Evidence map›Paper›PMID 42242424›Full record

ArticleThe journal of allergy and clinical immunology. In practice2026

IMPACT (Immune Monitoring and Phenotype Assessment of Clinical Trajectory) for Patients With Inborn Errors of Immunity From the Primary Immune Deficiency Treatment Consortium (PIDTC).

Danielle E Arnold, Hesham Eissa, Elizabeth Dunn, Geoffrey D E Cuvelier, Nicola A M Wright, Rebecca A Marsh, Suhag Parikh, Blachy Davila Saldana, Mark T Vander Lugt, Rosa Bacchetta and 19 more

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In one paragraph

Article in The journal of allergy and clinical immunology. In practice, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 1 paper.

0numbers the graph read from it
0cells of the map it votes in
1citing 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

1 citing paper in PubMed.

  1. Article
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

29 authors.

Danielle E ArnoldImmune Deficiency Cellular Therapy, National Cancer Institute, Bethesda, Md.
Hesham EissaChildren's Hospital of Colorado, Department of Pediatrics, Blood and Marrow Transplant and Cellular Therapeutics, University of Colorado, Aurora, Colo.
Elizabeth DunnPediatric Allergy, Immunology, and Blood and Marrow Transplantation Division, University of California San Francisco, San Francisco, Calif.
Geoffrey D E CuvelierPediatric Oncology and Transplantation, Alberta Children's Hospital, University of Calgary, Calgary, Alberta, Canada.
Nicola A M WrightSection of Hematology/Immunology, Alberta Children's Hospital, University of Calgary, Calgary, Alberta, Canada.
Rebecca A MarshBone Marrow Transplantation and Immune Deficiency, Cincinnati Children's Hospital Medical Center, University of Cincinnati, Cincinnati, Ohio.
Suhag ParikhAflac Cancer and Blood Disorders Center, Children's Healthcare of Atlanta, Department of Pediatrics, Emory University School of Medicine, Atlanta, Ga.
Blachy Davila SaldanaBone Marrow Transplantation and Immune Deficiency, Cincinnati Children's Hospital Medical Center, University of Cincinnati, Cincinnati, Ohio.
Mark T Vander LugtDepartment of Pediatrics, Division of Pediatric Hematology/Oncology/BMT, University of Michigan, Ann Arbor, Mich.
Rosa BacchettaDepartment of Pediatrics, Division of Hematology, Oncology, Stem Cell Transplantation and Regenerative Medicine, Institute for Stem Cell Biology and Regenerative Medicine, Center for Definitive and Curative Medicine, Stanford University, Stanford, Calif.
Lisa Forbes SatterPediatrics, Immunology, Allergy and Retrovirology, Baylor College of Medicine/Texas Children's Hospital, Houston, Tex.
Shanmuganathan ChandrakasanImmune Dysregulation / Immunohematology and Immune Defect Transplant Program, Aflac Cancer and Blood Disorders Center, Children's Healthcare of Atlanta, Emory University, Atlanta, Ga.
Erinn KellnerDivision of Allergy/Immunology, Cincinnati Children's Hospital and University of Cincinnati, Cincinnati, Ohio.
Eric J AllenspachDivision of Immunology, Department of Pediatrics, University of Washington and Seattle Children's Research Institute, Seattle, Wash.
Erica G SchmittDepartment of Pediatrics, Division of Rheumatology and Immunology, Washington University School of Medicine, St Louis, Mo.
Brent LoganDivision of Biostatistics, Institute for Health and Equity, Center for International Blood and Marrow Transplant Research, Medical College of Wisconsin, Milwaukee, Wis.
Lauri BurroughsPediatrics, Hematology/Oncology, Fred Hutchinson Cancer Center, University of Washington School of Medicine, and Seattle Children's Hospital, Seattle, Wash.
Morton J CowanPediatric Allergy, Immunology, and Blood and Marrow Transplantation Division, University of California San Francisco, San Francisco, Calif.
Linda M GriffithDivision of Allergy, Immunology and Transplantation, National Institute of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, Md.
Luigi NotarangeloLaboratory of Clinical Immunology and Microbiology, National Institute of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, Md.
Sung-Yun PaiImmune Deficiency Cellular Therapy, National Cancer Institute, Bethesda, Md.
Michael A PulsipherDivision of Hematology and Oncology, Intermountain Primary Children's Hospital, Huntsman Cancer Institute, Spencer Fox Eccles School of Medicine at the University of Utah, Salt Lake City, Utah.
Christopher C DvorakPediatric Allergy, Immunology, and Blood and Marrow Transplantation Division, University of California San Francisco, San Francisco, Calif.
Elie HaddadDepartment of Pediatrics, Department of Microbiology, Immunology and Infectious Diseases, University of Montreal, CHU Sainte-Justine Azrieli Research Center, Montreal, Quebec, Canada.
Jennifer M PuckPediatric Allergy, Immunology, and Blood and Marrow Transplantation Division, University of California San Francisco, San Francisco, Calif.
Troy R TorgersonAllen Institute for Immunology, Seattle, Wash.
Jennifer W LeidingInstitute for Clinical and Translational Research and Cancer and Blood Disorders Institute, Johns Hopkins All Children's Hospital, St. Petersburg, Fla; Department of Pediatrics, Division of Allergy and Immunology, Johns Hopkins University, Baltimore, Md.
Jennifer HeimallChildren's Hospital of Philadelphia, Philadelphia, Pa.
Alice Y ChanPediatric Allergy, Immunology, and Blood and Marrow Transplantation Division, University of California San Francisco, San Francisco, Calif; Pediatric Rheumatology Division, University of California San Francisco, San Francisco, Calif. Electronic address: alice.chan1@ucsf.edu.

