Outcomes following treatment for ADA-deficient severe combined immunodeficiency: a report from the PIDTC.

D E Cuvelier, Geoffrey; R Logan, Brent; E Prockop, Susan; H Buckley, Rebecca; Y Kuo, Caroline; M Griffith, Linda; Liu, Xuerong; Yip, Alison; S Hershfield, Michael; G Ayoub, Paul; B Moore, Theodore; J Dorsey, Morna; J O'Reilly, Richard; Kapoor, Neena; Pai, Sung-Yun; Kapadia, Malika; L Ebens, Christen; R Forbes Satter, Lisa; M Burroughs, Lauri; Petrovic, Aleksandra; Chellapandian, Deepak; Heimall, Jennifer; C Shyr, David; Rayes, Ahmad; J Bednarski, Jeffrey; Chandra, Sharat; Chandrakasan, Shanmuganathan; P Gillio, Alfred; Madden, Lisa; C Quigg, Troy; H Caywood, Emi; J Dávila Saldaña, Blachy; DeSantes, Kenneth; Eissa, Hesham; D Goldman, Frederick; Rozmus, Jacob; J Shah, Ami; T Vander Lugt, Mark; S Thakar, Monica; E Parrott, Roberta; Martinez, Caridad; W Leiding, Jennifer; R Torgerson, Troy; A Pulsipher, Michael; D Notarangelo, Luigi; J Cowan, Morton; C Dvorak, Christopher; Haddad, Elie; M Puck, Jennifer; B Kohn, Donald

doi:10.1182/blood.2022016196

Public

Outcomes following treatment for ADA-deficient severe combined immunodeficiency: a report from the PIDTC.

Shared by NobleBlocks on Aug 18, 2022 • 12:00 AM UTC

Authors:

Geoffrey D E Cuvelier

Brent R Logan

Susan E Prockop

Abstract

Adenosine deaminase (ADA) deficiency causes ∼13% of cases of severe combined immune deficiency (SCID). Treatments include enzyme replacement therapy (ERT), hematopoietic cell transplant (HCT), and gene therapy (GT). We evaluated 131 patients with ADA-SCID diagnosed between 1982 and 2017 who were enr...

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