Outcome of hematopoietic stem cell transplantation for adenosine deaminase deficient severe combined immunodeficiency

Amel Hassan, Claire Booth, Alex Brightwell, Zoe Allwood, Paul Veys, Kanchan Rao, Manfred Hönig, Wilhelm Friedrich, Andrew Gennery, Mary Slatter, Robbert Bredius, Andrea Finocchi, Caterina Cancrini, Alessandro Aiuti, Fulvio Porta, Arnalda Lanfranchi, Michela Ridella, Colin Steward, Alexandra Filipovich, Rebecca MarshVictoria Bordon, Saleh Al-Muhsen, Hamoud Al-Mousa, Zobaida Alsum, Hasan Al-Dhekri, Abdulaziz Al Ghonaium, Carsten Speckmann, Alain Fischer, Nizar Mahlaoui, Kim E Nichols, Eyal Grunebaum, Daifulah Al Zahrani, Chaim M Roifman, Jaap Boelens, E Graham Davies, Marina Cavazzana-Calvo, Luigi Notarangelo, H Bobby Gaspar

Research output: Contribution to journalArticle (Academic Journal)peer-review

101 Citations (Scopus)

Abstract

Deficiency of the purine salvage enzyme adenosine deaminase leads to severe combined immunodeficiency (ADA-SCID). Hematopoietic cell transplantation (HCT) can lead to permanent cure of SCID, however little data are available on outcome of HCT for ADA-SCID in particular. In this multicenter retrospective study, we analyzed outcome of HCT in 106 patients with ADA-SCID who received a total of 119 transplants. HCT from matched sibling and family donors (MSD, MFD) had significantly better overall survival (OS) (86% and 81%) in comparison to HCT from matched unrelated (66%; p
Original languageEnglish
JournalBlood
DOIs
Publication statusPublished - 2012

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