TRM at 5 years after transplant were 91%, 91% and 9%, respectively. All surviving patients remained free of any SCA-related events after transplantation. Our results confirm that it is possible to offer a greater than 90% chance of cure to children with SCA. HSCT should be considered the standard of care for who have an HLA-identical donor, before complications result from the sickling of RBC.
authors: 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: G
authors: G
authors: Lucarelli
authors: 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: A
authors: A
authors: Isgrò
authors: 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: P
authors: P
authors: Sodani
authors: 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: M
authors: M
authors: Marziali
authors: 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: J
authors: J
authors: Gaziev
authors: 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: K
authors: K
authors: Paciaroni
authors: 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: C
authors: C
authors: Gallucci
authors: 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: L
authors: L
authors: Cardarelli
authors: 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: M
authors: M
authors: Ribersani
authors: 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: C
authors: C
authors: Alfieri
authors: 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: G
authors: G
authors: De Angelis
authors: 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: D
authors: D
authors: Armiento
authors: 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: M
authors: M
authors: Andreani
authors: 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: M
authors: M
authors: Testi
authors: ANMI Onlus, Centro Studi Microcitemie, Rome, Italy.
authors: A
authors: A
authors: Amato
authors: Sickle Cell Foundation Nigeria, Lagos, Nigeria.
authors: O O
authors: OO
authors: Akinyanju
authors: National Hospital, Abuja, Nigeria.
authors: T T
authors: TT
authors: Wakama
mesh_terms: D000293:Adolescent; D000328:Adult; D064591:Allografts; D000755:Anemia, Sickle Cell / Q000208:ethnology / Q000401:mortality / Q000628:therapy*; D044383:Black People; D002648:Child; D002675:Child, Preschool; D018572:Disease-Free Survival; D005260:Female; D018380:Hematopoietic Stem Cell Transplantation; D006801:Humans; D007223:Infant; D012189:Retrospective Studies; D035781:Siblings; D015996:Survival Rate