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Excellent outcome of matched unrelated donor transplantation in paediatric aplastic anaemia following failure with immunosuppressive therapy: a United Kingdom multicentre retrospective experience
Author(s) -
Samarasinghe Sujith,
Steward Colin,
Hiwarkar Prashant,
Saif Muhammad Ameer,
Hough Rachael,
Webb David,
Norton Alice,
Lawson Sarah,
Qureshi Amrana,
Connor Philip,
Carey Peter,
Skinner Rod,
Vora Ajay,
Pelidis Maria,
Gibson Brenda,
Stewart Graham,
Keogh Steve,
Goulden Nick,
Bonney Denise,
Stubbs Mathew,
Amrolia Persis,
Rao Kanchan,
Meyer Stefan,
Wynn Rob,
Veys Paul
Publication year - 2012
Publication title -
british journal of haematology
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.907
H-Index - 186
eISSN - 1365-2141
pISSN - 0007-1048
DOI - 10.1111/j.1365-2141.2012.09066.x
Subject(s) - medicine , alemtuzumab , cyclophosphamide , fludarabine , transplantation , surgery , regimen , total body irradiation , hematopoietic stem cell transplantation , retrospective cohort study , pediatrics , chemotherapy
Summary 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, ‐ DRB 1, ‐ DQB 1) 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 .