ArticleFrontiers in immunology2020
Hematopoietic Cell Transplantation in Patients With Primary Immune Regulatory Disorders (PIRD): A Primary Immune Deficiency Treatment Consortium (PIDTC) Survey.
Open access · goldAbstract read
In one paragraph
Article in Frontiers in immunology, 2020. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 40 papers, 2 of them syntheses that pooled it.
0numbers the graph read from it
0cells of the map it votes in
40citing papers in PubMed, 2 pooled it
4.5field-weighted citation impact, top 4% 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.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
40 citing papers in PubMed, 2 syntheses or guidelines pooled it, 80 citations in OpenAlex.
- Systematic literature reviews to identify epidemiological, clinical, economic and health-related quality of life evidence in activated PI3Kδ syndrome (APDS).BMC immunology · 2025Pooled it
- Genetic Etiologies and Outcomes in Malignancy and Mortality in Activated Phosphoinositide 3-Kinase Delta Syndrome: A Systematic Review.Advances in therapy · 2025Pooled it
- How I Treat: Transplantation for primary immune regulatory diseases and inflammatory disorders.Journal of human immunity · 2026Article
- TLR8 gain-of-function: on the highway to the danger zone.Blood advances · 2026Article
- Spectrum of Primary Immune Regulatory Disorders in Children in a Highly Consanguineous Population: Report from a National Registry.Journal of clinical immunology · 2026Article
- CAR T cell therapy for children with rheumatic disease: the time is now.Nature reviews. Rheumatology · 2025Review
- A hypomorphic Il2rb mutant mouse model recapitulates and reveals mechanisms of human T cell immune dysregulation in IL-2Rβ deficiency.Cell reports · 2025Article
- The Neuroimmune Axis in Gastrointestinal Disorders - An Underrecognized Problem.Current gastroenterology reports · 2025Review
- Multidisciplinary approach to treating complex immune dysregulation disorders: an adaptive model for institutional implementation.Frontiers in immunology · 2025Review
- Tregopathy in focus.Frontiers in immunology · 2025Article
- Immunodeficiency: Overview of primary immune regulatory disorders (PIRDs).Allergy and asthma proceedings · 2024Review
- Changing outcomes of stem cell transplantation in primary immunodeficiencies: Results from a tertiary-care charitable trust hospital in Mumbai.The journal of allergy and clinical immunology. Global · 2023Article
- Autoimmunity in Primary Immunodeficiencies (PID).Clinical reviews in allergy & immunology · 2023Review
- Alemtuzumab and CXCL9 levels predict likelihood of sustained engraftment after reduced-intensity conditioning HCT.Blood advances · 2023Article
- Separating the Wheat From the Chaff in Asthma and Bronchiectasis: The Saga Trajectory of a Patient With Adult-Onset RAG1 Deficiency.The journal of allergy and clinical immunology. In practice · 2023Article
- Impact of Genetic Diagnosis on the Outcome of Hematopoietic Stem Cell Transplant in Primary Immunodeficiency Disorders.Journal of clinical immunology · 2023Review
- Correcting inborn errors of immunity: From viral mediated gene addition to gene editing.Seminars in immunology · 2023Review
- Article
- Personalized hematopoietic stem cell transplantation for inborn errors of immunity.Frontiers in immunology · 2023Review
- Treg in inborn errors of immunity: gaps, knowns and future perspectives.Frontiers in immunology · 2023Review
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
60 authors at 20 institutions in 5 countries.
Alice Y ChanDivision of Pediatric Allergy, Immunology, BMT, Benioff Children's Hospital, University of California, San Francisco, San Francisco, CA, United States.
Jennifer W LeidingDepartment of Pediatrics, Johns Hopkins All Children's Hospital, University of South Florida, St. Petersburg, FL, United States.
Xuerong LiuDivision of Biostatistics, Medical College of Wisconsin, Milwaukee, WI, United States.
Brent R LoganDivision of Biostatistics, Medical College of Wisconsin, Milwaukee, WI, United States.
