Evidence map›Paper›PMID 31376032›Full record

ArticleJournal of clinical immunology2019

Chronic Granulomatous Disease-Associated IBD Resolves and Does Not Adversely Impact Survival Following Allogeneic HCT.

Rebecca A Marsh, Jennifer W Leiding, Brent R Logan, Linda M Griffith, Danielle E Arnold, Elie Haddad, E Liana Falcone, Ziyan Yin, Kadam Patel, Erin Arbuckle and 45 more

Erratum issued Registry-linked trialOpen access · greenAbstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in Journal of clinical immunology, 2019. The graph could read no effect estimate from its abstract, so it casts no vote on the map. An erratum has been issued. It is linked to trial NCT02082353 (Analysis of Patients Treated for Chronic Granulomatous Disease Since January 1, 1995), which is not on this map. Cited by 34 papers.

0numbers the graph read from it
0cells of the map it votes in
34citing papers in PubMed
2.4field-weighted citation impact, top 11% of its field
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.

NCT02082353 unknown statusnot on this map

Analysis of Patients Treated for Chronic Granulomatous Disease Since January 1, 1995

TypeobservationalSponsorNational Institute of Allergy and Infectious Diseases (NIAID)Ran2014 to 2021Enrolled1,480ConditionsGranulomatous Disease, Chronic
3 · Its place in the literature

Who cites it

34 citing papers in PubMed, 61 citations in OpenAlex.

  1. Article
  2. How I Treat: Chronic granulomatous disease.Journal of human immunity · 2026
    Review
  3. Article
  4. Review
  5. Article
  6. Review
  7. Review
  8. Article
  9. Neutrophil diversity and function in health and disease.Signal transduction and targeted therapy · 2024
    Review
  10. Article
  11. Article
  12. Review
  13. Article
  14. Article
  15. Article
  16. Review
  17. Observational
  18. Article
  19. Article
  20. Review
4 · The record

Corrections and comments

5 · Who and what money

Authors and funding

55 authors at 20 institutions in 2 countries.

