Neurodevelopmental outcomes following hematopoietic cell transplantation for patients with severe combined immunodeficiency (SCID): A PIDTC study
Hematopoietic cell transplantation (HCT) is a potentially curative treatment for severe combined immunodeficiency (SCID). Since the initiation of newborn screening (NBS), survival rates have improved significantly, but the impact of HCT upon neurodevelopment for patients with SCID requires more investigation. We performed a cross-sectional study of subjects with SCID in North America to assess the impact of NBS, transplant conditioning regimen, and genotype on neurodevelopmental outcomes after HCT. 69 subjects with SCID from 17 PIDTC centers (excluding those with ADA deficiency), ages 6-16 years, received comprehensive standardized neurodevelopmental testing of cognitive, behavioral, and emotional function. Compared with the normative population, our subjects performed in the average range. We found no impact of NBS, chemotherapy conditioning, or genotype. Multivariate analysis revealed a significant decrease in IQ in subjects whose families earned <$50,000 per year. We recommend that children treated by HCT for SCID be monitored with periodic cognitive and behavioral assessments for deficits that could potentially impact long-term ND outcomes.
Authors
- Hilary Haines (ORCID: https://orcid.org/0009-0002-7516-1178)
- Deepak Chellapandian (ORCID: https://orcid.org/0000-0003-3518-9790)
- Ahmad Rayes (ORCID: https://orcid.org/0000-0002-6352-9740)
- Michael A. Pulsipher (ORCID: https://orcid.org/0000-0003-3030-8420)
- Mark T. Vander Lugt (ORCID: https://orcid.org/0000-0001-9714-4454)
- Lisa Forbes Satter (ORCID: https://orcid.org/0000-0001-9402-1934)
- Jennifer M. Puck (ORCID: https://orcid.org/0000-0001-6623-6276)
- Sung‐Yun Pai (ORCID: https://orcid.org/0000-0002-0158-8147)
- Nicola Wright (ORCID: https://orcid.org/0000-0003-3144-4969)
- Hannah-Lise Schofield (ORCID: https://orcid.org/0000-0002-1520-6373)
- Brent R. Logan (ORCID: https://orcid.org/0000-0002-4473-4044)
- Caridad Martinez (ORCID: https://orcid.org/0000-0001-7141-2457)
- Luigi Daniele Notarangelo (ORCID: https://orcid.org/0000-0002-8335-0262)
- Rebecca Marsh (ORCID: https://orcid.org/0000-0002-9645-0617)
- Sharon A. Kidd (ORCID: https://orcid.org/0000-0002-7562-8682)
- Christen L. Ebens (ORCID: https://orcid.org/0000-0003-2430-911X)
- Hannah Lust (ORCID: https://orcid.org/0000-0002-3579-5996)
- Lauren Sanchez (ORCID: https://orcid.org/0000-0001-9062-8181)
- Christopher C. Dvorak (ORCID: https://orcid.org/0000-0002-6146-3952)
- Donald B. Kohn (ORCID: https://orcid.org/0000-0003-1840-6087)
- Jennifer Heimall (ORCID: https://orcid.org/0000-0001-7939-5102)
- Morton J. Cowan (ORCID: https://orcid.org/0000-0002-1088-8045)
- Barbara Spitzer (ORCID: https://orcid.org/0000-0003-1603-1381)
- Virdette L. Brumm (ORCID: https://orcid.org/0009-0002-6451-8506)
- Élie Haddad (ORCID: https://orcid.org/0000-0003-2446-6879)
- Ami J. Shah (ORCID: https://orcid.org/0000-0001-5700-6188)
- Jeffrey J. Bednarski (ORCID: https://orcid.org/0000-0001-6902-0876)
- Sharat Chandra (ORCID: https://orcid.org/0000-0002-2933-814X)
- Joseph H. Oved (ORCID: https://orcid.org/0000-0002-1129-5129)
- Lauri M. Burroughs (ORCID: https://orcid.org/0000-0003-1124-4740)
- Jennifer W. Leiding (ORCID: https://orcid.org/0000-0002-7573-9390)
- Farheen Chunara (ORCID: https://orcid.org/0009-0000-3547-4511)
- Neena Kapoor (ORCID: https://orcid.org/0000-0002-3352-9651)
- Aleksandra Petrovič (ORCID: https://orcid.org/0000-0002-3303-7032)
- Linda M. Griffith (ORCID: https://orcid.org/0009-0008-7368-3275)
- L.D. Christopher (ORCID: https://orcid.org/0009-0008-1369-2102)
- Lynnette Harris (ORCID: https://orcid.org/0009-0001-1611-6118)
Institutions
- Cincinnati Children's Hospital Medical Center (US)
- Hackensack University Medical Center (US)
- Primary Children's Hospital (US)
- Memorial Sloan Kettering Cancer Center (US)
- Children's Hospital of Philadelphia (US)
- Children's Hospital of Los Angeles (US)
- Johns Hopkins University (US)
- University of California, Los Angeles (US)
- University of Calgary (CA)
- University of California, San Francisco (US)
- Baylor College of Medicine (US)
- California Institute for Regenerative Medicine (US)
- Medical College of Wisconsin (US)
- Washington University in St. Louis (US)
- University of Michigan (US)
- Huntsman Cancer Institute (US)
- Centre Hospitalier Universitaire Sainte-Justine (CA)
- University of Alabama at Birmingham (US)
- Institute for Stem Cell Biology and Regenerative Medicine (IN)
- Fred Hutch Cancer Center (US)
- Johns Hopkins All Children's Hospital (US)
- Seattle Children's Hospital (US)
- Michigan Medicine (US)
- Versiti Blood Center of Wisconsin (US)
- Lurie Children's Hospital (US)
- National Institute of Allergy and Infectious Diseases (US)
- University of Minnesota Medical Center (US)
- UCSF Benioff Children's Hospital (US)
- National Cancer Institute (US)
- Texas Children's Hospital (US)
- Robert H. Lurie Comprehensive Cancer Center of Northwestern University
- University of San Francisco (US)
- University of Pennsylvania (US)
Publication Details
- Journal
- Journal of Human Immunity
- Published
- 2025-11-05
- DOI
- https://doi.org/10.70962/jhi.20250163
- Primary Topic
- Immunodeficiency and Autoimmune Disorders
- Type
- article
- Field-Weighted Citation Impact
- 0.00
Funders
- U.S. Department of Health and Human Services
- Amgen
- Immune Deficiency Foundation
- California Institute of Regenerative Medicine
- Swedish Orphan Biovitrum
- Grifols
- National Institutes of Health
- Health Resources and Services Administration
- National Heart, Lung, and Blood Institute
- Office of Naval Research
- National Cancer Institute
- National Institute of Allergy and Infectious Diseases
- National Center for Advancing Translational Sciences