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Similar outcome of upfront-unrelated and matched sibling stem cell transplantation in idiopathic paediatric aplastic anaemia. A study on behalf of the UK Paediatric BMT Working Party, Paediatric Diseases Working Party and Severe Aplastic Anaemia Working Party of EBMT

Authors :
Persis Amrolia
Rachael Hough
Sujith Samarasinghe
Judith C. W. Marsh
Mark Velangi
Peter Bader
Ajay Vora
Brenda Gibson
Antonio M. Risitano
Austin G. Kulasekararaj
Jakob Passweg
Neha Bhatnagar
Anna Maria Ewins
Alicia Rovó
Roderick Skinner
Régis Peffault de Latour
Mahmoud Aljurf
Andrea Bacigalupo
Marta Pillon
Elisa Carraro
Josu de la Fuente
Colin G. Steward
Britta Höchsmann
Hubert Schrezenmeier
Anja van Biezen
André Tichelli
Carlo Dufour
Rob Wynn
Paul Veys
Gérard Socié
Dufour, Carlo
Veys, Paul
Carraro, Elisa
Bhatnagar, Neha
Pillon, Marta
Wynn, Rob
Gibson, Brenda
Vora, Ajay J.
Steward, Colin G.
Ewins, Anna M.
Hough, Rachael E.
de la Fuente, Josu
Velangi, Mark
Amrolia, Persis J.
Skinner, Roderick
Bacigalupo, Andrea
Risitano, ANTONIO MARIA
Socie, Gerard
Peffault de Latour, Regi
Passweg, Jakob
Rovo, Alicia
Tichelli, André
Schrezenmeier, Hubert
Hochsmann, Britta
Bader, Peter
van Biezen, Anja
Aljurf, Mahmoud D.
Kulasekararaj, Austin
Marsh, Judith C.
Samarasinghe, Sujith
Source :
British Journal of Haematology, 171(4), 585-594, British Journal of Haematology
Publication Year :
2015

Abstract

We explored the feasibility of unrelated donor haematopoietic stem cell transplant (HSCT) upfront without prior immunosuppressive therapy (IST) in paediatric idiopathic severe aplastic anaemia (SAA). This cohort was then compared to matched historical controls who had undergone first-line therapy with a matched sibling/family donor (MSD) HSCT (n = 87) or IST with horse antithymocyte globulin and ciclosporin (n = 58) or second-line therapy with unrelated donor HSCT post-failed IST (n = 24). The 2-year overall survival in the upfront cohort was 96 ± 4% compared to 91 ± 3% in the MSD controls (P = 0·30) and 94 ± 3% in the IST controls (P = 0·68) and 74 ± 9% in the unrelated donor HSCT post-IST failure controls (P = 0·02).The 2-year event-free survival in the upfront cohort was 92 ± 5% compared to 87 ± 4% in MSD controls (P = 0·37), 40 ± 7% in IST controls (P = 0·0001) and 74 ± 9% in the unrelated donor HSCT post-IST failure controls (n = 24) (P = 0·02). Outcomes for upfront-unrelated donor HSCT in paediatric idiopathic SAA were similar to MSD HSCT and superior to IST and unrelated donor HSCT post-IST failure. Front-line therapy with matched unrelated donor HSCT is a novel treatment approach and could be considered as first-line therapy in selected paediatric patients who lack a MSD. © 2015 John Wiley & Sons Ltd.

Details

Language :
English
Database :
OpenAIRE
Journal :
British Journal of Haematology, 171(4), 585-594, British Journal of Haematology
Accession number :
edsair.doi.dedup.....ddedb9e22244ae43911d589e8f768984