مرکز منطقه ای اطلاع رساني علوم و فناوري - Allogeneic hematopoietic stem cell transplantation in children with acquired severe aplastic anemia: Analysis of 15 patients

Abstract :

Aim: The data of allogeneic hematopoietic stem cell transplantation (HSCT) in children with acquired aplastic anemia between February 2006 and July 2010 were analyzed retrospectively.Materials and Methods: Fifteen patients with a median age of 12 years underwent transplantation including 13 matched sibling donors (MSD), 1 matched related donor (MRD) and 1 matched unrelated donor (MUD). One patient, who had been transplanted from an unrelated donor, received a second transplantation successfully after a primary graft failure from the same donor. The median time to transplantation from diagnosis was 6 months (range: 3-29 months). Conditioning regimens were ATG (40 mg/kg) and “cyclophosphamide” (200 mg/kg) in 12 patients and “fludarabine” (150 mg/m2) was added in 3 patients who received many transfusions. The stem cell source was peripheral blood in 11 (69%) transplantations. Cyclosporin and short term methotrexate were given for graft-versus-host disease (GVHD) prophylaxis in all patients.Results: The median duration of time to neutrophil and platelet engraftment was 17 and 18 days, respectively. Grade II acute GVHD developed in 1(7%) patient, and limited chronic GVHD developed in another patient (7%). Lymphoprolipherative disease appeared in a patient who received transplantation from a MUD. The overall 5-year survival rate was 100% and EFS was 94%. All recipients are alive without any graft failure in a median 3.6 years (range: 1.4-5.4 years).Conclusion: In our experience, HSCT from a MSD remains the first choice of treatment for pediatric patients with severe aplastic anemia. For patients without a MSD, transplantation from an unrelated or alternative related donor can also result in long-term survival.