Funding

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
The Impact of COVID-19 on People Living with Rare Diseases and Their FamiliesU2CTR002818 · NCATS · CINCINNATI CHILDRENS HOSP MED CTR · PI Michal Kouril · 2019 to 2026
$51.2M
Training of Clinical Investigators in Primary Immune DeficienciesU54AI082973 · NIAID · UNIVERSITY OF CALIFORNIA, SAN FRANCISCO · PI MALECH, HARRY · 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
Primary Immune Deficiency Treatment Consortium Annual Scientific MeetingR13AI094943 · NIAID · UNIVERSITY OF CALIFORNIA, SAN FRANCISCO · PI PUCK, JENNIFER M. · 2011 to 2025
$239k
NCATS NIH HHS U2C TR002818NCI NIH HHS U24 CA076518NHLBI NIH HHS U01 HL069294NIAID NIH HHS R13 AI094943NIAID NIH HHS R24 AI184316NIAID NIH HHS U54 AI082973NINDS NIH HHS U54 NS064808
6 · The paper itself

Abstract

backgroundEvaluating natural history and outcomes of rare inborn errors of immunity (IEIs) has been challenging due to the diverse disease manifestations and lack of standardized data.

objectiveTo develop a set of standardized, quantitative, generalizable data collection modules to measure organ dysfunction in IEIs, modeled on the previously validated Common Terminology Criteria for Adverse Event system for grading treatment toxicity.

methodsDisease conditions were organized by organ system. Experts from the Primary Immune Deficiency Treatment Consortium identified essential features of severe combined immunodeficiency, chronic granulomatous disease, Wiskott-Aldrich syndrome, and primary immune regulatory disorders and compared these to Common Terminology Criteria for Adverse Event conditions, adding and refining features as needed. Dates of onset and resolution, disease-specific laboratory values, and therapeutic interventions were also incorporated. Modules were reviewed by protocol teams, key stakeholders, clinical coordinators, and statisticians to improve feasibility and completeness.

resultsWe developed a tool called IMPACT (Immune Monitoring and Phenotype Assessment of Clinical Trajectory) containing 15 modules assessing 196 disease conditions. A grading system from 1 to 5 (mild, moderate, severe, life-threatening, fatal) permits longitudinal tracking of disease condition severity. Outcomes of interventions can be measured quantitatively by comparing scores before and after treatment.

conclusionsStandardized, quantitative assessment tools for IEIs can yield uniform data sets to illuminate their natural history and evaluate outcomes of interventions, not only to understand specific diseases but also to compare effects between disease entities. Tools, such as IMPACT, are essential for therapeutic trials for rare, complex multisystem diseases such as IEIs and other disorders with protean manifestations.

Indexed as

Immunologic Deficiency SyndromesHumansMonitoring, ImmunologicPhenotypeCase report formCTCAEIMPACTInborn errors of immunityInflammatory diseaseLate effectsNatural historyPrimary immune regulatory disorderStandardized assessment

Identifiers

PMID42242424
PMCPMC13450914

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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.