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

Samarasinghe, S. et al. (2012) Excellent outcome of matched unrelated donor transplantation in paediatric aplastic anaemia following failure with immunosuppressive therapy: a United Kingdom multicentre retrospective experience. British Journal of Haematology, 157(3), pp. 339-346. (doi: 10.1111/j.1365-2141.2012.09066.x)

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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.

Item Type:Articles
Status:Published
Refereed:Yes
Glasgow Author(s) Enlighten ID:Stewart, Dr Graham and Gibson, Professor Brenda
Authors: Samarasinghe, S., Steward, C., Hiwarkar, P., Saif, M.A., Hough, R., Webb, D., Norton, A., Lawson, S., Qureshi, A., Connor, P., Carey, P., Skinner, R., Vora, A., Pelidis, M., Gibson, B., Stewart, G., Keogh, S., Goulden, N., Bonney, D., Stubbs, M., Amrolia, P., Rao, K., Meyer, S., Wynn, R., and Veys, P.
College/School:College of Medical Veterinary and Life Sciences > School of Medicine, Dentistry & Nursing
Journal Name:British Journal of Haematology
Journal Abbr.:Brit. J. Haematol.
ISSN:0007-1048
ISSN (Online):1365-2141
Published Online:29 February 2012

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