Evidence map›Paper›PMID 41608119›Full record

ArticleJournal of human immunity2026

Neurodevelopmental outcomes following hematopoietic cell transplantation for patients with severe combined immunodeficiency (SCID): A PIDTC study.

Virdette L Brumm, Sharon A Kidd, Brent R Logan, Farheen Chunara, Jennifer Heimall, Linda M Griffith, Donald B Kohn, Lauren Sanchez, Jeffrey J Bednarski, Caridad Martinez and 27 more

Abstract read
In one paragraph

Article in Journal of human immunity, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.

0numbers the graph read from it
0cells of the map it votes in
0citing papers in PubMed
–field-weighted citation impact
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

0 citing papers in PubMed.

No citing paper in PubMed yet.

4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

37 authors.

Virdette L BrummDivision of Pediatric Allergy, Immunology, and Blood and Marrow Transplantation, Department of Pediatrics, University of California San Francisco School of Medicine and UCSF Benioff Children's Hospital, San Francisco, CA, USA.ORCID https://orcid.org/0009-0002-6451-8506
Sharon A KiddDivision of Pediatric Allergy, Immunology, and Blood and Marrow Transplantation, Department of Pediatrics, University of California San Francisco School of Medicine and UCSF Benioff Children's Hospital, San Francisco, CA, USA.ORCID https://orcid.org/0000-0002-7562-8682
Brent R LoganDivision of Biostatistics, Medical College of Wisconsin, Milwaukee, WI, USA.ORCID https://orcid.org/0000-0002-4473-4044
Farheen ChunaraDivision of Biostatistics, Medical College of Wisconsin, Milwaukee, WI, USA.ORCID https://orcid.org/0009-0000-3547-4511
Jennifer HeimallDivision of Allergy and Immunology, Department of Pediatrics, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA, USA.ORCID https://orcid.org/0000-0001-7939-5102
Linda M GriffithDivision of Allergy, Immunology and Transplantation, National Institute of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, MD, USA.ORCID https://orcid.org/0009-0008-7368-3275
Donald B KohnMicrobiology, Immunology & Molecular Genetics, University of California Los Angeles, Los Angeles, CA, USA.ORCID https://orcid.org/0000-0003-1840-6087
Lauren SanchezDivision of Pediatric Allergy, Immunology, and Blood and Marrow Transplantation, Department of Pediatrics, University of California San Francisco School of Medicine and UCSF Benioff Children's Hospital, San Francisco, CA, USA.ORCID https://orcid.org/0000-0001-9062-8181
Jeffrey J BednarskiDivision of Hematology and Oncology, Department of Pediatrics, Washington University School of Medicine, St. Louis, MO, USA.ORCID https://orcid.org/0000-0001-6902-0876
Caridad MartinezCenter for Cell and Gene Therapy, Baylor College of Medicine, Texas Children's Hospital, Houston, TX, USA.ORCID https://orcid.org/0000-0001-7141-2457
Mark Vander LugtBlood and Marrow Transplant Program, University of Michigan, Ann Arbor, Ann Arbor, MI, USA.ORCID https://orcid.org/0000-0001-9714-4454
Neena KapoorTransplantation and Cellular Therapy Section, Cancer and Blood Diseases Institute, Children's Hospital Los Angeles, Keck School of Medicine, University of Southern California, Los Angeles, CA, USA.ORCID https://orcid.org/0000-0002-3352-9651
Nicola WrightDepartment of Pediatrics, University of Calgary, Calgary, Canada.ORCID https://orcid.org/0000-0003-3144-4969
Barbara SpitzerChildren's Cancer Institute, Hackensack University Medical Center, Hackensack, NJ, USA.ORCID https://orcid.org/0000-0003-1603-1381
Joseph H OvedStem Cell Transplantation and Cellular Therapy, MSK Kids, Memorial Sloan Kettering Cancer Center, New York, NY, USA.ORCID https://orcid.org/0000-0002-1129-5129
Sharat ChandraBone Marrow Transplantation and Immune Deficiency, Cincinnati Children's Hospital Medical Center, Cincinnati, OH, USA.ORCID https://orcid.org/0000-0002-2933-814X
Deepak ChellapandianCenter for Cell and Gene Therapy for Non-Malignant Conditions, Johns Hopkins All Children's Hospital, St Petersburg, FL, USA.ORCID https://orcid.org/0000-0003-3518-9790
