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Newborn screening reduces survival disparities in SCID after stem cell transplant: A PIDTC report
Lena E Winestone1,2, Brent R Logan3,4, Xuerong Liu3,4
1Division of Pediatric Allergy, Immunology, and Blood and Marrow Transplantation, University of California, San Francisco, San Francisco, CA, USA.
Insights
Black and Hispanic children with severe combined immunodeficiency (SCID) face higher mortality after hematopoietic cell transplantation (HCT). Newborn screening (NBS) for SCID can eliminate these survival disparities, improving outcomes for all infants.
Area of Science:
- Immunology
- Pediatric Hematology/Oncology
- Public Health
Background:
- Severe combined immunodeficiency (SCID) is a group of rare genetic disorders that affect the immune system.
- Hematopoietic cell transplantation (HCT) is a primary treatment for SCID, but survival rates can vary by race and ethnicity.
- Existing data suggest disparities in HCT outcomes for Black and Hispanic children with SCID.
Purpose of the Study:
- To investigate racial and ethnic disparities in survival outcomes for children with SCID undergoing HCT.
- To evaluate the impact of newborn screening (NBS) on these disparities.
Main Methods:
- Retrospective analysis of 796 children with SCID who received HCT between 1982 and 2020.
- Data sourced from the Primary Immune Deficiency Treatment Consortium.
- Statistical analysis adjusting for age and infection to compare survival rates across racial and ethnic groups, and assess the effect of NBS.
Main Results:
- Black and Asian/Pacific Islander patients had significantly lower overall survival compared to non-Hispanic White patients.
- Hispanic patients experienced lower event-free survival.
- Black patients with SCID faced more than double the hazard of death, even after adjustments.
- Newborn screening (NBS) was linked to earlier diagnosis, reduced infection rates before HCT, and eliminated survival disparities between Black and non-Hispanic White patients.
Conclusions:
- Racial and ethnic disparities exist in SCID outcomes following HCT.
- Newborn screening (NBS) is a critical system-level intervention that can mitigate these disparities and improve survival for all children with SCID.
- Universal NBS programs are essential for achieving equitable healthcare outcomes in pediatric immunodeficiency treatment.
Abstract:
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.
