ArticleThe Journal of allergy and clinical immunology2014
Primary Immune Deficiency Treatment Consortium (PIDTC) report.
5 registry-linked trialsOpen access · hybridAbstract read
In one paragraph
Article in The Journal of allergy and clinical immunology, 2014. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to 5 registered trials, which are not on this map. Cited by 24 papers, 1 of them a synthesis that pooled it.
0numbers the graph read from it
0cells of the map it votes in
24citing papers in PubMed, 1 pooled it
4.4field-weighted citation impact, top 5% of its field
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.
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.
A Prospective Natural History Study of Diagnosis, Treatment and Outcomes of Children With SCID Disorders (RDCRN PIDTC-6901)
A Retrospective and Cross-Sectional Analysis of Patients Treated for SCID Since January 1,1968 (RDCRN PIDTC-6902)
Analysis of Patients Treated for Wiskott-Aldrich Syndrome Since January 1, 1990 (RDCRN PIDTC-6904)
Analysis of Patients Treated for Chronic Granulomatous Disease Since January 1, 1995
NCT03538899 phase1 / phase2recruitingnot on this mapstarted 2018, after this paper: background citation
Phase I/II Safety and Efficacy Study of Gene Transfer for Artemis-Deficient Severe Combined Immunodeficiency (ART-SCID) in Newly Diagnosed Patients Using Self-Inactivating Lentiviral Vector (AProArt) to Transduce Autologous CD34 Hematopoietic Cells
Who cites it
24 citing papers in PubMed, 1 synthesis or guideline pooled it, 85 citations in OpenAlex.
- Overview of next-generation sequencing to the molecular diagnosis of inborn errors of immunity in Brazil: a systematic review.Frontiers in immunology · 2026Pooled it
- Posttransplantation late complications increase over time for patients with SCID: A Primary Immune Deficiency Treatment Consortium (PIDTC) landmark study.The Journal of allergy and clinical immunology · 2024Article
- Article
- Genetic screening in a Brazilian cohort with inborn errors of immunity.BMC genomic data · 2023Article
- Gene therapy in PIDs, hemoglobin, ocular, neurodegenerative, and hemophilia B disorders.Open life sciences · 2021Review
- Lower T Regulatory and Th17 Cell Populations Predicted by RT-PCR-AmplifiedFrontiers in immunology · 2020Article
- Multicenter Outcome of Hematopoietic Stem Cell Transplantation for Primary Immune Deficiency Disorders in India.Frontiers in immunology · 2020Article
- Humoral immunodeficiencies: conferred risk of infections and benefits of immunoglobulin replacement therapy.Transfusion · 2018Review
- Transplantation of Hematopoietic Stem Cells for Primary Immunodeficiencies in Brazil: Challenges in Treating Rare Diseases in Developing Countries.Journal of clinical immunology · 2018Article
- Prevalence of Granulomas in Patients With Primary Immunodeficiency Disorders, United States: Data From National Health Care Claims and the US Immunodeficiency Network Registry.Journal of clinical immunology · 2018Article
- Quality of Life, Treatment Beliefs, and Treatment Satisfaction in Children Treated for Primary Immunodeficiency with SCIg.Journal of clinical immunology · 2017Article
- Protein Kinase C δ: a Gatekeeper of Immune Homeostasis.Journal of clinical immunology · 2016Review
- The Primary Immune Deficiency Treatment Consortium: how can it improve definitive therapy for PID?Expert review of clinical immunology · 2016Article
- Primary Immune Deficiency Treatment Consortium (PIDTC) update.The Journal of allergy and clinical immunology · 2016Review
- Deletion of Wiskott-Aldrich syndrome protein triggers Rac2 activity and increased cross-presentation by dendritic cells.Nature communications · 2016Article
- Flow Cytometry, a Versatile Tool for Diagnosis and Monitoring of Primary Immunodeficiencies.Clinical and vaccine immunology : CVI · 2016Review
- Clinical development of gene therapy: results and lessons from recent successes.Molecular therapy. Methods & clinical development · 2016Review
- Late-Onset Combined Immunodeficiency with a Novel IL2RG Mutation and Probable Revertant Somatic Mosaicism.Journal of clinical immunology · 2015Article
- Longitudinal Evaluation of Immune Reconstitution and B-cell Function After Hematopoietic Cell Transplantation for Primary Immunodeficiency.Journal of clinical immunology · 2015Article
- Hematopoietic Stem Cell Transplantation for Severe Combined Immunodeficiency.Current pediatrics reports · 2015Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
53 authors at 20 institutions in 5 countries.
Linda M GriffithDivision of Allergy, Immunology and Transplantation, National Institute of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, Md. Electronic address: LGriffith@niaid.nih.gov.
