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Record W2089324774 · doi:10.1016/j.jaci.2013.07.052

Primary Immune Deficiency Treatment Consortium (PIDTC) report

2013· article· en· W2089324774 on OpenAlexafffund
Linda M. Griffith, Morton J. Cowan, Luigi D. Notarangelo, Donald B. Kohn, Jennifer M. Puck, Sung‐Yun Pai, Barbara Ballard, Sarah Corey Bauer, Jack Bleesing, Marcia Boyle, Amy Brower, Rebecca H. Buckley, Mirjam van der Burg, Lauri M. Burroughs, Fabio Candotti, Andrew J. Cant, Talal A. Chatila, Charlotte Cunningham‐Rundles, Mary C. Dinauer, Christopher C. Dvorak, Alexandra H. Filipovich, Thomas A. Fleisher, Hubert B. Gaspar, Tayfun Güngör, Élie Haddad, Emily Hovermale, Faith Huang, Mary E. Hurley, Sumathi Iyengar, Elizabeth M. Kang, Brent R. Logan, Janel Long-Boyle, Harry L. Malech, Sean McGhee, Fred Modell, Vicki Modell, Hans D. Ochs, Richard J. O’Reilly, Robertson Parkman, David J. Rawlings, John M. Routes, William T. Shearer, Trudy N. Small, Heather Smith, Kathleen E. Sullivan, Paul Szabolcs, Adrian J. Thrasher, Troy R. Torgerson, Paul Veys, Kenneth I. Weinberg, Juan Carlos Zúñiga‐Pflücker

Bibliographic record

VenueJournal of Allergy and Clinical Immunology · 2013
Typearticle
Languageen
FieldImmunology and Microbiology
TopicImmunodeficiency and Autoimmune Disorders
Canadian institutionsUniversity of TorontoCentre Hospitalier Universitaire Sainte-Justine
FundersEunice Kennedy Shriver National Institute of Child Health and Human DevelopmentNational Institute of Neurological Disorders and StrokeNational Cancer InstituteNational Heart, Lung, and Blood InstituteManton Center for Orphan Disease Research, Boston Children's HospitalFeinberg School of MedicineUniversity of California, San FranciscoHarvard Stem Cell InstituteBaxter Healthcare CorporationNational Institutes of HealthNational Center for Advancing Translational SciencesBentham-Moxon TrustUniversity of California, Los AngelesMedacUniversity of TorontoUniversity of Southern CaliforniaWellcome TrustUniversity College LondonChildren's Hospital Los AngelesCSL BehringNational Institute for Health and Care ResearchTowson UniversityUniversity of South FloridaUniversity of WashingtonPerelman School of Medicine, University of PennsylvaniaImmune Deficiency FoundationTexas Children's HospitalUniversität ZürichSchool of MedicineNewcastle UniversityUniversity of PennsylvaniaHospital for Sick ChildrenSchool of Medicine, Duke UniversityNorthwestern UniversityNIH Clinical CenterNational Institute of Allergy and Infectious DiseasesGrifolsChildren's Hospital of Philadelphiabluebird bioCincinnati Children's Hospital Medical CenterMarch of Dimes Foundation
KeywordsPrimary immunodeficiencyMedicineFamily medicineSevere combined immunodeficiencyTransplantationNatural historyPediatricsDiseasePathologyInternal medicine

Abstract

fetched live from OpenAlex

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.

Fetched live from OpenAlex and de-inverted. Abstracts are not stored in this database: the inverted indexes are 8.6 GB of the frame’s 9.3 GB of text, and the host has 13 GB free.

How this classification was reachedexpand

Full frame machine prediction

Teacher imitation

Not calibrated prevalence, not ground truth. Human validation pending. The Gemma side is a direct model label for every work in the frame, read from the title-only record. The Codex side is a classifier learned from the 10,348 direct Codex labels and calibrated to design-weighted sample rates; fields without enough sample support carry no Codex call. Candidate is the union of the two sides; consensus is their intersection. These outputs are machine_predicted_unvalidated and are not human labels.

metaresearch head score (Codex)0.008
metaresearch head score (Gemma)0.011
Version: metacan-v3-hybrid-931329e0061cValidation status: machine_predicted_unvalidated
Candidate categoriesnone
Consensus categoriesnone
DomainCandidate signal: none · Consensus signal: none
Study designCandidate signal: Not applicable · Consensus signal: Not applicable
GenreCandidate signal: Other · Consensus signal: Other
Teacher disagreement score0.018
Threshold uncertainty score0.062

Distilled classifier scores by category (both heads)

CategoryCodexGemma
Metaresearch0.0080.011
Meta-epidemiology (narrow)0.0000.000
Meta-epidemiology (broad)0.0000.001
Bibliometrics0.0010.001
Science and technology studies0.0020.000
Scholarly communication0.0030.001
Open science0.0010.002
Research integrity0.0030.003
Insufficient payload (model declined to judge)0.0180.004

Machine scores (provisional)

The two teacher heads of the student model, read on this work. A score orders the frame for review; it never asserts a category, and the validation status ships verbatim with every row.

Baseline scores from an immature model (maturity gate not passed, 7 training rounds). Scores rank; they never assert a category.

Opus teacher head0.017
GPT teacher head0.278
Teacher spread0.261 · how far apart the two teachers sit on this one work
Validation statusscore_only:v0-immature-baseline · verbatim from the scoring run: score_only means the number may rank works, and no category label ships from it

Classification

machine, unvalidated

Machine predicted; a candidate call from one source (direct Gemma or distilled Codex), not a consensus.

The models applied no category: nothing in the taxonomy fit this work.
Study designNot applicable
Domainnot available
GenreOther

How this classification was reached, model by model and score by score, is at the end of the page under "How this classification was reached".

Quick stats

Citations86
Published2013
Admission routes2
Has abstractyes

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