TY - JOUR T1 - Bone marrow transplant in patients with sickle cell anaemia. Experience in one centre JO - Anales de Pediatría (English Edition) T2 - AU - García Morin,Marina AU - Cela,Elena AU - Garrido,Carmen AU - Bardón Cancho,Eduardo AU - Aguado del Hoyo,Alejandra AU - Pascual,Cristina AU - Pérez-Corral,Ana AU - Beléndez,Cristina SN - 23412879 M3 - 10.1016/j.anpede.2016.03.012 DO - 10.1016/j.anpede.2016.03.012 UR - https://analesdepediatria.org/en-bone-marrow-transplant-in-patients-articulo-S234128791730008X AB - IntroductionSickle 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. Material and methodA 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). ResultsAllo-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. ConclusionsAllo-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. ER -