Morton J CowanDivision of Allergy/Immunology and Blood and Marrow Transplantation, Department of Pediatrics and UCSF Benioff Children's Hospital, University of California San Francisco, San Francisco, Calif.
Luigi D NotarangeloDivision of Immunology, the Manton Center for Orphan Disease Research, Children's Hospital, and Harvard Stem Cell Institute, Harvard Medical School, Boston, Mass.
Donald B KohnDepartments of Microbiology, Immunology and Molecular Genetics, University of California Los Angeles, Los Angeles, Calif.
Jennifer M PuckDivision of Allergy/Immunology and Blood and Marrow Transplantation, Department of Pediatrics and UCSF Benioff Children's Hospital, University of California San Francisco, San Francisco, Calif; Institute for Human Genetics, University of California San Francisco, San Francisco, Calif.
Sung-Yun PaiPediatric Hematology/Oncology, Children's Hospital, Harvard Medical School, Boston, Mass.
Barbara BallardImmune Deficiency Foundation, Towson, Md.
Sarah C BauerDevelopmental and Behavioral Pediatrics, Lurie Children's Hospital of Chicago, Northwestern Feinberg School of Medicine, Chicago, Ill.
Jack J H BleesingDepartment of Pediatrics, Cincinnati Children's Hospital Medical Center, Cincinnati, Ohio.
Marcia BoyleImmune Deficiency Foundation, Towson, Md.
Amy BrowerNewborn Screening Translational Research Network, American College of Medical Genetics and Genomics, Bethesda, Md.
Rebecca H BuckleyPediatric Allergy and Immunology, Duke University School of Medicine, Durham, NC.
Mirjam van der BurgMolecular Immunology, Erasmus Medical Center, Rotterdam, The Netherlands.
Lauri M BurroughsPediatric Hematology/Oncology, Fred Hutchinson Cancer Research Center, University of Washington School of Medicine, Seattle, Wash.
Fabio CandottiGenetics & Molecular Biology Branch, National Human Genome Research Institute, National Institutes of Health, Bethesda, Md.
Andrew J CantPediatric Immunology and Infectious Diseases and Pediatric Bone Marrow Transplant, Newcastle General Hospital, Newcastle upon Tyne, United Kingdom.
Talal ChatilaPediatric Allergy/Immunology, Children's Hospital, Harvard Medical School, Boston, Mass.
Charlotte Cunningham-RundlesPediatric Clinical Immunology, Mount Sinai School of Medicine, New York, NY.
Mary C DinauerPediatric Hematology/Oncology, Washington University School of Medicine, St Louis, Mo.
Christopher C DvorakDivision of Allergy/Immunology and Blood and Marrow Transplantation, Department of Pediatrics and UCSF Benioff Children's Hospital, University of California San Francisco, San Francisco, Calif.
Alexandra H FilipovichPediatric Clinical Immunology, Division of Hematology/Oncology, Cincinnati Children's Hospital Medical Center, Cincinnati, Ohio.
Thomas A FleisherDepartment of Laboratory Medicine, Clinical Center, National Institutes of Health, Bethesda, Md.
Hubert Bobby GasparPediatric Immunology, Center for Immunodeficiency, Institute of Child Health, Great Ormond Street Hospital, University College London, London, United Kingdom.
Tayfun GungorPediatric Immunology and Blood and Marrow Transplantation, Children's Hospital Zurich, University of Zurich, Zurich, Switzerland.
Elie HaddadPediatric Immunology, Mother and Child Ste-Justine Hospital, Montreal, Quebec, Canada.
Emily HovermaleImmune Deficiency Foundation, Towson, Md.
Faith HuangPediatric Allergy/Immunology, Mount Sinai Medical Center, New York, NY.
Alan HurleyChronic Granulomatous Disease Association, San Marino, Calif.
Mary HurleyChronic Granulomatous Disease Association, San Marino, Calif.
Sumathi IyengarWiskott-Aldrich Foundation, Smyrna, Ga.
Elizabeth M KangLaboratory of Host Defenses, National Institute of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, Md.
Brent R LoganCenter for International Blood and Marrow Transplant Research and Division of Biostatistics, Medical College of Wisconsin, Milwaukee, Wis.
Janel R Long-BoyleDepartment of Clinical Pharmacy, School of Pharmacy, University of California, San Francisco, Calif.
Harry L MalechLaboratory of Host Defenses, National Institute of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, Md.
Sean A McGheePediatric Allergy/Immunology, Lucile Packard Children's Hospital, Stanford University Medical Center, Stanford, Calif.