Rebecca A MarshDepartment of Pediatrics, University of Cincinnati, and Division of Bone Marrow Transplantation and Immune Deficiency, Cincinnati Children's Hospital Medical Center, Cincinnati, OH, USA.
Jennifer W LeidingDivision of Allergy and Immunology, Department of Pediatrics, Johns Hopkins-All Children's Hospital, University of South Florida, St. Petersburg, FL, USA.
Brent R LoganDivision of Biostatistics, Medical College of Wisconsin, Milwaukee, WI, USA.
Linda M GriffithDivision of Allergy, Immunology, and Transplantation, National Institute of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, MD, USA.
Danielle E ArnoldAllergy and Immunology, The Children's Hospital of Philadelphia, Philadelphia, PA, USA.
Elie HaddadImmunology-Rheumatology Division, Department of Pediatrics, University of Montreal, Montreal, QC, Canada.
E Liana FalconeDivision of Immunity and Viral Infections, Institut de Recherches Cliniques de Montréal, Montréal, QC, Canada; and Department of Medicine, Université de Montréal, Montréal, QC, Canada.
Ziyan YinDivision of Biostatistics, Medical College of Wisconsin, Milwaukee, WI, USA.
Kadam PatelDivision of Biostatistics, Medical College of Wisconsin, Milwaukee, WI, USA.
Erin ArbuckleDepartment of Pediatrics, Duke University, Durham, NC, USA.
Jack J BleesingDepartment of Pediatrics, University of Cincinnati, and Division of Bone Marrow Transplantation and Immune Deficiency, Cincinnati Children's Hospital Medical Center, Cincinnati, OH, USA.
Kathleen E SullivanAllergy and Immunology, The Children's Hospital of Philadelphia, Philadelphia, PA, USA.
Jennifer HeimallAllergy and Immunology, The Children's Hospital of Philadelphia, Philadelphia, PA, USA.
Lauri M BurroughsFred Hutchinson Cancer Research Center, Seattle Children's Hospital, The University of Washington School of Medicine, Seattle, WA, USA.
Suzanne Skoda-SmithSeattle Children's Hospital, Seattle, WA, USA.
Shanmuganathan ChandrakasanDivision of Bone Marrow Transplant, Aflac Cancer and Blood Disorders Center, Children's Healthcare of Atlanta, Emory University School of Medicine, Atlanta, GA, USA.
Lolie C YuDivision of Hematology/Oncology and Hematopoietic Stem Cell Transplantation, The Center for Cancer and Blood Disorders, Children's Hospital/Louisiana State University Medical Center, New Orleans, LA, USA.
Benjamin R OshrineCancer and Blood Disorders Institute, Johns Hopkins All Children's Hospital, St. Petersburg, FL, USA.
Geoffrey D E CuvelierManitoba Blood and Marrow Transplant Program, CancerCare Manitoba, University of Manitoba, Winnipeg, MB, Canada.
Monica S ThakarFred Hutchinson Cancer Research Center, Seattle Children's Hospital, The University of Washington School of Medicine, Seattle, WA, USA.
Karin ChenDivision of Allergy and Immunology, Department of Pediatrics, University of Utah School of Medicine, Salt Lake City, UT, USA.
Pierre TeiraCHU Sainte-Justine, Hematology-Oncology Division, Department of Pediatrics, University of Montreal, Montreal, QC, Canada.
Shalini ShenoyDivision of Pediatric Hematology/Oncology/Bone Marrow Transplantation, Washington University School of Medicine and St. Louis Children's Hospital, St. Louis, MO, USA.
Rachel PhelanPediatric Blood and Marrow Transplant Program, Division of Hematology, Oncology, and Blood and Marrow Transplantation, Medical College of Wisconsin, Milwaukee, WI, USA.
Lisa R ForbesDepartment of Pediatrics, Baylor College of Medicine, Houston, TX, USA, and Section of Allergy, Immunology and Retrovirology, Texas Children's Hospital William T. Shearer Center for Human Immunobiology, Houston, TX, USA.
Deepak ChellapandianBlood and Marrow Transplant Program, Johns Hopkins All Children's Hospital, St. Petersburg, FL, USA.
Blachy J Dávila SaldañaDivision of Blood and Marrow Transplantation, Children's National Medical Center, Washington, DC, USA, and Department of Pediatrics, The George Washington University, Washington, DC, USA.
Ami J ShahDivision of Stem Cell Transplantation and Regenerative Medicine, Stanford School of Medicine, Lucille Packard Children's Hospital, Palo Alto, CA, USA.
Katja G WeinachtDivision of Stem Cell Transplantation and Regenerative Medicine, Department of Pediatrics, Stanford School of Medicine, Stanford, CA, USA.
