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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.
Br J Haematol. 2012 May; 157(3):339-46.BJ

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.

Authors+Show Affiliations

Department of Paediatric & Adolescent Haematology, Great North Children's Hospital, Newcastle upon Tyne, UK.No affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info available

Pub Type(s)

Evaluation Study
Journal Article
Multicenter Study

Language

eng

PubMed ID

22372373

Citation

Samarasinghe, Sujith, et al. "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, vol. 157, no. 3, 2012, pp. 339-46.
Samarasinghe S, Steward C, Hiwarkar P, et al. Excellent outcome of matched unrelated donor transplantation in paediatric aplastic anaemia following failure with immunosuppressive therapy: a United Kingdom multicentre retrospective experience. Br J Haematol. 2012;157(3):339-46.
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., ... Veys, P. (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), 339-46. https://doi.org/10.1111/j.1365-2141.2012.09066.x
Samarasinghe S, et al. Excellent Outcome of Matched Unrelated Donor Transplantation in Paediatric Aplastic Anaemia Following Failure With Immunosuppressive Therapy: a United Kingdom Multicentre Retrospective Experience. Br J Haematol. 2012;157(3):339-46. PubMed PMID: 22372373.
* Article titles in AMA citation format should be in sentence-case
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, Y1 - 2012/02/29/ PY - 2011/10/28/received PY - 2012/01/16/accepted PY - 2012/3/1/entrez PY - 2012/3/1/pubmed PY - 2012/6/2/medline SP - 339 EP - 46 JF - British journal of haematology JO - Br. J. Haematol. VL - 157 IS - 3 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. SN - 1365-2141 UR - https://www.unboundmedicine.com/medline/citation/22372373/Excellent_outcome_of_matched_unrelated_donor_transplantation_in_paediatric_aplastic_anaemia_following_failure_with_immunosuppressive_therapy:_a_United_Kingdom_multicentre_retrospective_experience_ L2 - https://doi.org/10.1111/j.1365-2141.2012.09066.x DB - PRIME DP - Unbound Medicine ER -