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Record W4408905115 · doi:10.1182/blood.2023023717

Reducing clinical trial eligibility barriers for patients with MDS: an icMDS position statement

2025· article· en· W4408905115 on OpenAlexaff
Uma Borate, Kelly Pugh, Allyson Waller, Rina Li Welkie, Ying Huang, Jan Philipp Bewersdorf, Maximilian Stahl, Amy E. DeZern, Uwe Platzbecker, Mikkael A. Sekeres, Andrew H. Wei, Rena Buckstein, Gail J. Roboz, Michael R. Savona, Sanam Loghavi, Robert P. Hasserjian, Pierre Fenaux, David A. Sallman, Christopher S. Hourigan, Matteo Giovanni Della Porta, Stephen D. Nimer, Richard F. Little, Valeria Santini, Fabio Efficace, Justin Taylor, Guillermo Garcia‐Manero, Olatoyosi Odenike, Tae Kon Kim, Stephanie Halene, Rami S. Komrokji, Elizabeth A. Griffiths, Peter L. Greenberg, Mina L. Xu, Zhuoer Xie, Rafael Bejar, Guillermo Sanz, Mrinal M. Patnaik, María E. Figueroa, Hetty E. Carraway, Omar Abdel‐Wahab, Daniel T. Starczynowski, Eric Padron, Jacqueline Boultwood, Steven D. Gore, Naval Daver, Jane E. Churpek, Ravindra Majeti, John M. Bennett, Alan F. List, Andrew M. Brunner, Amer M. Zeidan

Bibliographic record

VenueBlood · 2025
Typearticle
Languageen
FieldMedicine
TopicAcute Myeloid Leukemia Research
Canadian institutionsHealth Sciences CentreSunnybrook Health Science Centre
FundersNational Institute of Diabetes and Digestive and Kidney DiseasesNational Cancer InstituteNational Heart, Lung, and Blood Institute
KeywordsClinical trialMedicinePopulationIntensive care medicineFamily medicineInternal medicine

Abstract

fetched live from OpenAlex

ABSTRACT: Excessively restrictive inclusion and exclusion criteria in clinical trials are one of many barriers to clinical trial enrollment for patients with myelodysplastic syndromes/neoplasms (MDSs). Many organizations are developing efforts to increase clinical trial eligibility; yet, several recent publications focused on patients with MDS suggest that many patients with this disease may be excluded from clinical trials unnecessarily. Clinical trial eligibility should reflect the phase of the study and risks of the agent being studied. Phase 3 trials should be less restrictive than early-phase trials to represent the real-world population as closely as possible. We hypothesize that many clinical trials, particularly phase 3 trials, have unnecessarily restrictive eligibility criteria. This study aims to evaluate the most common eligibility criteria according to phase of trial and to determine whether criteria correspond with drug safety signals. We identified MDS clinical trials registered on ClinicalTrials.gov from 1 January 2000 to 1 September 2023 and analyzed the eligibility criteria of 191 therapeutic MDS trials. We found that categorical inclusion and exclusion criteria are remarkably similar in representation across trial phases. Additionally, only 13% of trials are concordant with drug safety signals, suggesting that the eligibility criteria are often arbitrary. On behalf of the icMDS (International Consortium for Myelodysplastic Syndromes), an association of international MDS experts, we provide a position statement on restrictive eligibility criteria for MDS clinical trials that should be avoided with the aim of removing barriers to clinical trial enrollment.

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)0.511
metaresearch head score (Gemma)0.526
Version: metacan-v3-hybrid-931329e0061cValidation status: machine_predicted_unvalidated
Candidate categoriesMetaresearch
Consensus categoriesMetaresearch
DomainCandidate signal: Methods · Consensus signal: none
Study designCandidate signal: Not applicable · Consensus signal: Not applicable
GenreCandidate signal: Commentary · Consensus signal: Commentary
Teacher disagreement score0.489
Threshold uncertainty score0.603

Distilled classifier scores by category (both heads)

CategoryCodexGemma
Metaresearch0.5110.526
Meta-epidemiology (narrow)0.0020.002
Meta-epidemiology (broad)0.0030.004
Bibliometrics0.0060.008
Science and technology studies0.0060.006
Scholarly communication0.0150.007
Open science0.0070.010
Research integrity0.0260.028
Insufficient payload (model declined to judge)0.0040.002

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 head0.033
GPT teacher head0.412
Teacher spread0.379 · 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; the direct Gemma label and the distilled Codex classifier agree on what is shown here.

Study designNot applicable
DomainMethods
GenreCommentary

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

Citations10
Published2025
Admission routes1
Has abstractyes

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