Lauri M BurroughsDepartment of Pediatrics, Fred Hutchinson Cancer Research Center, Seattle Children's Hospital, University of Washington School of Medicine, Seattle, WA, United States.
Eric J AllenspachDepartment of Pediatrics, Seattle Children's Hospital, University of Washington School of Medicine, Seattle, WA, United States.
Suzanne Skoda-SmithDepartment of Pediatrics, Seattle Children's Hospital, University of Washington School of Medicine, Seattle, WA, United States.
Gulbu UzelLaboratory of Clinical Infectious Diseases, National Institute of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, MD, United States.
Luigi D NotarangeloLaboratory of Clinical Infectious Diseases, National Institute of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, MD, United States.
Mary SlatterPrimary Immunodeficiency Group, Paediatric Immunology and Haematopoietic Stem Cell Transplantation, Translational and Clinical Research Institute, Great North Childrens' Hospital, Newcastle University, Newcastle upon Tyne, United Kingdom.
Andrew R GenneryPrimary Immunodeficiency Group, Paediatric Immunology and Haematopoietic Stem Cell Transplantation, Translational and Clinical Research Institute, Great North Childrens' Hospital, Newcastle University, Newcastle upon Tyne, United Kingdom.
Angela R SmithPediatric Blood and Marrow Transplant, University of Minnesota, Minneapolis, MN, United States.
Sung-Yun PaiDivision of Pediatric Hematology-Oncology, Boston Children's Hospital, Boston, MA, United States.
Michael B JordanDivision of Bone Marrow Transplantation and Immune Deficiency, Department of Pediatrics, Cincinnati Children's Hospital Medical Center, University of Cincinnati, Cincinnati, OH, United States.
Rebecca A MarshDivision of Bone Marrow Transplantation and Immune Deficiency, Department of Pediatrics, Cincinnati Children's Hospital Medical Center, University of Cincinnati, Cincinnati, OH, United States.
Morton J CowanDivision of Pediatric Allergy, Immunology, BMT, Benioff Children's Hospital, University of California, San Francisco, San Francisco, CA, United States.
Christopher C DvorakDivision of Pediatric Allergy, Immunology, BMT, Benioff Children's Hospital, University of California, San Francisco, San Francisco, CA, United States.
John A CraddockTexas Children's Cancer Center, Center for Cell and Gene Therapy, Baylor College of Medicine, Houston, TX, United States.
Susan E ProckopStem Cell Transplant and Cellular Therapy Service, Department of Pediatrics, Memorial Sloan Kettering Cancer Center, New York, NY, United States.
Shanmuganathan ChandrakasanDivision of Bone Marrow Transplant, Aflac Cancer and Blood Disorders Center, Children's Healthcare of Atlanta, Emory University School of Medicine, Atlanta, GA, United States.
Neena KapoorSection of Transplantation and Cellular Therapy, Cancer and Blood Disease Institute, Keck School of Medicine, Children's Hospital Los Angeles, University of Southern California, Los Angeles, CA, United States.
Rebecca H BuckleyDepartments of Pediatrics and Immunology, Duke University School of Medicine, Durham, NC, United States.
Suhag ParikhDepartments of Pediatrics and Immunology, Duke University School of Medicine, Durham, NC, United States.
Deepak ChellapandianCancer and Blood Disorders Institute, Blood and Marrow Transplant Program, Johns Hopkins All Children's Hospital, St. Petersburg, FL, United States.
Benjamin R OshrineCancer and Blood Disorders Institute, Blood and Marrow Transplant Program, Johns Hopkins All Children's Hospital, St. Petersburg, FL, United States.
Jeffrey J BednarskiDepartment of Pediatrics, Washington University School of Medicine, St. Louis, MO, United States.
Megan A CooperDepartment of Pediatrics, Washington University School of Medicine, St. Louis, MO, United States.
Shalini ShenoyDepartment of Pediatrics, Washington University School of Medicine, St. Louis, MO, United States.
