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.

Authors:
Carlo Dufour
Carlo Dufour
G. Gaslini Children's Hospital
Italy
Marta Pillon
Marta Pillon
University of Padova
Italy
Gerard Socie
Gerard Socie
Service d'Hématologie Greffe
Alicia Rovo
Alicia Rovo
Basel University Hospital
Switzerland
Elisa Carraro
Elisa Carraro
University of Padova
Italy
Andrea Bacigalupo
Andrea Bacigalupo
Universita' Cattolica del Sacro Cuore
Milano | Italy
Rosi Oneto
Rosi Oneto
University of Pavia
Italy
Jakob Passweg
Jakob Passweg
University Hospital Basel
Basel | Switzerland

Br J Haematol 2015 May 14;169(4):565-73. Epub 2015 Feb 14.

Clinical and Experimental Haematology Unit, G Gaslini Childrens' Hospital, Genova, Italy.

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.

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http://dx.doi.org/10.1111/bjh.13297DOI Listing
May 2015
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References

(Supplied by CrossRef)
Upfront matched and mismatched unrelated donor transplantation in paediatric idiopathic severe aplastic anaemia: a United Kingdom multicentre retrospective experience
Bhatnagar et al.
Bone Marrow Transplantation 2014
Front-line immunosuppressive treatment of acquired aplastic anemia
Dufour et al.
Bone Marrow Transplantation 2013

Kalbfleisch et al.
1980

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