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

Content Provider	Scilit
Author	Hassan, Amel Booth, Claire Brightwell, Alex Allwood, Zoe Veys, Paul Rao, Kanchan Hönig, Manfred Friedrich, Wilhelm Gennery, Andrew Slatter, Mary Bredius, Robbert Finocchi, Andrea Cancrini, Caterina Aiuti, Alessandro Porta, Fulvio Lanfranchi, Arnalda Ridella, Michela Steward, Colin Filipovich, Alexandra Marsh, Rebecca Bordon, Victoria Al-Muhsen, Saleh Al-Mousa, Hamoud Alsum, Zobaida Al-Dhekri, Hasan Ghonaium, Abdulaziz Al Speckmann, Carsten Fischer, Alain Mahlaoui, Nizar Nichols, Kim E. Grunebaum, Eyal Zahrani, Daifulah Al Roifman, Chaim M. Boelens, Jaap Davies, E. Graham Cavazzana-Calvo, Marina Notarangelo, Luigi Gaspar, H. Bobby
Copyright Year	2012
Description	Deficiency of the purine salvage enzyme adenosine deaminase leads to SCID (ADA-SCID). Hematopoietic cell transplantation (HCT) can lead to a 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 (MSDs, MFDs) had significantly better overall survival (86% and 81%) in comparison with HCT from matched unrelated (66%; P < .05) and haploidentical donors (43%; P < .001). Superior overall survival was also seen in patients who received unconditioned transplants in comparison with myeloablative procedures (81% vs 54%; P < .003), although in unconditioned haploidentical donor HCT, nonengraftment was a major problem. Long-term immune recovery showed that regardless of transplant type, overall T-cell numbers were similar, although a faster rate of T-cell recovery was observed after MSD/MFD HCT. Humoral immunity and donor B-cell engraftment was achieved in nearly all evaluable surviving patients and was seen even after unconditioned HCT. These data detail for the first time the outcomes of HCT for ADA-SCID and show that, if patients survive HCT, long-term cellular and humoral immune recovery is achieved.
Related Links	https://ashpublications.org/blood/article-pdf/120/17/3615/1358132/zh804312003615.pdf
Ending Page	3624
Page Count	10
Starting Page	3615
DOI	10.1182/blood-2011-12-396879
Journal	Blood
Issue Number	17
Volume Number	120
Language	English
Publisher	American Society of Hematology
Publisher Date	2012-10-25
Access Restriction	Open
Subject Keyword	Research and Experimental Medicine Hematopoietic Survival Transplantation Ada Scid Hct for Ada Outcome of Hct Journal: Blood (Vol- 127, Issue- 17)
Content Type	Text
Resource Type	Article