title: Haematopoietic stem cell transplantation in Nigerian sickle cell anaemia children patients.
abstract: BACKGROUND
  Sickle cell anaemia (SCA) remains associated with high risks of morbidity and early death. Children with SCA are at high risk for ischaemic stroke and transient ischaemic attacks, secondary to intracranial arteriopathy involving carotid and cerebral arteries. Allogeneic haematopoietic stem cell transplantation (HSCT) is the only curative treatment for SCA. We report our experience with transplantation in a group of patients with the Black African variant of SCA.
  
  
  METHODS
  This study included 31 consecutive SCA patients who underwent bone marrow transplantation from human leukocyte antigen (HLA)-identical sibling donors between 2010 and 2014 following a myeloablative-conditioning regimen.
  
  
  RESULTS
  The median patient age was 10 years (range 2-17 years). Before transplantation, 14 patients had recurrent, painful, vaso-occlusive crisis; ten patients had recurrent painful crisis in association with acute chest syndrome; three patients experienced ischaemic stroke and recurrent vaso-occlusive crisis; two patients experienced ischaemic stroke; one patient exhibited leukocytosis; and one patient exhibited priapism. Of the 31 patients, 28 survived without sickle cell disease, with Lansky/Karnofsky scores of 100. All surviving patients remained free of any SCA-related events after transplantation.
  
  
  CONCLUSIONS
  The protocols used for the preparation to the transplant in thalassaemia are very effective also in the other severe haemoglobinopathy as in the sickle cell anaemia with 90% disease free survival. Today, if a SCA patient has a HLA identical family member, the cellular gene therapy through the transplantation of the allogeneic haemopoietic cell should be performed. Tomorrow, hopefully, the autologous genetically corrected stem cell will break down the wall of the immunological incompatibility.
authors.affiliation: International Center for Transplantation in Thalassemia and Sickle Cell Anemia, Mediterranean Institute of Hematology, Policlinic 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 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 Tor Vergata, Rome, Italy.
authors.forename: Javid
authors.initials: J
authors.lastname: Gaziev
authors.affiliation: International Center for Transplantation in Thalassemia and Sickle Cell Anemia, Mediterranean Institute of Hematology, Policlinic 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 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 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 Tor Vergata, Rome, Italy.
authors.forename: Gioia De
authors.initials: GD
authors.lastname: Angelis
authors.affiliation: International Center for Transplantation in Thalassemia and Sickle Cell Anemia, Mediterranean Institute of Hematology, Policlinic 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 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 Tor Vergata, Rome, Italy.
authors.forename: Andrea
authors.initials: A
authors.lastname: Roveda
authors.affiliation: Sickle Cell Foundation Nigeria, Lagos, Nigeria.
authors.forename: Olufemi O
authors.initials: OO
authors.lastname: Akinyanju
authors.affiliation: National Hospital, Abuja, Nigeria.
authors.forename: T Thompson
authors.initials: TT
authors.lastname: Wakama
authors.affiliation: Department of Haematology and Blood Transfusion, College of Medicine, Lagos University Teaching Hospital, Lagos, Nigeria.
authors.forename: Festus Olusola
authors.initials: FO
authors.lastname: Olowoselu
authors.affiliation: Department of Haematology and Blood Transfusion, College of Medicine, Lagos University Teaching Hospital, Lagos, Nigeria.
authors.forename: Adewumi
authors.initials: A
authors.lastname: Adediran
authors.affiliation: International Center for Transplantation in Thalassemia and Sickle Cell Anemia, Mediterranean Institute of Hematology, Policlinic Tor Vergata, Rome, Italy.
authors.forename: Guido
authors.initials: G
authors.lastname: Lucarelli
keywords: Bone marrow transplantation; children; sickle cell anaemia