Fred ModellJeffrey Modell Foundation, New York, NY.
Vicki ModellJeffrey Modell Foundation, New York, NY.
Hans D OchsCenter for Immunity and Immunotherapy, Seattle Children's Hospital Research Institute, University of Washington School of Medicine, Seattle, Wash.
Richard J O'ReillyPediatrics and Immunology, Memorial Sloan Kettering Cancer Center, New York, NY.
Robertson ParkmanDivision of Research Immunology/B.M.T., Children's Hospital Los Angeles, Los Angeles, Calif.
David J RawlingsPediatric Immunology, Seattle Children's Research Institute, University of Washington School of Medicine, Seattle, Wash.
John M RoutesPediatric Allergy and Clinical Immunology, Children's Hospital of Wisconsin, Medical College of Wisconsin, Milwaukee, Wis.
William T ShearerPediatric Allergy & Immunology, Texas Children's Hospital, Baylor College of Medicine, Houston, Tex.
Trudy N SmallPediatric Bone Marrow Transplant Service, Memorial Sloan Kettering Cancer Center, New York, NY.
Heather SmithSCID Angels for Life Foundation, Lakeland, Fla.
Kathleen E SullivanPediatric Immunology, Children's Hospital of Philadelphia, Perelman School of Medicine, University of Pennsylvania, Philadelphia, Pa.
Paul SzabolcsBone Marrow Transplantation and Cellular Therapies, Children's Hospital of Pittsburgh, University of Pittsburgh Medical Center, Pittsburgh, Pa.
Adrian ThrasherPediatric Immunology, Center for Immunodeficiency, Institute of Child Health, Great Ormond Street Hospital, University College London, London, United Kingdom.
Troy R TorgersonPediatric Rheumatology, Seattle Children's Research Institute, University of Washington School of Medicine, Seattle, Wash.
Paul VeysBlood and Marrow Transplantation, Institute of Child Health, Great Ormond Street Hospital, London, United Kingdom.
Kenneth WeinbergPediatric Stem Cell Transplantation and Hematology/Oncology, Lucile Packard Children's Hospital, Stanford University School of Medicine, Stanford, Calif.
Juan Carlos Zuniga-PfluckerDepartment of Immunology, University of Toronto, Toronto, Ontario, Canada.
workshop participants
Harvard University · USImmune Deficiency Foundation · USUniversity of Washington · USCincinnati Children's Hospital Medical Center · USJeffrey Modell Foundation · USMemorial Sloan Kettering Cancer Center · USNational Institute of Allergy and Infectious Diseases · USUCSF Benioff Children's Hospital · USUniversity College London · GBUniversity of California, San Francisco · USAmerican College of Medical Genetics · USCentre Hospitalier Universitaire Sainte-Justine · CAChildren's Hospital of Los Angeles · USChildren's Hospital of Wisconsin · USDuke University · USErasmus MC · NLGreat Ormond Street Hospital · GBIcahn School of Medicine at Mount Sinai · USLurie Children's Hospital · USMedical College of Wisconsin · US
Funding
Virus & Reservoirs CoreP30AI045008 · NIAID · UNIVERSITY OF PENNSYLVANIA · PI ROBERT GROSS · 1999 to 2026
$78.6MVIROSOME/PROTEOLIPOSOME MEDIATED GENE TRANSFERP01HL053749 · NHLBI · ST. JUDE CHILDREN'S RESEARCH HOSPITAL · PI WEISS, MITCHELL J · 1994 to 2022
$43.6MTraining of Clinical Investigators in Primary Immune DeficienciesU54AI082973 · NIAID · UNIVERSITY OF CALIFORNIA, SAN FRANCISCO · PI PUCK, JENNIFER M. · 2009 to 2023
$23.3MData Management and Coordinating Center (DMCC)U54NS064808 · NINDS · UNIVERSITY OF SOUTH FLORIDA · PI KRISCHER, JEFFREY P · 2009 to 2013
$16.5MDevelopment and conduct of allogeneic stem cell transplant and autologous stem cell gene therapy for inherited immune deficienciesZIAAI000989 · NIAID · NATIONAL INSTITUTE OF ALLERGY AND INFECTIOUS DISEASES · PI KANG, ELIZABETH · 2009 to 2025
$12.4MGene Therapy Basic Research to Treat Inherited Primary Immune DeficienciesZIAAI000644 · NIAID · NATIONAL INSTITUTE OF ALLERGY AND INFECTIOUS DISEASES · PI MALECH, HARRY L · 2009 to 2025
$12.1MVector ProductionP01AI097100 · NIAID · SEATTLE CHILDREN'S HOSPITAL · PI KIEM, HANS-PETER · 2012 to 2016
