Journal of human immunity · 2026

Newborn screening reduces survival disparities in SCID after stem cell transplant: A PIDTC report.

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Winestone Lena E, Logan Brent R, Liu Xuerong, Williams Olatundun, Dunn Elizabeth A, Thakar Monica S, Kidd Sharon A, Mousallem Talal, Dorsey Morna J, O'Reilly Richard J, Kapoor Neena, Forbes Satter Lisa, Kapadia Malika, Jyonouchi Soma C, Chandra Sharat, Ebens Christen L, Chellapandian Deepakbabu, Chaudhury Sonali, Chen Karin, Dávila Saldaña Blachy J, Rayes Ahmad, Quigg Troy C, Chandrakasan Shanmuganathan, Bednarski Jeffrey J, DeSantes Kenneth B, Teira Pierre, Gillio Alfred P, Eissa Hesham, Knutsen Alan P, Aquino Victor M, Shereck Evan B, Moore Theodore B, Caywood Emi H, Vander Lugt Mark T, Rozmus Jacob, Broglie Larisa, Yu Lolie C, Shah Ami J, Joshi Avni Y, Buckley Rebecca H, Dara Jasmeen, Oved Joseph H, Abdel Azim Hisham, Martinez Caridad A, Prockop Susan, Sullivan Kathleen E, Bleesing Jack J, Keller Michael D, Madden Lisa M, Parikh Suhag, Shenoy Shalini, Seroogy Christine M, Rubin Tamar, Licata Jennifer, Andolina Jeffrey R, Kohn Donald B, Burroughs Lauri, Leiding Jennifer W, Marsh Rebecca A, Notarangelo Luigi D, Pai Sung-Yun, Torgerson Troy R, Pulsipher Michael A, Heimall Jennifer, Cuvelier Geoffrey D E, Griffith Linda M, Puck Jennifer M, Dvorak Christopher C, Cowan Morton J, Haddad Elie

Аннотация

Black race and Hispanic ethnicity are associated with higher mortality in severe combined immunodeficiency (SCID) following hematopoietic cell transplantation (HCT), though mechanisms remain unclear. We evaluated 796 children with SCID who received nonsibling HCT between 1982 and 2020 using data from the Primary Immune Deficiency Treatment Consortium. Overall survival for Black (aHR 2.47, 95%CI 1.64, 3.71) and Asian/Pacific Islander patients (aHR 1.82, 95%CI 1.00, 3.30) was significantly lower compared with non-Hispanic White patients, while Hispanic patients had lower event-free survival (aHR 1.83, 95%CI 1.27, 2.63) compared with non-Hispanic White patients. Even after adjusting for age and infection, Black patients with SCID had more than twofold hazard of death compared with non-Hispanic White patients. NBS was associated with earlier diagnosis, reduced infection at HCT, and elimination of survival disparities between Black and non-Hispanic White patients. These findings suggest that universal, system-level interventions such as NBS can mitigate disparities in outcomes for children with SCID.

Newborn screening reduces survival disparities in SCID after stem cell transplant: A PIDTC report.

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