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.

Dufour, Carlo; Pillon, Marta; Sociè, Gerard; Rovó, Alicia; Carraro, Elisa; Bacigalupo, Andrea; Oneto, Rosi; Passweg, Jakob; Risitano, Antonio; Tichelli, Andrè; Peffault de Latour, Regis; Schrezenmeier, Hubert; Hocshmann, Britta; Peters, Christina; Kulasekararaj, Austin; Van Biezen, Anja; Samarasinghe, Sujith; Hussein, Ayad Ahmed; Ayas, Mouhab; Aljurf, Mahmoud; ... (2015). 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. British journal of haematology, 169(4), pp. 565-573. Wiley-Blackwell 10.1111/bjh.13297

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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.

Item Type:

Journal Article (Original Article)

Division/Institute:

04 Faculty of Medicine > Department of Haematology, Oncology, Infectious Diseases, Laboratory Medicine and Hospital Pharmacy (DOLS) > Clinic of Haematology and Central Haematological Laboratory

UniBE Contributor:

Rovó, Alicia

Subjects:

600 Technology > 610 Medicine & health

ISSN:

0007-1048

Publisher:

Wiley-Blackwell

Language:

English

Submitter:

Verena Zwahlen

Date Deposited:

09 Apr 2015 09:50

Last Modified:

10 Sep 2017 19:18

Publisher DOI:

10.1111/bjh.13297

PubMed ID:

25683884

Uncontrolled Keywords:

aplastic anaemia, children, haematopoietic stem cell transplantation, immunosuppression

BORIS DOI:

10.7892/boris.66109

URI:

https://boris.unibe.ch/id/eprint/66109

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