ArticleThe Journal of allergy and clinical immunology2024
Posttransplantation late complications increase over time for patients with SCID: A Primary Immune Deficiency Treatment Consortium (PIDTC) landmark study.
Open access · greenAbstract readMulticenter Study
In one paragraph
Article in The Journal of allergy and clinical immunology, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 9 papers, 1 of them a synthesis that pooled it.
0numbers the graph read from it
0cells of the map it votes in
9citing papers in PubMed, 1 pooled it
2.5field-weighted citation impact, top 10% of its field
What it found
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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.
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Who cites it
9 citing papers in PubMed, 1 synthesis or guideline pooled it, 15 citations in OpenAlex.
- Late effects after allogeneic hematopoietic stem cell transplantation in patients with primary immunodeficiency.Frontiers in immunology · 2026Pooled it
- IMPACT (Immune Monitoring and Phenotype Assessment of Clinical Trajectory) for Patients With Inborn Errors of Immunity From the Primary Immune Deficiency Treatment Consortium (PIDTC).The journal of allergy and clinical immunology. In practice · 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
- Demographic, clinical, and immunological features in combined immunodeficiency patients: a comparative analysis of those with and without pulmonary manifestations - a multicenter study from Iran.BMC pulmonary medicine · 2026Article
- Neurodevelopmental outcomes following hematopoietic cell transplantation for patients with severe combined immunodeficiency (SCID): A PIDTC study.Journal of human immunity · 2026Article
- IL2RG-related immunodeficiencies: from SCID to atypical presentations.Frontiers in immunology · 2026Review
- Harmonized immune recovery monitoring after HCT: evidence and practical guidance from the Westhafen Intercontinental Group.Blood advances · 2025Review
- Haematopoietic stem cell transplant for inborn errors of immunity-Who and when?British journal of haematology · 2025Review
- First pilot study for newborn screening of severe T and B lymphopenias in ColombiaBiomedica : revista del Instituto Nacional de Salud · 2024Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
58 authors at 20 institutions in 2 countries.
Hesham EissaDivision of Pediatric Hematology-Oncology-BMT, University of Colorado, Aurora, Wash. Electronic address: Hesham.eissa@cuanschutz.edu.
Monica S ThakarFred Hutchinson Cancer Center, Seattle, Wash; Department of Pediatrics, University of Washington, Seattle, Wash.
Ami J ShahPediatrics [Hematology/Oncology/Stem Cell Transplantation and Regenerative Medicine], Stanford University/Lucille Packard Children's Hospital, Palo Alto, Calif.
Brent R LoganDivision of Biostatistics, Medical College of Wisconsin, Milwaukee, Wis.
Linda M GriffithDivision of Allergy, Immunology and Transplantation, National Institute of Allergy and Infectious Diseases (NIAID), National Institutes of Health (NIH), Bethesda, Md.
Huaying DongDivision of Biostatistics, Medical College of Wisconsin, Milwaukee, Wis.
Roberta E ParrottDuke University Medical Center, Durham, NC.
Richard J O'ReillyDepartment of Pediatrics, Bone Marrow Transplant Service, Memorial Sloan Kettering Cancer Center, New York, NY.
Jasmeen DaraDivision of 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, Calif.
Neena KapoorDivision of Hematology, Oncology and Blood and Marrow Transplant, Children's Hospital Los Angeles, Los Angeles, Calif.
Lisa Forbes SatterImmunology, Allergy, and Rheumatology, Baylor College of Medicine, Texas Children's Hospital, Houston, Tex.
Sharat ChandraDivision of Bone Marrow Transplantation and Immune Deficiency, Cincinnati Children's Hospital Medical Center, Cincinnati, Ohio; Department of Pediatrics, University of Cincinnati School of Medicine, Cincinnati, Ohio.
Malika KapadiaBoston Children's Hospital, Dana-Farber Cancer Institute, Boston, Mass.
Shanmuganathan ChandrakasanBone Marrow Transplantation and Immune Deficiency, Children's Healthcare of Atlanta, Atlanta, Ga.
Alan KnutsenSt Louis University, Cardinal Glennon Children's Hospital, St Louis, Mo.
Soma C JyonouchiDivision of Allergy and Immunology, Children's Hospital of Philadelphia, Philadelphia, Pa; Department of Pediatrics, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, Pa.
Lyndsay MolinariMethodist Children's Hospital of South Texas, San Antonio, Tex.
Ahmad RayesDivision of Hematology, Oncology, Transplantation, and Immunology, Primary Children's Hospital, Huntsman Cancer Institute, Spense Fox Eccles School of Medicine at the University of Utah, Salt Lake City, Utah.
Christen L EbensDivision of Pediatric Blood and Marrow Transplant and Cellular Therapy, University of Minnesota Masonic Children's Hospital, Minneapolis, Minn.
