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Publication Details
AFRICAN RESEARCH NEXUS
SHINING A SPOTLIGHT ON AFRICAN RESEARCH
biochemistry, genetics and molecular biology
Outcome of hematopoietic stem cell transplantation for adenosine deaminase-deficient severe combined immunodeficiency
Blood, Volume 120, No. 17, Year 2012
Notification
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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. © 2012 by The American Society of Hematology.
Authors & Co-Authors
Hassan, Amel Siddik
United Kingdom, London
Ucl Great Ormond Street Institute of Child Health
Booth, Claire
United Kingdom, London
Ucl Great Ormond Street Institute of Child Health
Brightwell, Alex
United Kingdom, London
Ucl Great Ormond Street Institute of Child Health
Allwood, Zoe
United Kingdom, London
Ucl Great Ormond Street Institute of Child Health
Veys, Paul A.
United Kingdom, London
Great Ormond Street Hospital for Children Nhs Foundation Trust
Rao, Kanchana
United Kingdom, London
Great Ormond Street Hospital for Children Nhs Foundation Trust
Hoenig, Manfred
Germany, Ulm
Universitätsklinikum Ulm
Friedrich, Wilhelim W.
Germany, Ulm
Universitätsklinikum Ulm
Gennery, Andrew R.
United Kingdom, Newcastle
University of Newcastle Upon Tyne, Faculty of Medical Sciences
Slatter, Mary Anne
United Kingdom, Newcastle
University of Newcastle Upon Tyne, Faculty of Medical Sciences
Bredius, Robbert G.M.
Netherlands, Leiden
Leids Universitair Medisch Centrum
Finocchi, Andrea
Italy, Rome
Università Degli Studi Di Roma Tor Vergata
Cancrini, Caterina
Italy, Rome
Università Degli Studi Di Roma Tor Vergata
Aïuti, Alessandro
Italy, Rome
Università Degli Studi Di Roma Tor Vergata
Italy, Milan
San Raffaele Telethon Institute for Gene Therapy
Porta, Fulvio A.
Italy, Brescia
Università Degli Studi Di Brescia
Lanfranchi, Arnalda
Italy, Brescia
Università Degli Studi Di Brescia
Ridella, Michela
Italy, Brescia
Università Degli Studi Di Brescia
Steward, Colin G.
United Kingdom, Bristol
Bristol Royal Hospital for Children
Filipovich, Alexandra Hult
United States, Cincinnati
Cincinnati Children's Hospital Medical Center
Marsh, Rebecca A.
United States, Cincinnati
Cincinnati Children's Hospital Medical Center
Bordon, Victoria
Belgium, Ghent
Universitair Ziekenhuis Gent
Al-Muhsen, Saleh Zaid
Saudi Arabia, Riyadh
College of Medicine
Saudi Arabia, Riyadh
King Faisal Specialist Hospital and Research Centre
Al-Mousa, Hamoud A.
Saudi Arabia, Riyadh
King Faisal Specialist Hospital and Research Centre
Alsum, Zobaida
Saudi Arabia, Riyadh
College of Medicine
Saudi Arabia, Riyadh
King Faisal Specialist Hospital and Research Centre
Al-Dhekri, Hasan
Saudi Arabia, Riyadh
King Faisal Specialist Hospital and Research Centre
Al-Ghonaium, Abdulaziz
Saudi Arabia, Riyadh
King Faisal Specialist Hospital and Research Centre
Speckmann, Carsten
Germany, Freiburg Im Breisgau
Universitätsklinikum Freiburg
Fischer, Alain M.
France, Paris
Hôpital Necker Enfants Malades
Mahlaoui, Nizar
France, Paris
Hôpital Necker Enfants Malades
Nichols, Kim E.
United States, Philadelphia
The Children's Hospital of Philadelphia
Grunebaum, Eyal
Canada, Toronto
Hospital for Sick Children University of Toronto
Al-Zahrani, Daifulah M.
Canada, Toronto
Hospital for Sick Children University of Toronto
Roifman, Chaim M.
Canada, Toronto
Hospital for Sick Children University of Toronto
Boelens, Jaap Jan
Netherlands, Utrecht
University Medical Center Utrecht
Davies, E. Graham
United Kingdom, London
Ucl Great Ormond Street Institute of Child Health
Cavazzana-Calvo, Marina
France, Paris
Hôpital Necker Enfants Malades
France, Paris
Ap-hp Assistance Publique - Hopitaux de Paris
France, Paris
Université Paris Cité
Notarangelo, Luigi Daniele
United States, Boston
Harvard Medical School
Gaspar, Hubert Bobby
United Kingdom, London
Ucl Great Ormond Street Institute of Child Health
Statistics
Citations: 38
Authors: 38
Affiliations: 21
Identifiers
Doi:
10.1182/blood-2011-12-396879
ISSN:
00064971
Study Design
Cohort Study