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

Andrea Bacigalupo, Carlo Dufour, Marta Pillon, Gerard Sociè, Alicia Rovò, Elisa Carraro, Rosi Oneto, Jakob Passweg, Antonio Risitano, Andrè Tichelli, Regis Peffault De Latour, Hubert Schrezenmeier, Britta Hocshmann, Christina Peters, Austin Kulasekararaj, Anja Van Biezen, Sujith Samarasinghe, Ayad Ahmed Hussein, Mouhab Ayas, Mahmoud AljurfJudith Marsh

Risultato della ricerca: Contributo in rivistaArticolo in rivista

62 Citazioni (Scopus)

Abstract

Summary: 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.
Lingua originaleEnglish
pagine (da-a)565-573
Numero di pagine9
RivistaBritish Journal of Haematology
Volume169
DOI
Stato di pubblicazionePubblicato - 2015

Keywords

  • Allografts
  • Anemia, Aplastic
  • Aplastic anaemia
  • Child
  • Child, Preschool
  • Children
  • Databases, Factual
  • Disease-Free Survival
  • Europe
  • Female
  • Graft Rejection
  • Graft vs Host Disease
  • Haematopoietic stem cell transplantation
  • Hematology
  • Hematopoietic Stem Cell Transplantation
  • Humans
  • Immunosuppression
  • Infant
  • Infant, Newborn
  • Male
  • Survival Rate

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