Blachy J Davila SaldanaDivision of Blood and Marrow Transplantation, Children's National Health System, George Washington University School of Medicine and Health Sciences, Washington, DC, United States.
Lisa R ForbesDepartment of Pediatrics, Immunology, Allergy, and Retrovirology Baylor College of Medicine, Texas Children's Hospital William T. Shearer Center for Human Immunobiology, Houston, TX, United States.
Caridad MartinezCenter for Cell and Gene Therapy, Baylor College of Medicine, Texas Children's Hospital Cancer Center, Houston, TX, United States.
Elie HaddadDepartment of Pediatrics, University of Montreal, Montreal, QC, Canada.
David C ShyrDepartment of Pediatrics, University of Utah School of Medicine, Salt Lake City, UT, United States.
Karin ChenDepartment of Pediatrics, University of Utah School of Medicine, Salt Lake City, UT, United States.
Kathleen E SullivanChildren's Hospital of Philadelphia, Perelman School of Medicine at University of Pennsylvania, Philadelphia, PA, United States.
Jennifer HeimallChildren's Hospital of Philadelphia, Perelman School of Medicine at University of Pennsylvania, Philadelphia, PA, United States.
Nicola WrightDepartment of Pediatrics, Alberta Children's Hospital, University of Calgary, Calgary, AB, Canada.
Monica BhatiaPediatric Stem Cell Transplantation, Columbia University College of Physicians and Surgeons, New York, NY, United States.
Geoffrey D E CuvelierManitoba Blood and Marrow Transplant Program, CancerCare Manitoba, University of Manitoba, Winnipeg, MB, Canada.
Frederick D GoldmanDepartment of Pediatrics, University of Alabama at Birmingham, Birmingham, AL, United States.
Isabelle MeytsLaboratory of Inborn Errors of Immunity, Department of Immunology, Microbiology and Transplantation, KU Leuven, Leuven, Belgium.
Holly K MillerPhoenix Children's Hospital, Phoenix, AZ, United States.
Markus G SeidelResearch Unit for Pediatric Hematology and Immunology, Department of Pediatrics and Adolescent Medicine, Medical University Graz, Graz, Austria.
Mark T Vander LugtDepartment of Pediatrics, University of Michigan, Ann Arbor, MI, United States.
Rosa BacchettaDivision of Stem Cell Transplantation and Regenerative Medicine, Department of Pediatrics, Stanford School of Medicine, Stanford, CA, United States.
Katja G WeinachtDivision of Stem Cell Transplantation and Regenerative Medicine, Department of Pediatrics, Stanford School of Medicine, Stanford, CA, United States.
Jeffrey R AndolinaDepartment of Pediatrics, Golisano Children's Hospital, University of Rochester Medical Center, Rochester, NY, United States.
Emi CaywoodNemours/Alfred I duPont Hospital for Children, Wilmington, DE, United States.
Hey ChongUPMC Children's Hospital of Pittsburgh, Pittsburgh, PA, United States.
Maria Teresa de la MorenaDepartment of Pediatrics, Seattle Children's Hospital, University of Washington School of Medicine, Seattle, WA, United States.
Victor M AquinoDepartment of Pediatrics, University of Texas Southwestern Medical Center Dallas, Dallas, TX, United States.
Evan ShereckDepartment of Pediatrics, Oregon Health & Science University, Portland, OR, United States.
Jolan E WalterDivision of Allergy and Immunology, Department of Pediatrics, Morsani College of Medicine, University of South Florida, St. Petersburg, FL, United States.
Morna J DorseyDivision of Pediatric Allergy, Immunology, BMT, Benioff Children's Hospital, University of California, San Francisco, San Francisco, CA, United States.
Christine M SeroogyDepartment of Pediatrics, University of Wisconsin School of Medicine and Public Health, Madison, WI, United States.
Linda M GriffithDivision of Allergy, Immunology and Transplantation, National Institute of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, MD, United States.
Donald B KohnDepartment of Pediatrics, David Geffen School of Medicine at University of California, Los Angeles, CA, United States.
