Excellent outcome of matched unrelated donor transplantation in paediatric aplastic anaemia following failure with immunosuppressive therapy: a United Kingdom multicentre retrospective experience

Sujith Samarasinghe, Colin Steward, Prashant Hiwarkar, Muhammad Ameer Saif, Rachael Hough, David Webb, Alice Norton, Sarah Lawson, Amrana Qureshi, Philip Connor, Peter Carey, Rod Skinner, Ajay Vora, Maria Pelidis, Brenda Gibson, Graham Stewart, Steve Keogh, Nick Goulden, Denise Bonney, Mathew StubbsPersis Amrolia, Kanchan Rao, Stefan Meyer, Rob Wynn, Paul Veys

Research output: Contribution to journalArticle (Academic Journal)peer-review

83 Citations (Scopus)

Abstract

We retrospectively analysed the outcome of consecutive children with idiopathic severe aplastic anaemia in the United Kingdom who received immunosuppressive therapy (IST) or matched unrelated donor (MUD) haematopoietic stem cell transplantation (HSCT). The 6-month cumulative response rate following rabbit antithymocyte globulin (ATG)/ciclosporin (IST) was 32·5% (95% CI 19·3-46·6) (n = 43). The 5-year estimated failure-free survival (FFS) following IST was 13·3% (95% confidence interval [CI] 4·0-27·8). In contrast, in 44 successive children who received a 10-antigen (HLA-A, -B, -C, -DRB1, -DQB1) MUD HSCT there was an excellent estimated 5-year FFS of 95·01% (95% CI 81·38-98·74). Forty of these children had failed IST previously. HSCT conditioning was a fludarabine, cyclophosphamide and alemtuzumab (FCC) regimen and did not include radiotherapy. There were no cases of graft failure. Median donor chimerism was 100% (range 88-100%). A conditioning regimen, such as FCC that avoids total body irradiation is ideally suited in children. Our data suggest that MUD HSCT following IST failure offers an excellent outcome and furthermore, if a suitable MUD can be found quickly, MUD HSCT may be a reasonable alternative to IST.
Original languageEnglish
Pages (from-to)339-46
Number of pages8
JournalBritish Journal of Haematology
Volume157
Issue number3
DOIs
Publication statusPublished - 2012

Bibliographical note

Joint first author

Fingerprint Dive into the research topics of 'Excellent outcome of matched unrelated donor transplantation in paediatric aplastic anaemia following failure with immunosuppressive therapy: a United Kingdom multicentre retrospective experience'. Together they form a unique fingerprint.

Cite this