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

• Published in: British journal of haematology 2012-05, Vol.157 (3), p.339-346
• Main Authors: 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
• Format: Article
• Language: English
• Subjects: Adolescent; Anemia; Anemia, Aplastic - therapy; Antilymphocyte serum; Antilymphocyte Serum - therapeutic use; Aplastic Anaemia; Biological and medical sciences; Child; Child, Preschool; Children; Chimerism; Cyclophosphamide; Cyclosporine - therapeutic use; Data processing; Donors; Female; fludarabine; Graft vs Host Disease - etiology; Grafts; Hematologic and hematopoietic diseases; Hematopoietic Stem Cell Transplantation - methods; Histocompatibility antigen HLA; Humans; Immunosuppressive agents; Immunosuppressive Agents - therapeutic use; immunosuppressive therapy; Infant; Kaplan-Meier Estimate; Leukemias. Malignant lymphomas. Malignant reticulosis. Myelofibrosis; Male; Medical sciences; Monoclonal antibodies; Opportunistic Infections - etiology; Other diseases. Hematologic involvement in other diseases; paediatric aplastic anaemia; Pediatrics; Rabbit ATG; Radiation; Radiotherapy; Recurrence; Rejection; Retrospective Studies; stem cell transplantation; Transplantation; Transplantation Chimera; Transplantation Conditioning - methods; Treatment Failure; Treatment Outcome; Unrelated Donors
• ISSN: 0007-1048; 1365-2141
• DOI: 10.1111/j.1365-2141.2012.09066.x

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, ‐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.