ArticleLancet (London, England)2023
Measuring the effect of newborn screening on survival after haematopoietic cell transplantation for severe combined immunodeficiency: a 36-year longitudinal study from the Primary Immune Deficiency Treatment Consortium.
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In one paragraph
Article in Lancet (London, England), 2023. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 68 papers, 2 of them syntheses that pooled it.
0numbers the graph read from it
0cells of the map it votes in
68citing papers in PubMed, 2 pooled it
16.6field-weighted citation impact, top 1% of its field
What it found
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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.
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.
Who cites it
68 citing papers in PubMed, 2 syntheses or guidelines pooled it, 101 citations in OpenAlex.
- Newborn screening for severe combined immunodeficiency in the NHS newborn blood spot screening programme: a systematic review.Health technology assessment (Winchester, England) · 2026Pooled it
- Overview of next-generation sequencing to the molecular diagnosis of inborn errors of immunity in Brazil: a systematic review.Frontiers in immunology · 2026Pooled it
- How I Treat: Transplantation for primary immune regulatory diseases and inflammatory disorders.Journal of human immunity · 2026Article
- The cost-benefit of newborn screening for X-linked agammaglobulinemia and related B-cell lymphopenia.Immunologic research · 2026Review
- Nationwide TREC/KREC-based newborn screening and clinical outcomes in Japan.Journal of human immunity · 2026Article
- Second-tier genetics improves newborn screening accuracy for SCID and other T cell deficiencies.Journal of human immunity · 2026Article
- Newborn screening reduces survival disparities in SCID after stem cell transplant: A PIDTC report.Journal of human immunity · 2026Article
- Interpreting abnormal severe combined immunodeficiency (SCID) newborn screens: Perinatal predictors and clinical implications.Annals of allergy, asthma & immunology : official publication of the American College of Allergy, Asthma, & Immunology · 2026Article
- Inborn Errors of Immunity Genotype: A Retrospective Study from the United Arab Emirates.Journal of clinical immunology · 2026Article
- Evolution of Inborn Errors of Immunity in China: Discoveries and Treatment Paradigms.Clinical reviews in allergy & immunology · 2026Review
- Implementation of TREC/KREC Newborn Screening in a High-Birth-Rate Population: A Pilot Study of 5000 Neonates in South Kazakhstan.International journal of neonatal screening · 2026Article
- Allogeneic hematopoietic cell transplantation for partial RAG deficiency in children and adults: Excellent outcomes with a reduced-intensity posttransplantation cyclophosphamide-based approach.The Journal of allergy and clinical immunology · 2026Article
- Optimizing TREC- and KREC-based newborn screening: Risk-stratified algorithms significantly reduce referrals.Pediatric allergy and immunology : official publication of the European Society of Pediatric Allergy and Immunology · 2026Article
- Updated EBMT/ESID inborn errors working party guidelines for haematopoietic stem cell transplantation for inborn errors of immunity and metabolism.Bone marrow transplantation · 2026Review
- Excellent survival after allogeneic HSCT in screened SCID patients and donor type dependent naïve T cell reconstitution.Bone marrow transplantation · 2026Article
- Inherited human TFIIIA deficiency disrupts T cell development.medRxiv : the preprint server for health sciences · 2026Article
- Transient B cell lymphopenia revealed by KRECs newborn screening: Post-screening referral strategies, clinical course, and follow-up.Pediatric allergy and immunology : official publication of the European Society of Pediatric Allergy and Immunology · 2026Article
- Development of Clinical Pathways for Early Diagnosis and Management of SCID, SMA, and XLA Through Newborn Screening in Malaysia.International journal of neonatal screening · 2026Review
- Bringing the rare into sight: applications of haploidentical stem cell transplantation in the treatment of rare diseases.Bone marrow transplantation · 2026Review
- Dominant-negative PSMB10 disrupts immunoproteasome assembly and leads to transient T lymphopenia.Journal of human immunity · 2026Article
8 more citing papers are in PubMed but not listed here.
