title: Outcome of aplastic anaemia in children. A study by the severe aplastic anaemia and paediatric disease working parties of the European group blood and bone marrow transplant.
abstract: This study analysed the outcome of 563 Aplastic Anaemia (AA) children aged 0-12 years reported to the Severe Aplastic Anaemia Working Party database of the European Society for Blood and Marrow Transplantation, according to treatment received. Overall survival (OS) after upfront human leucocyte antigen-matched family donor (MFD) haematopoietic stem cell transplantation (HSCT) or immunosuppressive treatment (IST) was 91% vs. 87% (P 0·18). Event-free survival (EFS) after upfront MFD HSCT or IST was 87% vs. 33% (P 0·001). Ninety-one of 167 patients (55%) failed front-line IST and underwent rescue HSCT. The OS of this rescue group was 83% compared with 91% for upfront MFD HSCT patients and 97% for those who did not fail IST up-front (P 0·017). Rejection was 2% for MFD HSCT and HSCT post-IST failure (P 0·73). Acute graft-versus-host disease (GVHD) grade II-IV was 8% in MFD graft vs. 25% for HSCT post-IST failure (P < 0·0001). Chronic GVHD was 6% in MFD HSCT vs. 20% in HSCT post-IST failure (P < 0·0001). MFD HSCT is an excellent therapy for children with AA. IST has a high failure rate, but remains a reasonable first-line choice if MFD HSCT is not available because high OS enables access to HSCT, which is a very good rescue option.
authors: Clinical and Experimental Haematology Unit, G Gaslini Childrens' Hospital, Genova, Italy.
authors: Carlo
authors: C
authors: Dufour
authors: Marta
authors: M
authors: Pillon
authors: Gerard
authors: G
authors: Sociè
authors: Alicia
authors: A
authors: Rovò
authors: Elisa
authors: E
authors: Carraro
authors: Andrea
authors: A
authors: Bacigalupo
authors: Rosi
authors: R
authors: Oneto
authors: Jakob
authors: J
authors: Passweg
authors: Antonio
authors: A
authors: Risitano
authors: Andrè
authors: A
authors: Tichelli
authors: Regis
authors: R
authors: Peffault de Latour
authors: Hubert
authors: H
authors: Schrezenmeier
authors: Britta
authors: B
authors: Hocshmann
authors: Christina
authors: C
authors: Peters
authors: Austin
authors: A
authors: Kulasekararaj
authors: Anja
authors: A
authors: Van Biezen
authors: Sujith
authors: S
authors: Samarasinghe
authors: Ayad Ahmed
authors: AA
authors: Hussein
authors: Mouhab
authors: M
authors: Ayas
authors: Mahmoud
authors: M
authors: Aljurf
authors: Judith
authors: J
authors: Marsh
mesh_terms: D064591:Allografts; D000741:Anemia, Aplastic / Q000401:mortality* / Q000628:therapy*; D002648:Child; D002675:Child, Preschool; D016208:Databases, Factual; D018572:Disease-Free Survival; D005060:Europe / Q000453:epidemiology; D005260:Female; D006084:Graft Rejection / Q000401:mortality* / Q000628:therapy; D006086:Graft vs Host Disease / Q000401:mortality* / Q000628:therapy; D018380:Hematopoietic Stem Cell Transplantation; D006801:Humans; D007165:Immunosuppression Therapy; D007223:Infant; D007231:Infant, Newborn; D008297:Male; D015996:Survival Rate
keywords: aplastic anaemia; children; haematopoietic stem cell transplantation; immunosuppression