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

Media type: E-Article
Title: Excellent outcome of matched unrelated donor transplantation in paediatric aplastic anaemia following failure with immunosuppressive therapy: a United Kingdom multicentre retrospective experience
Contributor: 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;
imprint: Wiley, 2012
Published in: British Journal of Haematology
Language: English
DOI: 10.1111/j.1365-2141.2012.09066.x
ISSN: 0007-1048; 1365-2141
Keywords: Hematology
Origination:
Footnote:
Description: <jats:title>Summary</jats:title><jats:p>We retrospectively analysed the outcome of consecutive children with idiopathic severe aplastic anaemia in the United Kingdom who received immunosuppressive therapy (<jats:styled-content style="fixed-case">IST</jats:styled-content>) or matched unrelated donor (<jats:styled-content style="fixed-case">MUD</jats:styled-content>) haematopoietic stem cell transplantation (<jats:styled-content style="fixed-case">HSCT</jats:styled-content>). The 6‐month cumulative response rate following rabbit antithymocyte globulin (<jats:styled-content style="fixed-case">ATG</jats:styled-content>)/ciclosporin (<jats:styled-content style="fixed-case">IST</jats:styled-content>) was 32·5% (95% <jats:styled-content style="fixed-case">CI</jats:styled-content> 19·3–46·6) (<jats:italic>n</jats:italic> = 43). The 5‐year estimated failure‐free survival (<jats:styled-content style="fixed-case">FFS</jats:styled-content>) following <jats:styled-content style="fixed-case">IST</jats:styled-content> was 13·3% (95% confidence interval [<jats:styled-content style="fixed-case">CI</jats:styled-content>] 4·0–27·8). In contrast, in 44 successive children who received a 10‐antigen (<jats:styled-content style="fixed-case">HLA</jats:styled-content>‐A, ‐B, ‐C, ‐<jats:styled-content style="fixed-case">DRB</jats:styled-content>1, ‐<jats:styled-content style="fixed-case">DQB</jats:styled-content>1) <jats:styled-content style="fixed-case">MUD HSCT</jats:styled-content> there was an excellent estimated 5‐year <jats:styled-content style="fixed-case">FFS</jats:styled-content> of 95·01% (95% <jats:styled-content style="fixed-case">CI</jats:styled-content> 81·38–98·74). Forty of these children had failed <jats:styled-content style="fixed-case">IST</jats:styled-content> previously. <jats:styled-content style="fixed-case">HSCT</jats:styled-content> conditioning was a fludarabine, cyclophosphamide and alemtuzumab (<jats:styled-content style="fixed-case">FCC</jats:styled-content>) 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 <jats:styled-content style="fixed-case">FCC</jats:styled-content> that avoids total body irradiation is ideally suited in children. Our data suggest that <jats:styled-content style="fixed-case">MUD HSCT</jats:styled-content> following <jats:styled-content style="fixed-case">IST</jats:styled-content> failure offers an excellent outcome and furthermore, if a suitable <jats:styled-content style="fixed-case">MUD</jats:styled-content> can be found quickly, <jats:styled-content style="fixed-case">MUD HSCT</jats:styled-content> may be a reasonable alternative to <jats:styled-content style="fixed-case">IST</jats:styled-content>.</jats:p>
Access State: Open Access

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