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Authors and funding
65 authors at 20 institutions in 2 countries.
Monica S ThakarFred Hutchinson Cancer Center, Seattle, WA, USA; Department of Pediatrics, University of Washington, Seattle, WA, USA. Electronic address: msthakar@fredhutch.org.
Brent R LoganDivision of Biostatistics, Medical College of Wisconsin, WI, USA; Center for International Blood and Marrow Transplant Research, Milwaukee, WI, USA.
Jennifer M PuckDivision of Pediatric Allergy, Immunology, and Blood and Marrow Transplantation, University of California San Francisco, CA, USA; UCSF Benioff Children's Hospital, San Francisco, CA, USA.
Elizabeth A DunnDivision of Pediatric Allergy, Immunology, and Blood and Marrow Transplantation, University of California San Francisco, CA, USA.
Rebecca H BuckleyDepartment of Allergy and Immunology, Department of Pediatrics and Immunology, Duke University Medical Center, Durham, NC, USA.
Morton J CowanDivision of Pediatric Allergy, Immunology, and Blood and Marrow Transplantation, University of California San Francisco, CA, USA; UCSF Benioff Children's Hospital, San Francisco, CA, USA.
Richard J O'ReillyStem Cell Transplantation and Cellular Therapy, MSK Kids, Memorial Sloan Kettering Cancer Center, New York, NY, USA.
Neena KapoorTransplant and Cell Therapy Program and Laboratory, Department of Pediatrics, Keck School of Medicine, University of Southern California, CA, USA; Hematology, Oncology and TCT, Children's Hospital Los Angeles, Los Angeles, CA, USA.
Lisa Forbes SatterDepartment of Pediatrics, Baylor College of Medicine, Houston, TX, USA; Immunology Allergy and Retrovirology, Center for Human Immunobiology, Texas Children's Hospital Infusion Center, Houston, TX, USA.
Sung-Yun PaiImmune Deficiency Cellular Therapy Program, Center for Cancer Research, National Cancer Institute (NCI)/NIH, Bethesda, MD, USA.
Jennifer HeimallDepartment of Pediatrics, Perelman School of Medicine at the University of Pennsylvania, PA, USA; Division of Allergy and Immunology, Children's Hospital of Philadelphia, Philadelphia, PA, USA.
Sharat ChandraDepartment of Pediatrics, University of Cincinnati College of Medicine, Cincinnati, OH, USA; Division of Bone Marrow Transplantation and Immune Deficiency, Cincinnati Children's Hospital Medical Center, Cincinnati, OH, USA.
Christen L EbensDepartment of Pediatrics, Division of Blood and Marrow Transplantation & Cellular Therapy, University of Minnesota, Minneapolis, MN, USA.
Deepak ChellapandianPediatric Oncology, Johns Hopkins University School of Medicine, Baltimore, MD, USA; Center for Cell and Gene Therapy for Nonmalignant Conditions, Johns Hopkins All Children's Hospital, St Petersburg, FL, USA.
Olatundun WilliamsDepartment of Pediatrics, Columbia University Irving Medical Center, New York, NY, USA; Division of Pediatric Hematology, Oncology and Stem Cell Transplantation, New York-Presbyterian Morgan Stanley Children's Hospital, New York, NY, USA.
Lauri M BurroughsFred Hutchinson Cancer Center, Seattle, WA, USA; Department of Pediatrics, University of Washington, Seattle, WA, USA.
Blachy Davila SaldanaDepartment of Pediatrics, George Washington University School of Medicine and Health Sciences, Washington DC, USA; Division of Blood and Marrow Transplantation and Center for Cancer and Immunology Research, Children's National Hospital, Washington DC, USA.
Ahmad RayesPediatric Immunology and Blood and Marrow Transplant Program, University of Utah, Salt Lake City, UT, USA; Intermountain Primary Children's Hospital, Salt Lake City, UT, USA.
