Immune reconstitution and survival of 100 SCID patients post-hematopoietic cell transplant: a PIDTC natural history study.

doi:10.1182/blood-2017-05-781849

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Article

Immune reconstitution and survival of 100 SCID patients post-hematopoietic cell transplant: a PIDTC natural history study.

Blood - 21 Dec 2017

Heimall Jennifer, Logan Brent R, Cowan Morton J, Notarangelo Luigi D, Griffith Linda M, Puck Jennifer M, Kohn Donald B, Pulsipher Michael A, Parikh Suhag, Martinez Caridad, Kapoor Neena, O'Reilly Richard, Boyer Michael, Pai Sung-Yun, Goldman Frederick, Burroughs Lauri, Chandra Sharat, Kletzel Morris, Thakar Monica, Connelly James, Cuvelier Geoff, Davila Saldana Blachy J, Shereck Evan, Knutsen Alan, Sullivan Kathleen E, DeSantes Kenneth, Gillio Alfred, Haddad Elie, Petrovic Aleksandra, Quigg Troy, Smith Angela R, Stenger Elizabeth, Yin Ziyan, Shearer William T, Fleisher Thomas, Buckley Rebecca H, Dvorak Christopher C

Abstract excerpt

The Primary Immune Deficiency Treatment Consortium (PIDTC) is enrolling children with severe combined immunodeficiency (SCID) to a prospective natural history study. We analyzed patients treated with allogeneic hematopoietic cell transplantation (HCT) from 2010 to 2014, including 68 patients with typical SCID and 32 with leaky SCID, Omenn syndrome, or reticular dysgenesis. Most (59%) patients were diagnosed by...

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PMID: 29021228
DOI: 10.1182/blood-2017-05-781849
PMCID: PMC5746165

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