Efficacy and safety of axatilimab at 3 different doses in patients with chronic graft-versus-host disease (cGVHD) and related bronchiolitis obliterans syndrome (BOS): results of a pivotal phase 2 study
Bibliographic record
Abstract
Rationale: Activity of the anti-CSF-1R monoclonal antibody axatilimab in cGVHD-associated BOS was previously shown in a phase 1/2 study ( NCT03604692 ). Methods: AGAVE-201, a pivotal phase 2, open-label, randomized, multicenter study of axatilimab in patients with recurrent/refractory cGVHD ( NCT04710576 ) randomized patients 1:1:1 to intravenous axatilimab at 0.3 mg/kg every 2 weeks (Q2W, n=80), 1 mg/kg Q2W (n=81), or 3 mg/kg every 4 weeks (Q4W, n=80). BOS and cGVHD diagnosis and response criteria followed 2014 National Institutes of Health cGVHD Consensus. The primary efficacy endpoint was cGVHD overall response rate (ORR) in the first 6 cycles (24 weeks). Safety endpoints included frequency and severity of treatment-emergent adverse events (TEAEs). Results: BOS was present in 108 (45%) patients. The primary ORR endpoint was met in all cohorts, and along with BOS response rate was highest at the 0.3 mg/kg dose (Table). The median time to BOS response was <3 months. TEAEs were dose dependent in frequency and severity, with majority on-target effects of CSF-1R blockade. TEAE-driven discontinuation occurred in 6% of all patients in the 0.3 mg/kg cohort and 16% of all study patients. Conclusions: Axatilimab dose of 0.3 mg/kg Q2W showed the best efficacy and safety in cGVHD and BOS. Axatilimab at the 0.3 mg/kg Q2W dose is being investigated in a Phase 2 study in idiopathic pulmonary fibrosis ( NCT06132256 ). erj;64/suppl_68/PA4778/T1 T1 t1 Characteristic (%) 0.3 mg/kg Q2W 1 mg/kg Q2w 3 mg/kg Q4W Baseline BOS 40 51 44 FEV1≤39%* 47 42 26 ORR 74 67 50 BOS response* 47 34 37 *Among patients with baseline BOS
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How this classification was reachedexpand
Full frame machine prediction
Teacher imitationNot 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.
Distilled classifier scores by category (both heads)
| Category | Codex | Gemma |
|---|---|---|
| Metaresearch | 0.002 | 0.001 |
| Meta-epidemiology (narrow) | 0.001 | 0.000 |
| Meta-epidemiology (broad) | 0.002 | 0.001 |
| Bibliometrics | 0.000 | 0.000 |
| Science and technology studies | 0.000 | 0.001 |
| Scholarly communication | 0.001 | 0.001 |
| Open science | 0.001 | 0.000 |
| Research integrity | 0.001 | 0.003 |
| Insufficient payload (model declined to judge) | 0.002 | 0.001 |
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.
score_only:v0-immature-baseline · verbatim from the scoring run: score_only means the number may rank works, and no category label ships from itClassification
machine, unvalidatedMachine predicted; a candidate call from one source (direct Gemma or distilled Codex), not a consensus.
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".