Lisa M MaddenPediatric Bone Marrow Transplant Program, Texas Transplant Institute, San Antonio, TX, USA.
Shanmuganathan ChandrakasanDepartment of Pediatrics, Emory University School of Medicine, Atlanta, GA, USA; Aflac Cancer and Blood Disorders Center, Children's Healthcare of Atlanta, Atlanta, GA, USA.
Jeffrey J BednarskiDepartment of Pediatrics, Washington University School of Medicine, St Louis, MO, USA.
Kenneth B DeSantesDepartment of Pediatrics, University of Wisconsin, Madison, WI, USA.
Geoffrey D E CuvelierUniversity of Manitoba, Winnipeg, MB, Canada; Manitoba Blood and Marrow Transplant Program, CancerCare Manitoba, Winnipeg, MB, Canada.
Pierre TeiraDepartment of Pediatrics and Department of Microbiology, Immunology and Infectious Diseases, University of Montreal, Montreal, QC, Canada; Centre Hospitalier Universitaire Sainte-Justine, Montreal, QC, Canada.
Alfred P GillioPediatric Stem Cell and Cellular Therapy Division, Joseph M Sanzari Children's Hospital at HMH Hackensack University Medical Center, Hackensack, NJ, USA.
Hesham EissaDepartment of Pediatrics, University of Colorado, Aurora, CO, USA; Bone Marrow Transplant and Cellular Therapeutics, Children's Hospital of Colorado, Aurora, CO, USA.
Alan P KnutsenPediatric Allergy and Immunology, St Louis University, St Louis, MO, USA; Jeffrey Modell Diagnostic & Research Center for Primary Immunodeficiencies, Cardinal Glennon Children's Hospital, St Louis, MO, USA.
Frederick D GoldmanDivision of Hematology/Oncology/BMT, Department of Pediatrics, University of Alabama, Birmingham, AL, USA.
Victor M AquinoDepartment of Pediatrics, University of Texas Southwestern Medical Center, Dallas, TX, USA.
Evan B ShereckDepartment of Pediatrics, Oregon Health & Science University, Portland, OR, USA.
Theodore B MoorePediatric Blood and Marrow Transplant Program, Division of Pediatric Hematology/Oncology in the Department of Pediatrics, University of California Los Angeles, Los Angeles, CA, USA.
Emi H CaywoodSidney Kimmel Medical College, Thomas Jefferson University, Philadelphia, PA, USA; Nemours Children's Health, Delaware, Wilmington, DE, USA.
Mark T Vander LugtDepartment of Pediatrics, University of Michigan, Ann Arbor, MI, USA.
Jacob RozmusDepartment of Pediatrics, University of British Columbia, Vancouver, BC, Canada; British Columbia Children's Hospital, Vancouver, BC, Canada.
Larisa BroglieCenter for International Blood and Marrow Transplant Research, Milwaukee, WI, USA; Department of Pediatrics, Medical College of Wisconsin, Milwaukee, WI, USA.
Lolie C YuLouisiana State University Health New Orleans School of Medicine, New Orleans, LA, USA; Children's Hospital of New Orleans, New Orleans, LA, USA.
Ami J ShahDivision of Hematology/Oncology/Stem Cell Transplantation and Regenerative Medicine, Department of Pediatrics, Stanford School of Medicine, Palo Alto, CA, USA.
Jeffrey R AndolinaDepartment of Pediatrics, Golisano Children's Hospital, University of Rochester Medical Center, Rochester, NY, USA.
Xuerong LiuDivision of Biostatistics, Medical College of Wisconsin, WI, USA.
Roberta E ParrottDepartment of Allergy and Immunology, Department of Pediatrics and Immunology, Duke University Medical Center, Durham, NC, USA.
Jasmeen DaraDivision of Pediatric Allergy, Immunology, and Blood and Marrow Transplantation, University of California San Francisco, CA, USA; UCSF Benioff Children's Hospital, San Francisco, CA, USA.