Pierre TeiraPaediatric Haematology Oncology, Ste-Justine Hospital, Montreal, Canada.
Blachy J Dávila SaldañaChildren's National Hospital, Washington, DC.
Lauri M BurroughsFred Hutchinson Cancer Center, Seattle, Wash; Department of Pediatrics, University of Washington, Seattle, Wash.
Sonali ChaudhuryHematology, Oncology, Neuro-oncology & Stem Cell Transplantation Division, Ann & Robert H. Lurie Children's Hospital of Chicago, Chicago, Ill.
Deepak ChellapandianCenter for Cell and Gene Therapy for Non-malignant Conditions, Johns Hopkins All Children's Hospital, St Petersburg, Fla.
Alfred P GillioChildren's Cancer Institute, Hackensack University Medical Center, Hackensack, NJ.
Fredrick GoldmanDivision of Pediatric Hematology and Oncology and Bone Marrow Transplant, University of Alabama at Birmingham, Birmingham, Ala.
Harry L MalechGenetic Immunotherapy Section, NIAID, NIH, Bethesda, Md.
Kenneth DeSantesDivision of Pediatric Hematology-Oncology & Bone Marrow Transplant, University of Wisconsin, American Family Children's Hospital, Madison, Wis.
Geoff D E CuvelierManitoba Blood and Marrow Transplant Program, CancerCare Manitoba, Winnipeg, Canada.
Jacob RozmusChildren's & Women's Health Centre of British Columbia, Vancouver, Canada.
Ralph QuinonesDivision of Pediatric Hematology-Oncology-BMT, University of Colorado, Aurora, Wash.
Lolie C YuDivision of Heme-Onc/HSCT, Children's Hospital/LSUHSC, New Orleans, La.
Larisa BroglieDepartment of Pediatrics, Division of Pediatric Hematology, Oncology, and Blood and Marrow Transplantation, Medical College of Wisconsin, Milwaukee, Wis.
Victor AquinoDivision of Pediatric Hematology and Oncology, The University of Texas Southwestern Medical Center, Dallas, Tex.
Evan ShereckDivision of Pediatric Hematology/Oncology, Oregon Health and Science University, Portland, Ore.
Theodore B MooreDepartment of Pediatric Hematology-Oncology, Mattel Children's Hospital, University of California, Los Angeles, Calif.
Mark T Vander LugtBlood and Marrow Transplant Program, University of Michigan, Ann Arbor, Mich.
Talal I MousallemDuke University Medical Center, Durham, NC.
Joeseph H OvedDepartment of Pediatrics, Bone Marrow Transplant Service, Memorial Sloan Kettering Cancer Center, New York, NY.
Morna DorseyDivision of 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, Calif.
Hisham Abdel-AzimDivision of Hematology, Oncology and Blood and Marrow Transplant, Children's Hospital Los Angeles, Los Angeles, Calif; Loma Linda University School of Medicine, Cancer Center, Children Hospital and Medical Center, Loma Linda, Calif.
Caridad MartinezCenter for Cell and Gene Therapy, Baylor College of Medicine, Texas Children's Hospital, Houston, Tex.
Jacob H BleesingDivision of Bone Marrow Transplantation and Immune Deficiency, Cincinnati Children's Hospital Medical Center, Cincinnati, Ohio; Department of Pediatrics, University of Cincinnati School of Medicine, Cincinnati, Ohio.
Susan ProckopBoston Children's Hospital, Dana-Farber Cancer Institute, Boston, Mass.
Donald B KohnUniversity of California, Los Angeles, Calif.
Jeffrey J BednarskiDepartment of Pediatrics, Washington University School of Medicine, St Louis, Mo.
Jennifer LeidingOrlando Health Arnold Palmer Hospital for Children, Orlando, Fla.
Rebecca A MarshDivision of Bone Marrow Transplantation and Immune Deficiency, Cincinnati Children's Hospital Medical Center, Cincinnati, Ohio; Department of Pediatrics, University of Cincinnati School of Medicine, Cincinnati, Ohio.
Troy TorgersonAllen Institute for Immunology, Seattle, Wash.
Luigi D NotarangeloLaboratory of Clinical Immunology and Microbiology, NIAID, NIH, Bethesda, Md.
Sung-Yun PaiCenter for Cancer Research, National Cancer Institute, NIH, Bethesda, Md.
Michael A PulsipherDivision of Hematology, Oncology, Transplantation, and Immunology, Primary Children's Hospital, Huntsman Cancer Institute, Spense Fox Eccles School of Medicine at the University of Utah, Salt Lake City, Utah.
Jennifer M PuckDivision of 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, Calif.
Christopher C DvorakDivision of 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, Calif.
