TY - JOUR
T1 - Excellent outcome of matched unrelated donor transplantation in paediatric aplastic anaemia following failure with immunosuppressive therapy: A United Kingdom multicentre retrospective experience
AU - Samarasinghe, Sujith
AU - Steward, Colin
AU - Hiwarkar, Prashant
AU - Saif, Muhammad Ameer
AU - Hough, Rachael
AU - Webb, David
AU - Norton, Alice
AU - Lawson, Sarah
AU - Qureshi, Amrana
AU - Connor, Philip
AU - Carey, Peter
AU - Skinner, Rod
AU - Vora, Ajay
AU - Pelidis, Maria
AU - Gibson, Brenda
AU - Stewart, Graham
AU - Keogh, Steve
AU - Goulden, Nick
AU - Bonney, Denise
AU - Stubbs, Mathew
AU - Amrolia, Persis
AU - Rao, Kanchan
AU - Meyer, Stefan
AU - Wynn, Rob
AU - Veys, Paul
PY - 2012/5
Y1 - 2012/5
N2 - 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. © 2012 Blackwell Publishing Ltd.
AB - 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. © 2012 Blackwell Publishing Ltd.
KW - Aplastic Anaemia
KW - Immunosuppressive therapy
KW - Paediatric aplastic anaemia
KW - Rabbit ATG
KW - Transplantation
U2 - 10.1111/j.1365-2141.2012.09066.x
DO - 10.1111/j.1365-2141.2012.09066.x
M3 - Article
SN - 0007-1048
VL - 157
SP - 339
EP - 346
JO - British Journal of Haematology
JF - British Journal of Haematology
IS - 3
ER -