Susan ProckopDepartment of Pediatrics, Harvard University Medical School, Boston, MA, USA; Dana Farber/Boston Children's Cancer and Blood Disorders Center, Boston, MA, USA.
Caridad A MartinezDepartment of Pediatrics, Baylor College of Medicine, Houston, TX, USA; Immunology Allergy and Retrovirology, Center for Human Immunobiology, Texas Children's Hospital Infusion Center, Houston, TX, USA.
Malika KapadiaDepartment of Pediatrics, Harvard University Medical School, Boston, MA, USA; Dana Farber/Boston Children's Cancer and Blood Disorders Center, Boston, MA, USA.
Soma C JyonouchiDepartment of Pediatrics, Perelman School of Medicine at the University of Pennsylvania, PA, USA; Division of Allergy and Immunology, Children's Hospital of Philadelphia, Philadelphia, PA, USA.
Kathleen E SullivanDepartment of Pediatrics, Perelman School of Medicine at the University of Pennsylvania, PA, USA; Division of Allergy and Immunology, Children's Hospital of Philadelphia, Philadelphia, PA, USA.
Jack J BleesingDepartment of Pediatrics, University of Cincinnati College of Medicine, Cincinnati, OH, USA; Division of Bone Marrow Transplantation and Immune Deficiency, Cincinnati Children's Hospital Medical Center, Cincinnati, OH, USA.
Sonali ChaudhuryNorthwestern University Feinberg School of Medicine, Chicago, IL, USA; Division of Pediatric Hematology/Oncology/Stem Cell Transplantation, Ann and Robert H. Lurie Children's Hospital of Chicago, Chicago, IL, USA.
Aleksandra PetrovicFred Hutchinson Cancer Center, Seattle, WA, USA; Department of Pediatrics, University of Washington, Seattle, WA, USA.
Michael D KellerDepartment of Pediatrics, George Washington University School of Medicine and Health Sciences, Washington DC, USA; Division of Blood and Marrow Transplantation and Center for Cancer and Immunology Research, Children's National Hospital, Washington DC, USA; Intermountain Primary Children's Hospital, Salt Lake City, UT, USA.
Troy C QuiggPediatrics, Michigan State University College of Human Medicine, Grand Rapids, MI, USA; Pediatric Blood and Marrow Transplant and Cellular Therapy Program, Helen DeVos Children's Hospital, Grand Rapids, MI, USA.
Suhag ParikhDepartment of Pediatrics, Emory University School of Medicine, Atlanta, GA, USA; Aflac Cancer and Blood Disorders Center, Children's Healthcare of Atlanta, Atlanta, GA, USA.
Shalini ShenoyDepartment of Pediatrics, Washington University School of Medicine, St Louis, MO, USA.
Christine SeroogyDivision of Allergy, Immunology, and Rheumatology, University of Wisconsin, Madison, WI, USA.
Tamar RubinDivision of Pediatric Allergy and Immunology, University of Manitoba, Winnipeg, MB, Canada.
Hélène DecaluweDepartment of Pediatrics and Department of Microbiology, Immunology and Infectious Diseases, University of Montreal, Montreal, QC, Canada; Centre Hospitalier Universitaire Sainte-Justine, Montreal, QC, Canada.
John M RoutesDepartment of Pediatrics, Medical College of Wisconsin, Milwaukee, WI, USA.
Troy R TorgersonExperimental Immunology, Allen Institute for Immunology, Seattle, WA, USA.
Jennifer W LeidingDivision of Allergy and Immunology, Department of Pediatrics, Johns Hopkins University, Baltimore, MD, USA.
Michael A PulsipherPediatric Immunology and Blood and Marrow Transplant Program, University of Utah, Salt Lake City, UT, USA; Intermountain Primary Children's Hospital, Salt Lake City, UT, USA.