Elie HaddadDepartment of Pediatrics and the Department of Microbiology, Immunology, and Infectious Diseases, University of Montreal, CHU Sainte-Justine, Montreal, Canada.
Rebecca H BuckleyDuke University Medical Center, Durham, NC.
Morton J CowanDivision of 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, Calif.
Jennifer HeimallDivision of Allergy and Immunology, Children's Hospital of Philadelphia, Philadelphia, Pa; Department of Pediatrics, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, Pa.
Duke Medical Center · USMedical College of Wisconsin · USUniversity of California, San Francisco · USBaylor College of Medicine · USCentre Hospitalier Universitaire Sainte-Justine · CAMemorial Sloan Kettering Cancer Center · USNational Institutes of Health · USUCSF Benioff Children's Hospital · USUniversity of Cincinnati · USUniversity of Colorado Anschutz Medical Campus · USUniversity of Washington · USAllen Institute for Immunology · USArnold Palmer Hospital for Children · USBoston Children's Hospital · USCancerCare Manitoba · CACardinal Glennon Children’s Medical Center · USCenter for Cancer Research · USChildren's Healthcare of Atlanta · USChildren's Hospital of Los Angeles · USChildren's Hospital of Philadelphia · US
Funding
UTAH REGIONAL CANCER CENTERP30CA042014 · NCI · UTAH STATE HIGHER EDUCATION SYSTEM--UNIVERSITY OF UTAH · PI KATHARINE S ULLMAN · 1986 to 2026
$72.6MUniversity of Minnesota Clinical and Translational Science Institute (UMN CTSI)UL1TR002494 · NCATS · UNIVERSITY OF MINNESOTA · PI BLAZAR, BRUCE R, WEISDORF, DANIEL J · 2018 to 2022
$34.9MRare 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 MALECH, HARRY · 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
$15.2MGene Therapy Basic Research to Treat Inherited Primary Immune DeficienciesZIAAI000644 · NIAID · NATIONAL INSTITUTE OF ALLERGY AND INFECTIOUS DISEASES · PI MALECH, HARRY L · 2009 to 2025
$12.1MRandomized 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
$8.5MInstitutional Career Development CoreKL2TR002492 · NCATS · UNIVERSITY OF MINNESOTA · PI INGBAR, DAVID H · 2018 to 2022
$6.3MUnrelated 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
$1.3MBlood and Marrow Transplant Clinical Trials NetworkU10HL069254 · NHLBI · UNIVERSITY OF UTAH · PI PULSIPHER, MICHAEL A · 2011 to 2016
$839kIntramural NIH HHS ZIA AI001222NCATS NIH HHS KL2 TR002492NCATS NIH HHS U01 TR001263NCATS NIH HHS UL1 TR002494NCI NIH HHS P30 CA042014NHLBI NIH HHS U10 HL069254NHLBI NIH HHS UG1 HL069254NIAID NIH HHS U01 AI126612NIAID NIH HHS U54 AI082973NINDS NIH HHS U54 NS064808
Abstract
backgroundThe Primary Immune Deficiency Treatment Consortium (PIDTC) enrolled children in the United States and Canada onto a retrospective multicenter natural history study of hematopoietic cell transplantation (HCT).
objectiveWe investigated outcomes of HCT for severe combined immunodeficiency (SCID).
methodsWe evaluated the chronic and late effects (CLE) after HCT for SCID in 399 patients transplanted from 1982 to 2012 at 32 PIDTC centers. Eligibility criteria included survival to at least 2 years after HCT without need for subsequent cellular therapy. CLE were defined as either conditions present at any time before 2 years from HCT that remained unresolved (chronic), or new conditions that developed beyond 2 years after HCT (late).
resultsThe cumulative incidence of CLE was 25% in those alive at 2 years, increasing to 41% at 15 years after HCT. CLE were most prevalent in the neurologic (9%), neurodevelopmental (8%), and dental (8%) categories. Chemotherapy-based conditioning was associated with decreased-height z score at 2 to 5 years after HCT (P < .001), and with endocrine (P < .001) and dental (P = .05) CLE. CD4 count of ≤500 cells/μL and/or continued need for immunoglobulin replacement therapy >2 years after transplantation were associated with lower-height z scores. Continued survival from 2 to 15 years after HCT was 90%. The presence of any CLE was associated with increased risk of late death (hazard ratio, 7.21; 95% confidence interval, 2.71-19.18; P < .001).
conclusionLate morbidity after HCT for SCID was substantial, with an adverse impact on overall survival. This study provides evidence for development of survivorship guidelines based on disease characteristics and treatment exposure for patients after HCT for SCID.
Indexed as
Hematopoietic Stem Cell TransplantationSevere Combined ImmunodeficiencyCanadaChildHumansIncidenceRetrospective StudiesTransplantation Conditioningbone marrow transplantationHCTlate effectsSCIDsurvivorship
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