title: [Bone marrow transplant in patients with sickle cell anaemia. Experience in one centre].
abstract: BACKGROUND
  Sickle cell disease (SCD), despite the improvement in the medical management, is still associated with severe morbidity and decreased survival. Allogenic hematopoietic stem cell transplantation (Allo-HSCT) currently provides the only curative therapy. A report is presented on our experience in children with SCD, who underwent Allo-HSCT in a single centre.
  
  
  METHODS
  A single centre descriptive study was conducted on patients with SCD who underwent a bone marrow transplant from an HLA-identical sibling donor between January 2010 and December 2014. Epidemiological, clinical and analytical parameters were collected with a follow-up to December 2015. Data are presented as frequencies, percentages, and medians (range).
  
  
  RESULTS
  Allo-HCST was performed in 11 patients (8 males) with a median age of 7 years (2-13), all of them with comorbidity prior to the HCST. A stable graft was achieved in 10 out of 11 patients, 9 of them with complete donor chimerism, and one patient with stable mixed chimerism after 1 year of allo-HSCT. One patient has secondary graft failure with re-appearance of symptoms associated with SCD on day 180. Complications of Allo-HSCT are: arterial hypertension 7/11, acute renal failure 3/11, CMV reactivation 9/11, neurological complications 4/11 (subarachnoid haemorrhage, seizure), and acute graft versus host disease (aGVHD) of the skin 6/11, one of whom developed grade iv intestinal aGVHD, causing his death (day 51). None of the patients developed chronic GVHD. The overall survival and event-free survival was 90.9% and 81.9%, respectively, with a median follow-up of 3.1 (1-5.7) years.
  
  
  CONCLUSIONS
  Allo-HSCT, the only curative therapy, remains associated with morbidity. There was a transplant related mortality in our study, consistent with multicentre studies (1/11), and with aGVHD being the main cause. Other problems still include graft failure (1/11), and neurological complications (4/11), although the permanent sequelae are mild.
authors.affiliation: Hematología-Oncología Pediátricas, Hospital General Universitario Gregorio Marañón, Madrid, España. Electronic address: m.garciamorin@gmail.com.
authors.forename: Marina
authors.initials: M
authors.lastname: García Morin
authors.affiliation: Hematología-Oncología Pediátricas, Hospital General Universitario Gregorio Marañón, Madrid, España.
authors.forename: Elena
authors.initials: E
authors.lastname: Cela
authors.affiliation: Hematología-Oncología Pediátricas, Hospital General Universitario Gregorio Marañón, Madrid, España.
authors.forename: Carmen
authors.initials: C
authors.lastname: Garrido
authors.affiliation: Hematología-Oncología Pediátricas, Hospital General Universitario Gregorio Marañón, Madrid, España.
authors.forename: Eduardo
authors.initials: E
authors.lastname: Bardón Cancho
authors.affiliation: Radiología Infantil, Hospital General Universitario Gregorio Marañón, Madrid, España.
authors.forename: Alejandra
authors.initials: A
authors.lastname: Aguado Del Hoyo
authors.affiliation: Servicios de Hematología y Hemoterapia, Hospital General Universitario Gregorio Marañón, Madrid, España.
authors.forename: Cristina
authors.initials: C
authors.lastname: Pascual
authors.affiliation: Servicios de Hematología y Hemoterapia, Hospital General Universitario Gregorio Marañón, Madrid, España.
authors.forename: Ana
authors.initials: A
authors.lastname: Pérez-Corral
authors.affiliation: Hematología-Oncología Pediátricas, Hospital General Universitario Gregorio Marañón, Madrid, España.
authors.forename: Cristina
authors.initials: C
authors.lastname: Beléndez
mesh_terms: D000293:Adolescent; D000755:Anemia, Sickle Cell / Q000601:surgery*; D016026:Bone Marrow Transplantation; D002648:Child; D002675:Child, Preschool; D005260:Female; D006801:Humans; D008297:Male; D012189:Retrospective Studies
keywords: Allogeneic hematopoietic stem cell transplantation from an HLA-identical sibling; Alo-trasplante de médula ósea de hermano HLA idéntico; Anemia de células falciformes; Sickle cell disease