Donald B KohnPediatric Blood and Marrow Transplant Program, Division of Pediatric Hematology/Oncology in the Department of Pediatrics, University of California Los Angeles, Los Angeles, CA, USA.
Linda M GriffithDivision of Allergy, Immunology and Transplantation, (NIAID)/NIH, Bethesda, MD, USA.
Elie HaddadDepartment of Pediatrics and Department of Microbiology, Immunology and Infectious Diseases, University of Montreal, Montreal, QC, Canada; Centre Hospitalier Universitaire Sainte-Justine, Montreal, QC, Canada.
Christopher C DvorakDivision of Pediatric Allergy, Immunology, and Blood and Marrow Transplantation, University of California San Francisco, CA, USA; UCSF Benioff Children's Hospital, San Francisco, CA, USA.
Luigi D NotarangeloLaboratory of Clinical Immunology and Microbiology, National Institute of Allergy and Infectious Diseases (NIAID)/NIH, Bethesda, MD, USA.
Medical College of Wisconsin · USUCSF Benioff Children's Hospital · USChildren's Hospital of Philadelphia · USAflac (United States) · USCenter for Cancer and Blood Disorders · USDuke Medical Center · USNational Institute of Allergy and Infectious Diseases · USUniversité de Montréal · CAUniversity of California, Los Angeles · USUniversity of Manitoba · CAUniversity of Washington · USUniversity of Wisconsin–Madison · USWashington University in St. Louis · USAllen Institute for Immunology · USBaylor College of Medicine · USCenter for Cancer Research · USCentre Hospitalier Universitaire Sainte-Justine · CAChildren's Hospital Colorado · USChildren's Hospital of Los Angeles · USCHRISTUS Transplant Institute · US
Funding
X-RAY CRYSTALLOGRAPHYP30CA008748 · NCI · SLOAN-KETTERING INSTITUTE FOR CANCER RES · PI Hongbiao Carl Lekaye · 1985 to 2026
$347.4MData Resource for Analyzing Blood &Marrow TransplantsU24CA076518 · NCI · MEDICAL COLLEGE OF WISCONSIN · PI Amy M Moskop, Bronwen Shaw · 1998 to 2026
$105.2MUTAH REGIONAL CANCER CENTERP30CA042014 · NCI · UTAH STATE HIGHER EDUCATION SYSTEM--UNIVERSITY OF UTAH · PI KATHARINE S ULLMAN · 1986 to 2026
$72.6MPB vs. BMU01HL069294 · NHLBI · MEDICAL COLLEGE OF WISCONSIN · PI HOROWITZ, MARY MARESCA · 2001 to 2010
$63.3MRare Diseases Data Management and Coordinating CenterU01TR001263 · NCATS · UNIVERSITY OF SOUTH FLORIDA · PI KRISCHER, JEFFREY P · 2014 to 2018
$28.1MTraining of Clinical Investigators in Primary Immune DeficienciesU54AI082973 · NIAID · UNIVERSITY OF CALIFORNIA, SAN FRANCISCO · PI DVORAK, CHRISTOPHER CRAIG, HADDAD, ELIE · 2009 to 2023
$23.3MData Management and Coordinating Center (DMCC)U54NS064808 · NINDS · UNIVERSITY OF SOUTH FLORIDA · PI KRISCHER, JEFFREY P · 2009 to 2013
$16.5MCharacterization of the cellular and molecular bases of inborn errors of immunityZIAAI001222 · NIAID · NATIONAL INSTITUTE OF ALLERGY AND INFECTIOUS DISEASES · PI NOTARANGELO, LUIGI · 2017 to 2025
$15.2MRandomized study of low versus moderate dose busulfan in transplant for severe combined immunodeficiencyU01AI126612 · NIAID · CHILDREN'S HOSPITAL OF LOS ANGELES · PI KEAN, LESLIE S, PULSIPHER, MICHAEL A · 2017 to 2023
$8.5MUnrelated Donor BMT vs. Immune Suppression for Newly Diagnosed Severe Aplastic Anemia in Children and Young Adults: BMT CTN Core Center Renewal for the Pediatric Blood and Marrow Transplant ConsortiumUG1HL069254 · NHLBI · CHILDREN'S HOSPITAL OF LOS ANGELES · PI KEAN, LESLIE S · 2017 to 2023