Avni JoshiDivision of Pediatric Allergy and Immunology, Mayo Clinic, Rochester, MN, USA.
Farid BouladDepartment of Pediatrics, BMT Service, Memorial Sloan Kettering Cancer Center, New York, NY, USA.
Troy C QuiggTexas Transplant Institute, Methodist Children's Hospital, San Antonio, TX, USA.
Christopher C DvorakPediatric Allergy, Immunology, and Blood and Marrow Transplant Division, San Francisco Benioff Children's Hospital, San Francisco, CA, USA.
Debi GrossmanGenetic Immunotherapy Section, Laboratory of Clinical Immunology and Microbiology, National Institute of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, MD, USA.
Troy TorgersonDepartment of Pediatrics, Divisions of Immunology/Rheumatology, University of Washington and Seattle Children's Hospital, Seattle, WA, USA.
Pamela GrahamGenetic Immunotherapy Section, Laboratory of Clinical Immunology and Microbiology, National Institute of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, MD, USA.
Vinod PrasadDivision of Pediatric Blood and Marrow Transplant, Duke University Medical Center, Durham, NC, USA.
Alan KnutsenPediatric Allergy and Immunology, Cardinal Glennon Children's Medical Center, Saint Louis University, St. Louis, MO, USA.
Hey ChongUPMC Children's Hospital of Pittsburgh, Pittsburgh, PA, USA.
Holly MillerCenter for Cancer and Blood Disorders, Phoenix Children's Hospital, Phoenix, AZ, USA.
M Teresa de la MorenaDepartment of Pediatrics/Immunology, University of Washington and Seattle Children's Hospital, Seattle, WA, USA.
Kenneth DeSantesAmerican Family Children's Hospital, University of Wisconsin, Madison, WI, USA.
Morton J CowanPediatric Allergy, Immunology, and Blood and Marrow Transplant Division, San Francisco Benioff Children's Hospital, San Francisco, CA, USA.
Luigi D NotarangeloLaboratory of Clinical Immunology and Microbiology, National Institute of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, MD, USA.
Donald B KohnDavid Geffen School of Medicine at University of California, Los Angeles, CA, USA.
Elizabeth StengerAflac Center and Blood Disorders Center, Children's Healthcare of Atlanta, Emory University, Atlanta, GA, USA.
Sung-Yun PaiHematology-Oncology, Boston Children's Hospital, Boston, MA, USA.
John M RoutesDivision of Allergy and Immunology, Department of Pediatrics, Medical College of Wisconsin, Milwaukee, WI, USA.
Jennifer M PuckPediatric Allergy, Immunology, and Blood and Marrow Transplant Division, San Francisco Benioff Children's Hospital, San Francisco, CA, USA.
Neena KapoorBlood and Marrow Transplant Program, Division of Hematology, Oncology and Blood and Marrow Transplantation, Children's Hospital Los Angeles, Keck School of Medicine, University of Southern California, Los Angeles, CA, USA.
Michael A PulsipherBlood and Marrow Transplant Program, Division of Hematology, Oncology and Blood and Marrow Transplantation, Children's Hospital Los Angeles, Keck School of Medicine, University of Southern California, Los Angeles, CA, USA.
Harry L MalechGenetic Immunotherapy Section, Laboratory of Clinical Immunology and Microbiology, National Institute of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, MD, USA.
Suhag ParikhDivision of Pediatric Blood and Marrow Transplant, Duke University, Durham, NC, USA.
Elizabeth M KangGenetic Immunotherapy Section, Laboratory of Clinical Immunology and Microbiology, National Institute of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, MD, USA. ekang@niaid.nih.gov.
submitted on behalf of the Primary Immune Deficiency Treatment Consortium
Medical College of Wisconsin · USNational Institute of Allergy and Infectious Diseases · USSeattle Children's Hospital · USChildren's Hospital of Philadelphia · USUCSF Benioff Children's Hospital · USBoston Children's Hospital · USCenter for Cancer and Blood Disorders · USChildren's Healthcare of Atlanta · USChildren's Hospital of Los Angeles · USDuke University · USJohns Hopkins University · USNational Institutes of Health · USUniversité de Montréal · CAUniversity of Cincinnati · USBaylor College of Medicine · USCancerCare Manitoba · CACardinal Glennon Children’s Medical Center · USCentre Hospitalier Universitaire Sainte-Justine · CAChildren's Hospital of Pittsburgh · USDuke Medical Center · US

