Similar outcome of upfront-unrelated and matched sibling stem cell transplantation in idiopathic paediatric aplastic anaemia. A study on behalf of the UK Paediatric BMT Working Party, Paediatric Diseases Working Party and Severe Aplastic Anaemia Working Party of EBMT
Dufour C (2015), British Journal of Haematology (Link to abstract)
The current first-line treatment for patients with SAA without a matched sibling donor is immune suppressive therapy. This study shows very good results with upfront unrelated donor hematopoietic stem cell transplantation (HSCT)[NW3] for these patients. In a cohort of 29 patients, the 2 year overall survival was 96% and event free survival was 92%, These outcomes were similar to the matched historical sibling donor control group and superior to the matched historical immune suppressive therapy control group.
* In a very small cohort using a different conditioning regimen than used in most centres, HSCT unrelated donors appeared to have similar outcomes to historical sibling-related donors.