$1.3MSafety and Efficacy of Itacitinib in treatment of JAK/STAT pathway disorders with activating mutationsUG3TR003908 · NCATS · BAYLOR COLLEGE OF MEDICINE · PI FORBES, LISA, TORGERSON, TROY R · 2021 to 2022
$1.2MBlood and Marrow Transplant Clinical Trials NetworkU10HL069254 · NHLBI · UNIVERSITY OF UTAH · PI PULSIPHER, MICHAEL A · 2011 to 2016
$839kIntramural NIH HHS ZIA AI001222NCATS NIH HHS U01 TR001263NCATS NIH HHS UG3 TR003908NCI NIH HHS P30 CA008748NCI NIH HHS P30 CA042014NCI NIH HHS U24 CA076518NHLBI NIH HHS U01 HL069294NHLBI NIH HHS U10 HL069254NHLBI NIH HHS UG1 HL069254NIAID NIH HHS R13 AI094943NIAID NIH HHS R21 AI156583NIAID NIH HHS U01 AI126612NIAID NIH HHS U54 AI082973NINDS NIH HHS U54 NS064808
Abstract
backgroundSevere combined immunodeficiency (SCID) is fatal unless durable adaptive immunity is established, most commonly through allogeneic haematopoietic cell transplantation (HCT). The Primary Immune Deficiency Treatment Consortium (PIDTC) explored factors affecting the survival of individuals with SCID over almost four decades, focusing on the effects of population-based newborn screening for SCID that was initiated in 2008 and expanded during 2010-18.
methodsWe analysed transplantation-related data from children with SCID treated at 34 PIDTC sites in the USA and Canada, using the calendar time intervals 1982-89, 1990-99, 2000-09, and 2010-18. Categorical variables were compared by χ
findingsFor 902 children with confirmed SCID, 5-year overall survival remained unchanged at 72%-73% for 28 years until 2010-18, when it increased to 87% (95% CI 82·1-90·6; n=268; p=0·0005). For children identified as having SCID by newborn screening since 2010, 5-year overall survival was 92·5% (95% CI 85·8-96·1), better than that of children identified by clinical illness or family history in the same interval (79·9% [69·5-87·0] and 85·4% [71·8-92·8], respectively [p=0·043]). Multivariable analysis demonstrated that the factors of active infection (hazard ratio [HR] 2·41, 95% CI 1·56-3·72; p<0·0001), age 3·5 months or older at HCT (2·12, 1·38-3·24; p=0·001), Black or African-American race (2·33, 1·56-3·46; p<0·0001), and certain SCID genotypes to be associated with lower overall survival during all time intervals. Moreover, after adjusting for several factors in this multivariable analysis, HCT after 2010 no longer conveyed a survival advantage over earlier time intervals studied (HR 0·73, 95% CI 0·43-1·26; p=0·097). This indicated that younger age and freedom from infections at HCT, both directly driven by newborn screening, were the main drivers for recent improvement in overall survival.
interpretationPopulation-based newborn screening has facilitated the identification of infants with SCID early in life, in turn leading to prompt HCT while avoiding infections. Public health programmes worldwide can benefit from this definitive demonstration of the value of newborn screening for SCID.
fundingNational Institute of Allergy and Infectious Diseases, Office of Rare Diseases Research, and National Center for Advancing Translational Sciences.
Indexed as
Graft vs Host DiseaseHematopoietic Stem Cell TransplantationSevere Combined ImmunodeficiencyHumansInfant, NewbornLongitudinal StudiesNeonatal ScreeningProportional Hazards Models
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