$11.9MDevelopment and Clinical Application of Gene and Cell therapies for Patients with Immune DeficienciesZIAAI000645 · NIAID · NATIONAL INSTITUTE OF ALLERGY AND INFECTIOUS DISEASES · PI DE RAVIN, SUK SEE · 2009 to 2025
$10.1MBasic Research on Hematopoietic Human Stem CellsZIAAI000988 · NIAID · NATIONAL INSTITUTE OF ALLERGY AND INFECTIOUS DISEASES · PI MALECH, HARRY L · 2009 to 2019
$6.5MGene Therapy Approaches for Primary ImmunodeficienciesZIAHG000122 · NHGRI · NATIONAL HUMAN GENOME RESEARCH INSTITUTE · PI CANDOTTI, FABIO · 2009 to 2014
$6.5MTARGETING PRIMARY IMMUNODEFICIENCY AMONG MINORITIESR18AI048693 · NIAID · MOUNT SINAI SCHOOL OF MEDICINE OF NYU · PI CUNNINGHAM-RUNDLES, CHARLOTTE · 2000 to 2016
$6.2MGene Therapy Clinical Trials for X-linked Severe Combined ImmunodeficiencyZIAAI000992 · NIAID · NATIONAL INSTITUTE OF ALLERGY AND INFECTIOUS DISEASES · PI DE RAVIN, SUK · 2009 to 2019
$4.1MIntramural NIH HHS Z99 AI999999NHLBI NIH HHS P01 HL053749NIAID NIH HHS P01 AI097100NIAID NIH HHS P30 AI045008NIAID NIH HHS R13 AI094943NIAID NIH HHS R13-AI094943NIAID NIH HHS R18 AI048693NIAID NIH HHS U54 AI082973NIAID NIH HHS U54-AI082973NICHD NIH HHS K12 HD068322NINDS NIH HHS U54 NS064808NINDS NIH HHS U54-NS064808
Abstract
The Primary Immune Deficiency Treatment Consortium (PIDTC) is a network of 33 centers in North America that study the treatment of rare and severe primary immunodeficiency diseases. Current protocols address the natural history of patients treated for severe combined immunodeficiency (SCID), Wiskott-Aldrich syndrome, and chronic granulomatous disease through retrospective, prospective, and cross-sectional studies. The PIDTC additionally seeks to encourage training of junior investigators, establish partnerships with European and other International colleagues, work with patient advocacy groups to promote community awareness, and conduct pilot demonstration projects. Future goals include the conduct of prospective treatment studies to determine optimal therapies for primary immunodeficiency diseases. To date, the PIDTC has funded 2 pilot projects: newborn screening for SCID in Navajo Native Americans and B-cell reconstitution in patients with SCID after hematopoietic stem cell transplantation. Ten junior investigators have received grant awards. The PIDTC Annual Scientific Workshop has brought together consortium members, outside speakers, patient advocacy groups, and young investigators and trainees to report progress of the protocols and discuss common interests and goals, including new scientific developments and future directions of clinical research. Here we report the progress of the PIDTC to date, highlights of the first 2 PIDTC workshops, and consideration of future consortium objectives.
Indexed as
Immunologic Deficiency SyndromesHematopoietic Stem Cell TransplantationHumansInfant, NewbornNeonatal ScreeningPilot ProjectsSocieties, ScientificADAAdenosine deaminaseAllogeneic hematopoietic cell transplantationCenter for International Blood and Marrow Transplant ResearchCGDChronic granulomatous diseaseCIBMTRclinical trialEBMTESIDEuropean Group for Blood and Marrow TransplantationEuropean Society for Immunodeficienciesgene therapyGene therapyGraft-versus-host diseaseGTGVHDHCTHematopoietic cell transplantationIEWPInborn Errors Working PartyMACMatched unrelated donorMUDMyeloablative conditioningNational Institute of Allergy and Infectious DiseasesNational Institutes of HealthNatural killerNBSNewborn screening for SCIDNIAIDNIHNKPIDPIDTCPrimary Immune Deficiency Treatment Consortiumprimary immunodeficiencyPrimary immunodeficiencyReduced-intensity conditioningRICSCETIDESCIDSevere combined immunodeficiencyStem Cell Transplantation for Immunodeficiencies in EuropeUnited States Immunodeficiency NetworkUSIDNETWASWiskott-Aldrich syndrome
Identifiers
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Registered trials
NCT01186913ClinicalTrials.gov
NCT01346150ClinicalTrials.gov
NCT02064933ClinicalTrials.gov
NCT02082353ClinicalTrials.gov
NCT03538899ClinicalTrials.gov
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.