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 Marsh; Victoria 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

doi:10.1182/blood-2011-12-396879

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 journal › Article (Academic Journal) › peer-review

142 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 language	English
Journal	Blood
DOIs	https://doi.org/10.1182/blood-2011-12-396879
Publication status	Published - 2012

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Hassan, A., Booth, C., Brightwell, A., Allwood, Z., Veys, P., Rao, K., Hönig, M., Friedrich, W., Gennery, A., Slatter, M., Bredius, R., Finocchi, A., Cancrini, C., Aiuti, A., Porta, F., Lanfranchi, A., Ridella, M., Steward, C., Filipovich, A., ... Gaspar, H. B. (2012). Outcome of hematopoietic stem cell transplantation for adenosine deaminase deficient severe combined immunodeficiency. Blood. https://doi.org/10.1182/blood-2011-12-396879

@article{1a32f14895c94c6bb0b01566d2f5f3ef,

title = "Outcome of hematopoietic stem cell transplantation for adenosine deaminase deficient severe combined immunodeficiency",

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",

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

year = "2012",

doi = "10.1182/blood-2011-12-396879",

language = "English",

journal = "Blood",

issn = "0006-4971",

publisher = "American Society of Hematology",

}

Hassan, A, Booth, C, Brightwell, A, Allwood, Z, Veys, P, Rao, K, Hönig, M, Friedrich, W, Gennery, A, Slatter, M, Bredius, R, Finocchi, A, Cancrini, C, Aiuti, A, Porta, F, Lanfranchi, A, Ridella, M, Steward, C, Filipovich, A, Marsh, R, Bordon, V, Al-Muhsen, S, Al-Mousa, H, Alsum, Z, Al-Dhekri, H, Al Ghonaium, A, Speckmann, C, Fischer, A, Mahlaoui, N, Nichols, KE, Grunebaum, E, Al Zahrani, D, Roifman, CM, Boelens, J, Davies, EG, Cavazzana-Calvo, M, Notarangelo, L & Gaspar, HB 2012, 'Outcome of hematopoietic stem cell transplantation for adenosine deaminase deficient severe combined immunodeficiency', Blood. https://doi.org/10.1182/blood-2011-12-396879

TY - JOUR

T1 - Outcome of hematopoietic stem cell transplantation for adenosine deaminase deficient severe combined immunodeficiency

AU - Hassan, Amel

AU - Booth, Claire

AU - Brightwell, Alex

AU - Allwood, Zoe

AU - Veys, Paul

AU - Rao, Kanchan

AU - Hönig, Manfred

AU - Friedrich, Wilhelm

AU - Gennery, Andrew

AU - Slatter, Mary

AU - Bredius, Robbert

AU - Finocchi, Andrea

AU - Cancrini, Caterina

AU - Aiuti, Alessandro

AU - Porta, Fulvio

AU - Lanfranchi, Arnalda

AU - Ridella, Michela

AU - Steward, Colin

AU - Filipovich, Alexandra

AU - Marsh, Rebecca

AU - Bordon, Victoria

AU - Al-Muhsen, Saleh

AU - Al-Mousa, Hamoud

AU - Alsum, Zobaida

AU - Al-Dhekri, Hasan

AU - Al Ghonaium, Abdulaziz

AU - Speckmann, Carsten

AU - Fischer, Alain

AU - Mahlaoui, Nizar

AU - Nichols, Kim E

AU - Grunebaum, Eyal

AU - Al Zahrani, Daifulah

AU - Roifman, Chaim M

AU - Boelens, Jaap

AU - Davies, E Graham

AU - Cavazzana-Calvo, Marina

AU - Notarangelo, Luigi

AU - Gaspar, H Bobby

PY - 2012

Y1 - 2012

N2 - 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

AB - 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

U2 - 10.1182/blood-2011-12-396879

DO - 10.1182/blood-2011-12-396879

M3 - Article (Academic Journal)

C2 - 22791287

SN - 0006-4971

JO - Blood

JF - Blood

ER -

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

Abstract

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