title: Excellent outcome of haploidentical hematopoietic stem cell transplantation in children and adolescents with acquired severe aplastic anemia.
abstract: We evaluated the outcome of children and adolescents with acquired severe aplastic anemia (SAA) who received haploidentical hematopoietic stem cell transplantation (HHCT) with in vitro T cell-depleted peripheral blood stem cells. Twelve patients with acquired SAA received a total of 15 HHCTs with in vitro CD3-depleted grafts between July 2009 and July 2012. Among the 12 patients, 11 achieved neutrophil engraftment at a median of 10 days (range, 9 to 13 days) after HHCT. One patient failed to achieve primary engraftment, and two experienced graft rejection soon after engraftment. All three patients who experienced early graft failure received a second HHCT and achieved sustained engraftment. Thus, the final engraftment rate was 100%. Acute graft-versus-host disease was assessed in 9 patients, excluding the 3 patients with early graft failure. Three of these patients developed acute graft-versus-host disease (two ≥ grade II and one with grade III). All 12 patients survived and were transfusion-independent at a median follow-up of 14.3 months (range, 4.1 to 40.7 months). Hematopoietic stem cell transplantation from haploidentical family donors with in vitro CD3 T cell depletion is a reasonable therapeutic option for children and adolescents with acquired SAA. Our future trial with a uniform protocol will help to solve the problems associated with HHCT and provide a valuable platform for the further development of HHCT as a therapy for SAA.
authors: Division of Pediatric Hematology/Oncology, Department of Pediatrics, University of Ulsan College of Medicine, Asan Medical Center Children's Hospital, Seoul, Korea. hojim@amc.seoul.kr
authors: Ho Joon
authors: HJ
authors: Im
authors: Kyung Nam
authors: KN
authors: Koh
authors: Eun Seok
authors: ES
authors: Choi
authors: Seongsoo
authors: S
authors: Jang
authors: Seog Woon
authors: SW
authors: Kwon
authors: Chan-Jeoung
authors: CJ
authors: Park
authors: Hyun-Sook
authors: HS
authors: Chi
authors: Jong Jin
authors: JJ
authors: Seo
mesh_terms: D000293:Adolescent; D000741:Anemia, Aplastic / Q000097:blood / Q000601:surgery*; D002648:Child; D002675:Child, Preschool; D005260:Female; D016179:Granulocyte Colony-Stimulating Factor / Q000008:administration & dosage; D006238:Haploidy; D018380:Hematopoietic Stem Cell Transplantation / Q000379:methods*; D006801:Humans; D008297:Male; D013601:T-Lymphocytes / Q000276:immunology; D016896:Treatment Outcome; D055815:Young Adult
chemical_list: D016179:Granulocyte Colony-Stimulating Factor