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Evaluation and Management of Deficiency of Adenosine Deaminase 2

2023· review· en· W4378783574 on OpenAlexaff
Pui Y. Lee, Brad A. Davidson, Roshini S. Abraham, Blanche P. Alter, Juan I. Aróstegui, Katherine Bell, Alexandre Bélot, Jenna Bergerson, Timothy J. Bernard, Paul Brogan, Yackov Berkun, Natalie Deuitch, Dimana Dimitrova, Sophie Georgin‐Lavialle, Marco Gattorno, Bodo Grimbacher, Hasan Hashem, Michael S. Hershfield, Rebecca Ichord, Kazushi Izawa, Jennifer A. Kanakry, Raju Khubchandani, Femke C. C. Klouwer, Evan A. Luton, Ada W. Man, Isabelle Meyts, Joris M. van Montfrans, Seza Özen, Janna Saarela, Gustavo Cordeiro, Aman Sharma, Ariane Soldatos, Rachel Sparks, Troy R. Torgerson, Ignacio Leandro Uriarte, Taryn Youngstein, Qing Zhou, Ivona Aksentijevich, Daniel L. Kastner, Eugene P. Chambers, Amanda K. Ombrello, Mary K. Makley, Kristen L. Hayner, Bridget E. Kling, Lex M. Cowsert, Julie S. Williams

Bibliographic record

VenueJAMA Network Open · 2023
Typereview
Languageen
FieldBiochemistry, Genetics and Molecular Biology
TopicAdenosine and Purinergic Signaling
Canadian institutionsUniversity of Manitoba
FundersFonds Wetenschappelijk OnderzoekBundesministerium für Bildung und ForschungNational Research FoundationVlaamse regeringEuropean CommissionCharles H. Hood FoundationRheumatology Research FoundationJeffrey Modell FoundationArthritis National Research Foundation
KeywordsAdenosine deaminaseAdenosine deaminase deficiencyAMP deaminaseAdenosineMedicineChemistryInternal medicine

Abstract

fetched live from OpenAlex

Importance: Deficiency of adenosine deaminase 2 (DADA2) is a recessively inherited disease characterized by systemic vasculitis, early-onset stroke, bone marrow failure, and/or immunodeficiency affecting both children and adults. DADA2 is among the more common monogenic autoinflammatory diseases, with an estimate of more than 35 000 cases worldwide, but currently, there are no guidelines for diagnostic evaluation or management. Objective: To review the available evidence and develop multidisciplinary consensus statements for the evaluation and management of DADA2. Evidence Review: The DADA2 Consensus Committee developed research questions based on data collected from the International Meetings on DADA2 organized by the DADA2 Foundation in 2016, 2018, and 2020. A comprehensive literature review was performed for articles published prior to 2022. Thirty-two consensus statements were generated using a modified Delphi process, and evidence was graded using the Oxford Center for Evidence-Based Medicine Levels of Evidence. Findings: The DADA2 Consensus Committee, comprising 3 patient representatives and 35 international experts from 18 countries, developed consensus statements for (1) diagnostic testing, (2) screening, (3) clinical and laboratory evaluation, and (4) management of DADA2 based on disease phenotype. Additional consensus statements related to the evaluation and treatment of individuals with DADA2 who are presymptomatic and carriers were generated. Areas with insufficient evidence were identified, and questions for future research were outlined. Conclusions and Relevance: DADA2 is a potentially fatal disease that requires early diagnosis and treatment. By summarizing key evidence and expert opinions, these consensus statements provide a framework to facilitate diagnostic evaluation and management of DADA2.

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)0.009
metaresearch head score (Gemma)0.040
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: none
GenreCandidate signal: Review · Consensus signal: Review
Teacher disagreement score0.009
Threshold uncertainty score0.047

Distilled classifier scores by category (both heads)

CategoryCodexGemma
Metaresearch0.0090.040
Meta-epidemiology (narrow)0.0010.000
Meta-epidemiology (broad)0.0010.001
Bibliometrics0.0020.001
Science and technology studies0.0010.001
Scholarly communication0.0010.002
Open science0.0010.002
Research integrity0.0020.002
Insufficient payload (model declined to judge)0.0020.001

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 head0.088
GPT teacher head0.388
Teacher spread0.300 · 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
GenreReview

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
Published2023
Admission routes1
Has abstractyes

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