Christen L EbensDivision of Blood and Marrow Transplantation and Cellular Therapy, Department of Pediatrics, University of Minnesota, Minneapolis, MN, USA.ORCID https://orcid.org/0000-0003-2430-911X
Aleks PetrovicFred Hutchinson Cancer Center, University of Washington School of Medicine and Seattle Children's Hospital, Seattle, WA, USA.ORCID https://orcid.org/0000-0002-3303-7032
Ahmad RayesPediatric Immunology and Hematopoietic Cell Transplantation/Cellular Therapy Program, Primary Children's Hospital, University of Utah, Salt Lake City, UT, USA.ORCID https://orcid.org/0000-0002-6352-9740
Hilary L HainesDivision of Pediatric Hematology and Oncology and Bone Marrow Transplant, University of Alabama at Birmingham, Birmingham AL, USA.ORCID https://orcid.org/0009-0002-7516-1178
Hannah LustDivision of Pediatric Hematology, Oncology, Stem Cell Transplantation, Ann & Robert H. Lurie Children's Hospital of Chicago, Northwestern University Feinberg School of Medicine, Chicago, IL, USA.ORCID https://orcid.org/0000-0002-3579-5996
Hannah-Lise Tirado SchofieldDivision of Oncology, Cellular Therapy and Transplant Section, Children's Hospital of Philadelphia, Philadelphia, PA, USA.ORCID https://orcid.org/0000-0002-1520-6373
Lauren ChristopherDivision of Developmental Medicine, University of California San Francisco, San Francisco, CA, USA.ORCID https://orcid.org/0009-0008-1369-2102
Lynnette L HarrisDepartment of Pediatrics, Psychology Service Baylor College of Medicine, Houston, TX, USA.ORCID https://orcid.org/0009-0001-1611-6118
Lisa Forbes SatterDivision of Immunology, Allergy and Retrovirology, Department of Pediatrics, Center for Human Immunobiology, Texas Childrens Hospital, Houston, TX, USA.ORCID https://orcid.org/0000-0001-9402-1934
Lauri BurroughsFred Hutchinson Cancer Center, University of Washington School of Medicine and Seattle Children's Hospital, Seattle, WA, USA.ORCID https://orcid.org/0000-0003-1124-4740
Christopher C DvorakDivision of Pediatric Allergy, Immunology, and Blood and Marrow Transplantation, Department of Pediatrics, University of California San Francisco School of Medicine and UCSF Benioff Children's Hospital, San Francisco, CA, USA.ORCID https://orcid.org/0000-0002-6146-3952
Elie HaddadDepartment of Pediatrics, Department of Microbiology, Immunology and Infectious Diseases, Center de Recherche Azrieli du Sainte-Justine University of Montreal, Montreal, Canada.ORCID https://orcid.org/0000-0003-2446-6879
Jennifer W LeidingCenter for Cell and Gene Therapy for Non-Malignant Conditions, Johns Hopkins All Children's Hospital, St Petersburg, FL, USA.ORCID https://orcid.org/0000-0002-7573-9390
Rebecca A MarshBone Marrow Transplantation and Immune Deficiency, Cincinnati Children's Hospital Medical Center, Cincinnati, OH, USA.ORCID https://orcid.org/0000-0002-9645-0617
Luigi D NotarangeloLaboratory of Clinical Immunology and Microbiology, National Institute of Allergy and Infectious Diseases/National Institute of Health, Bethesda, MD, USA.ORCID https://orcid.org/0000-0002-8335-0262
Sung Yun PaiImmune Deficiency - Cellular Therapy Program, Center for Cancer Research, National Cancer Institute/ National Institute of Health, Bethesda, MD, USA.ORCID https://orcid.org/0000-0002-0158-8147
Michael A PulsipherDivision on Hematology Oncology, Intermountain Primary Children's, Huntsman Cancer Institute, Spencer Fox Eccles School of Medicine at the University of Utah, Salt Lake City, UT, USA.ORCID https://orcid.org/0000-0003-3030-8420
Jennifer M PuckDivision of Pediatric Allergy, Immunology, and Blood and Marrow Transplantation, Department of Pediatrics, University of California San Francisco School of Medicine and UCSF Benioff Children's Hospital, San Francisco, CA, USA.ORCID https://orcid.org/0000-0001-6623-6276
Morton J CowanDivision of Pediatric Allergy, Immunology, and Blood and Marrow Transplantation, Department of Pediatrics, University of California San Francisco School of Medicine and UCSF Benioff Children's Hospital, San Francisco, CA, USA.ORCID https://orcid.org/0000-0002-1088-8045
Ami J ShahDepartment of Pediatrics, Pediatric Hematology Oncology, Stem Cell Transplantation and Regenerative Medicine, Stanford University, Palo Alto, CA, USA.ORCID https://orcid.org/0000-0001-5700-6188

