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Matching-adjusted indirect comparison (MAIC) of olutasidenib (OLU) and ivosidenib (IVO) in isocitrate dehydrogenase 1 (IDH1)-mutated relapsed/refractory (R/R) acute myeloid leukemia (AML).

2025· article· en· W4410809230 on OpenAlexaff
Justin M. Watts, Brian A. Jonas, Eunice S. Wang, Florence R. Wilson, Julie R. Park, Shannon Cope, Aaron Sheppard, Stéphane de Botton, Jörge E. Cortes

Bibliographic record

VenueJournal of Clinical Oncology · 2025
Typearticle
Languageen
FieldBiochemistry, Genetics and Molecular Biology
TopicProtein Degradation and Inhibitors
Canadian institutionsMD Precision (Canada)
Fundersnot available
KeywordsIsocitrate dehydrogenaseMedicineIDH1Myeloid leukemiaRefractory (planetary science)Nuclear medicineIDH2Cancer researchMutationEnzymeGeneBiologyGeneticsBiochemistry

Abstract

fetched live from OpenAlex

6546 Background: OLU and IVO are allosteric type II IDH1 inhibitors approved by the FDA and recommended by NCCN for IDH1 mut R/R AML patients based on single-arm trials. In the absence of a head-to-head trial, a MAIC was performed to estimate relative treatment effects of OLU vs. IVO in IDH1 mut R/R AML. Methods: Analyses used registrational data for OLU (Study 2102-HEM-101; N=147; individual-level data) and IVO (AG120-C-001; N=174; study-level data). A logistic propensity score model was used to estimate weights based on the first moment for Study 2102-HEM-101 patients to match AG120-C-001, including the following characteristics identified from a literature review, validated by clinical experts: number of prior systemic therapies, age, prior stem cell transplant, AML type, relapse type, cytogenetic risk, ECOG PS, and IDH1 mutation. Complete remission (CR) and CR + CR with partial hematological recovery (CRh) were summarized as odds ratios (ORs) and 95% confidence intervals (CIs). Duration of CR (DoCR), duration of CR+CRh, and OS were summarized in terms of difference in medians and 95% CIs. OS was also summarized in terms of hazard ratios (HRs) and restricted mean survival time (RMST). A simulated treatment comparison (STC) was performed as a sensitivity analysis. Results: Table 1 summarizes MAIC-adjusted estimates. Naïve and adjusted rates of CR and CR+CRh for OLU vs. IVO were comparable, but point estimates favored OLU for CR. Differences in median DoCR were not statistically significant but favored OLU over IVO. OLU had a significantly longer duration of CR+CRh than IVO. For OS, the naïve comparison suggested OLU was better than IVO (HR=0.72; 95% CI 0.55, 0.92), whereas the MAIC was uncertain but favored OLU. STC results were consistent with the MAIC. Conclusions: Naïve and adjusted rates of response for OLU vs. IVO were comparable (adjusted point estimate favored OLU for CR and IVO for CR+CRh), while a longer duration of CR+CRh was observed with OLU. Adjusted OS was similar between the two groups, although the HR favored OLU, and could not be estimated by response category given lack of patient characteristics and reduction in effective sample size (ESS). Results rely on the assumption of no unmeasured confounders which reflects a limitation of the methodology. MAIC results. Outcome OLU – adjusted (95% CI) IVO – observed (95% CI) MAIC OLU vs. IVO (95% CI) N ESS CR 27% 25% OR=1.12 (0.61, 2.08) 147 73.03 CR + CRh 29% 33% OR=0.83 (0.46, 1.50) 147 73.03 DoCR, median mos 21.3 (12.0, NE) 10.1 (6.5, 22.2) Diff=11.18 (-4.30, 22.72) 47 17.76 Duration of CR+CRh, median mos 17.5 (12.0, 29.1) 8.2 (5.6, 12.0) Diff=9.84 (3.24, 22.28) 51 21.06 OS, median mos 9.7 (5.6, 16.4) 9.0 (7.4, 10.2) HR=0.75 (0.53, 1.07) 147 73.03 OS, RMST mos 15.6 (12.1, 19.2) 12.2 (10.6, 13.9) Diff=3.39 (-0.51, 7.29) 147 73.03 N, sample size; NE, not estimable.

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)0.010
metaresearch head score (Gemma)0.021
Version: metacan-v3-hybrid-931329e0061cValidation status: machine_predicted_unvalidated
Candidate categoriesnone
Consensus categoriesnone
DomainCandidate signal: none · Consensus signal: none
Study designCandidate signal: Simulation or modeling · Consensus signal: none
GenreCandidate signal: Empirical · Consensus signal: Empirical
Teacher disagreement score0.010
Threshold uncertainty score0.051

Distilled classifier scores by category (both heads)

CategoryCodexGemma
Metaresearch0.0100.021
Meta-epidemiology (narrow)0.0010.000
Meta-epidemiology (broad)0.0030.007
Bibliometrics0.0010.001
Science and technology studies0.0000.001
Scholarly communication0.0010.001
Open science0.0010.001
Research integrity0.0010.002
Insufficient payload (model declined to judge)0.0070.000

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 head0.044
GPT teacher head0.389
Teacher spread0.345 · 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 designSimulation or modeling
Domainnot available
GenreEmpirical

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".

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Citations0
Published2025
Admission routes1
Has abstractyes

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