Evidence map›Paper›PMID 27262745›Full record

ReviewThe Journal of allergy and clinical immunology2016

Primary Immune Deficiency Treatment Consortium (PIDTC) update.

Linda M Griffith, Morton J Cowan, Luigi D Notarangelo, Donald B Kohn, Jennifer M Puck, William T Shearer, Lauri M Burroughs, Troy R Torgerson, Hélène Decaluwe, Elie Haddad and 1 more

4 registry-linked trialsAbstract readReview
In one paragraph

Review in The Journal of allergy and clinical immunology, 2016. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to 4 registered trials, which are not on this map. Cited by 30 papers.

0numbers the graph read from it
0cells of the map it votes in
30citing 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.

NCT01186913 enrolling by invitationnot on this map

A Prospective Natural History Study of Diagnosis, Treatment and Outcomes of Children With SCID Disorders (RDCRN PIDTC-6901)

TypeobservationalSponsorNational Institute of Allergy and Infectious Diseases (NIAID)Ran2010 to 2028Enrolled690ConditionsSevere Combined Immunodeficiency (SCID), Leaky SCID, Omenn Syndrome, Reticular Dysgenesis
NCT01346150 unknown statusnot on this map

A Retrospective and Cross-Sectional Analysis of Patients Treated for SCID Since January 1,1968 (RDCRN PIDTC-6902)

TypeobservationalSponsorNational Institute of Allergy and Infectious Diseases (NIAID)Ran2011 to 2023Enrolled1,007ConditionsSCID, ADA-SCID, XSCID, Leaky SCID
NCT02064933 completednot on this map

Analysis of Patients Treated for Wiskott-Aldrich Syndrome Since January 1, 1990 (RDCRN PIDTC-6904)

TypeobservationalSponsorNational Institute of Allergy and Infectious Diseases (NIAID)Ran2014 to 2019Enrolled305ConditionsWiskott-Aldrich Syndrome
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

30 citing papers in PubMed.

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  19. Review
  20. WhenFrontiers in immunology · 2019
    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

11 authors.

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, Children's Hospital, and Harvard Stem Cell Institute, Harvard Medical School, Boston, Mass.
Donald B KohnDepartments of Microbiology, Immunology & Molecular Genetics and Pediatrics, 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.
William T ShearerPediatric Allergy & Immunology, Texas Children's Hospital, Baylor College of Medicine, Houston, Tex.
Lauri M BurroughsPediatric Hematology/Oncology, Fred Hutchinson Cancer Research Center, University of Washington School of Medicine, Seattle, Wash.
Troy R TorgersonPediatric Rheumatology, Seattle Children's Research Institute, University of Washington School of Medicine, Seattle, Wash.
Hélène DecaluwePediatric Immunology and Pediatrics, Mother and Child Ste-Justine Hospital, University of Montreal, Montreal, Quebec, Canada.
Elie HaddadPediatric Immunology and Pediatrics, Mother and Child Ste-Justine Hospital, University of Montreal, Montreal, Quebec, Canada.
workshop participants

Funding

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 PUCK, JENNIFER M. · 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
Project 3: Gene Therapy for Correction of Blood DisordersP01HL122173 · NHLBI · FRED HUTCHINSON CANCER RESEARCH CENTER · PI KIEM, HANS-PETER · 2015 to 2019
$12.9M
Primary Immune Deficiency Treatment Consortium Annual Scientific MeetingR13AI094943 · NIAID · UNIVERSITY OF CALIFORNIA, SAN FRANCISCO · PI PUCK, JENNIFER M. · 2011 to 2025
$239k
Intramural NIH HHS Z99 AI999999NCATS NIH HHS U01 TR001263NHLBI NIH HHS P01 HL122173NIAID NIH HHS R13 AI094943NIAID NIH HHS U54 AI082973NINDS NIH HHS U54 NS064808
6 · The paper itself

Abstract

The Primary Immune Deficiency Treatment Consortium (PIDTC) is a collaboration of 41 North American centers studying therapy for rare primary immune deficiency diseases (PIDs), including severe combined immune deficiency (SCID), Wiskott-Aldrich syndrome (WAS), and chronic granulomatous disease (CGD). An additional 3 European centers have partnered with the PIDTC to study CGD. Natural history protocols of the PIDTC analyze outcomes of treatment for rare PIDs in multicenter longitudinal retrospective, prospective, and cross-sectional studies. Since 2009, participating centers have enrolled more than 800 subjects on PIDTC protocols for SCID, and enrollment in the studies on WAS and CGD is underway. Four pilot projects have been funded, and 12 junior investigators have received fellowship awards. Important publications of the consortium describe the outcomes of hematopoietic cell transplantation for SCID during 2000-2009, diagnostic criteria for SCID, and the pilot project of newborn screening for SCID in the Navajo Nation. The PIDTC Annual Scientific Workshops provide an opportunity to strengthen collaborations with junior investigators, patient advocacy groups, and international colleagues. Funded by the National Institute of Allergy and Infectious Diseases and the Office of Rare Diseases Research, National Center for Advancing Translational Sciences, the PIDTC has recently received renewal for another 5 years. Here we review accomplishments of the group, projects underway, highlights of recent workshops, and challenges for the future.

Indexed as

Voluntary Health AgenciesAnimalsClinical Studies as TopicHumansImmunologic Deficiency SyndromesIntersectoral CollaborationAllogeneic hematopoietic cell transplantationclinical trialgene therapyprimary immunodeficiency

Identifiers

PMID27262745
PMCPMC4986691

What OpenQuestion holds

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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.