Funding

X-RAY CRYSTALLOGRAPHYP30CA008748 · NCI · SLOAN-KETTERING INSTITUTE FOR CANCER RES · PI SELWYN M VICKERS · 1985 to 2026
$347.4M
Data Resource for Analyzing Blood &Marrow TransplantsU24CA076518 · NCI · MEDICAL COLLEGE OF WISCONSIN · PI Amy M Moskop, Bronwen Shaw · 1998 to 2026
$105.2M
PB vs. BMU01HL069294 · NHLBI · MEDICAL COLLEGE OF WISCONSIN · PI HOROWITZ, MARY MARESCA · 2001 to 2010
$63.3M
Training of Clinical Investigators in Primary Immune DeficienciesU54AI082973 · NIAID · UNIVERSITY OF CALIFORNIA, SAN FRANCISCO · PI PUCK, JENNIFER M. · 2009 to 2023
$23.3M
Data Management and Coordinating Center (DMCC)U54NS064808 · NINDS · UNIVERSITY OF SOUTH FLORIDA · PI KRISCHER, JEFFREY P · 2009 to 2013
$16.5M
Primary Immune Deficiency Treatment Consortium (PIDTC): A resource to drive scientific and clinical advances related to the treatment of inborn errors of immunityR24AI184316 · NIAID · UNIVERSITY OF CALIFORNIA, SAN FRANCISCO · PI Christopher Craig Dvorak, Elie Haddad · 2024 to 2026
$5.3M
Clinical and mechanistic studies defining optimal preparative approaches to infants with IL2RG/JAK3/RAG1/RAG2 SCID: a randomized trial of busulfan dosageU01AI184132 · NIAID · NATIONAL MARROW DONOR PROGRAM · PI JEFFERY J AULETTA, Michael A Pulsipher · 2024 to 2026
$3.2M
Primary Immune Deficiency Treatment Consortium Annual Scientific MeetingR13AI094943 · NIAID · UNIVERSITY OF CALIFORNIA, SAN FRANCISCO · PI PUCK, JENNIFER M. · 2011 to 2025
$239k
NCI NIH HHS P30 CA008748NCI NIH HHS U24 CA076518NHLBI NIH HHS U01 HL069294NIAID NIH HHS R13 AI094943NIAID NIH HHS R24 AI184316NIAID NIH HHS U01 AI184132NIAID NIH HHS U54 AI082973NINDS NIH HHS U54 NS064808
6 · The paper itself

Abstract

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.

Identifiers

PMID41608119
PMCPMC12829746

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