Funding

X-RAY CRYSTALLOGRAPHYP30CA008748 · NCI · SLOAN-KETTERING INSTITUTE FOR CANCER RES · PI Nicholas D Socci · 1985 to 2026
$347.4M
The Impact of COVID-19 on People Living with Rare Diseases and Their FamiliesU2CTR002818 · NCATS · CINCINNATI CHILDRENS HOSP MED CTR · PI Maurizio Macaluso, Michael Wagner · 2019 to 2026
$51.2M
Rare Diseases Data Management and Coordinating CenterU01TR001263 · NCATS · UNIVERSITY OF SOUTH FLORIDA · PI KRISCHER, JEFFREY P · 2014 to 2018
$28.1M
Training of Clinical Investigators in Primary Immune DeficienciesU54AI082973 · NIAID · UNIVERSITY OF CALIFORNIA, SAN FRANCISCO · PI PAI, SUNG-YUN · 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
Development 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
$12.4M
Gene Therapy Basic Research to Treat Inherited Primary Immune DeficienciesZIAAI000644 · NIAID · NATIONAL INSTITUTE OF ALLERGY AND INFECTIOUS DISEASES · PI MALECH, HARRY L · 2009 to 2025
$12.1M
Basic Research on Hematopoietic Human Stem CellsZIAAI000988 · NIAID · NATIONAL INSTITUTE OF ALLERGY AND INFECTIOUS DISEASES · PI MALECH, HARRY L · 2009 to 2019
$6.5M
Atlanta Pediatric Scholars ProgramK12HD072245 · NICHD · EMORY UNIVERSITY · PI Shari Barkin · 2012 to 2026
$5.5M
Role of reactive oxygen species and the microbiome in intestinal barrier homeostasisZIAAI001216 · NIAID · NATIONAL INSTITUTE OF ALLERGY AND INFECTIOUS DISEASES · PI HOLLAND, STEVEN · 2016 to 2025
$4.1M
Gene Therapy Clinical Trials for Chronic Granulomatous DiseaseZIAAI000991 · NIAID · NATIONAL INSTITUTE OF ALLERGY AND INFECTIOUS DISEASES · PI KANG, ELIZABETH · 2009 to 2019
$3.4M
Clinical Trials of Allogeneic Transplantation for Inherited Immune DeficienciesZ01AI000989 · NIAID · NIAID EXTRAMURAL ACTIVITIES · PI KANG, ELIZABETH · 2007 to 2008
$878k
Intramural NIH HHS Z01 AI000989NCATS NIH HHS U01 TR001263NCATS NIH HHS U2C TR002818NCI NIH HHS P30 CA008748NIAID NIH HHS R13 AI094943NIAID NIH HHS U54 AI082973NICHD NIH HHS K12 HD072245NINDS NIH HHS U54 NS064808
6 · The paper itself

Abstract

introductionInflammatory bowel disease (IBD) affects approximately 1/3 of patients with chronic granulomatous disease (CGD). Comprehensive investigation of the effect of allogeneic hematopoietic cell transplantation (HCT) on CGD IBD and the impact of IBD on transplant outcomes is lacking.

methodsWe collected data retrospectively from 145 patients with CGD who had received allogeneic HCT at 26 Primary Immune Deficiency Treatment Consortium (PIDTC) centers between January 1, 2005 and June 30, 2016.

resultsForty-nine CGD patients with IBD and 96 patients without IBD underwent allogeneic HCT. Eighty-nine percent of patients with IBD and 93% of patients without IBD engrafted (p = 0.476). Upper gastrointestinal acute GVHD occurred in 8.5% of patients with IBD and 3.5% of patients without IBD (p = 0.246). Lower gastrointestinal acute GVHD occurred in 10.6% of patients with IBD and 11.8% of patients without IBD (p = 0.845). The cumulative incidence of acute GVHD grades II-IV was 30% (CI 17-43%) in patients with IBD and 20% (CI 12-29%) in patients without IBD (p = 0.09). Five-year overall survival was equivalent for patients with and without IBD: 80% [CI 66-89%] and 83% [CI 72-90%], respectively (p = 0.689). All 33 surviving evaluable patients with a history of IBD experienced resolution of IBD by 2 years following allogeneic HCT.

conclusionsIn this cohort, allogeneic HCT was curative for CGD-associated IBD. IBD should not contraindicate HCT, as it does not lead to an increased risk of mortality. This study is registered at clinicaltrials.gov NCT02082353.

Indexed as

Hematopoietic Stem Cell TransplantationAdolescentAdultChildChild, PreschoolFemaleGraft vs Host DiseaseGranulomatous Disease, ChronicHumansIncidenceInfantInflammatory Bowel DiseasesLeukocyte CountMaleNeutrophilsPrognosisallogeneic bone marrow transplantationAllogeneic hematopoietic cell transplantationallogeneic hematopoietic stem cell transplantationchronic granulomatous diseaseinflammatory bowel diseaseprimary immunodeficiency

Identifiers

PMID31376032
PMCPMC6754755
OpenAlexW2966556522

What OpenQuestion holds

Textmetadata
LicenceTDM
Read underepoch 390

Registered trials

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.