Jennifer M PuckDivision of Pediatric Allergy, Immunology, BMT, Benioff Children's Hospital, University of California, San Francisco, San Francisco, CA, United States.
Michael A PulsipherSection of Transplantation and Cellular Therapy, Cancer and Blood Disease Institute, Keck School of Medicine, Children's Hospital Los Angeles, University of Southern California, Los Angeles, CA, United States.
Troy R TorgersonAllen Institute for Immunology and Department of Pediatrics, University of Washington, Seattle, WA, United States.
University of Washington · USJohns Hopkins University · USUniversity of California, San Francisco · USWashington University in St. Louis · USChildren's Cancer Center · USCincinnati Children's Hospital Medical Center · USDuke University · USMedical College of Wisconsin · USNational Institutes of Health · USStanford Medicine · USUCSF Benioff Children's Hospital · USUniversity of Pennsylvania · USUniversity of Southern California · USUniversity of Utah · USAlberta Children's Hospital · CAAlfred I. duPont Hospital for Children · USBaylor College of Medicine · USCancerCare Manitoba · CAChildren's Healthcare of Atlanta · USChildren's Hospital of Pittsburgh · 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
$105.2MPB 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
$15.2MBlood and Marrow Transplant Clinical Trials NetworkU10HL069254 · NHLBI · UNIVERSITY OF UTAH · PI PULSIPHER, MICHAEL A · 2011 to 2016
$839kTargeted non-genotoxic hematopoietic stem cell transplant conditioning approachK08HL141635 · NHLBI · EMORY UNIVERSITY · PI CHANDRAKASAN, SHANMUGANATHAN · 2019 to 2023
$815kPrimary Immune Deficiency Treatment Consortium Annual Scientific MeetingR13AI094943 · NIAID · UNIVERSITY OF CALIFORNIA, SAN FRANCISCO · PI PUCK, JENNIFER M. · 2011 to 2025
$239kNewborn Screening for SCID in a High-Risk PopulationR03HD060311 · NICHD · UNIVERSITY OF CALIFORNIA, SAN FRANCISCO · PI PUCK, JENNIFER M. · 2008 to 2009
$155kNCATS NIH HHS U01 TR001263NCI NIH HHS P30 CA008748NCI NIH HHS U24 CA076518NHLBI NIH HHS K08 HL141635NHLBI NIH HHS U01 HL069294NHLBI NIH HHS U10 HL069254NIAID NIH HHS R13 AI094943NIAID NIH HHS U54 AI082973NICHD NIH HHS R03 HD060311NINDS NIH HHS U54 NS064808
Abstract
Primary Immune Regulatory Disorders (PIRD) are an expanding group of diseases caused by gene defects in several different immune pathways, such as regulatory T cell function. Patients with PIRD develop clinical manifestations associated with diminished and exaggerated immune responses. Management of these patients is complicated; oftentimes immunosuppressive therapies are insufficient, and patients may require hematopoietic cell transplant (HCT) for treatment. Analysis of HCT data in PIRD patients have previously focused on a single gene defect. This study surveyed transplanted patients with a phenotypic clinical picture consistent with PIRD treated in 33 Primary Immune Deficiency Treatment Consortium centers and European centers. Our data showed that PIRD patients often had immunodeficient and autoimmune features affecting multiple organ systems. Transplantation resulted in resolution of disease manifestations in more than half of the patients with an overall 5-years survival of 67%. This study, the first to encompass disorders across the PIRD spectrum, highlights the need for further research in PIRD management.
Indexed as
Hematopoietic Stem Cell TransplantationAdolescentAdultAnimalsChildChild, PreschoolHumansInfantMiddle AgedPrimary Immunodeficiency DiseasesSurveys and QuestionnairesT-Lymphocytes, RegulatoryTreatment OutcomeYoung Adultautoimmunitygeneticshematopoietic cell transplantimmune dysregulationprimary immune deficiencies
Identifiers
What OpenQuestion holds
Textmetadata
LicenceCC BY
Read underepoch 390
Registered trials
None linked
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.