The role of allogeneic haematopoietic stem cell transplantation in the treatment of acquired aplastic anaemia in children - experience in the Czech Republic from 1991-2007
Abstract
Allogeneic haematopoietic stem cell transplantation has a defined role in the treatment scheme of acquired aplastic anaemia in children. This article summarises the development and current status of such treatment in paediatric patients in the Czech Republic, covering a period of 17 years (1991-2007).
During this period, a total of 38 children underwent transplantation, of which 74% were transplanted using grafts from HLA matched siblings (n=28) and 26% (n=10) from alternative donors. Median follow-up was 9.8 years (3.3-20.3). 5 year overall survival of the entire group is 92+-4.3% (CI 95%) - sibling donor group 96+-3.5%, alternative donor group 80+-12.6%.
The principal complication and cause of morbidity and mortality is GVHD - higher in the group of recipients of alternative grafts as expected. Our experience demonstrates the benefit of allogeneic hematopoietic stem cell transplantation in children suffering from acquired aplastic anaemia.
Very satisfactory results have been achieved mainly in the group of unrelated matched donors. The main trend has been to optimise conditioning regimens with the aim of reducing both early and late toxicity, also in view of possible late effects.