Real-world Canadian data on belumosudil therapy in heavily pretreated patients with steroid-refractory chronic graft-versus-host disease: treatment outcomes and risk factor analysis for failure-free survival
Bibliographic record
Abstract
Chronic graft-versus-host disease (cGvHD) remains one of the common causes of morbidity and mortality after allogeneic hematopoietic stem cell transplantation. Belumosudil (BEL), a selective ROCK2 inhibitor, has immunomodulatory and anti-fibrotic properties, offering a new therapeutic option. Real-world data (RWD) in heavily pretreated patients remain limited, particularly for combination of BEL with ruxolitinib (RUX). We conducted a multicenter, real-world study in 46 patients treated for refractory cGvHD with BEL under a Canadian compassionate program. Treatment outcomes were assessed using the NIH consensus response criteria for overall response rate (ORR), failure-free (FFS), overall survival (OS), and safety. Forty-six patients were included with a median follow-up of 11.4 months; the best ORR was 52% (n = 20/38). The FFS and OS rates at 12 months were 64.3% and 91.1%, respectively. Steroids were discontinued in 73% at 12 months. BEL combination therapy with RUX exhibited equivalent treatment outcomes to BEL monotherapy, although patients treated with drug combination presented with more advanced form of GvHD and mostly failed RUX therapy. A prognostic risk model based on prior acute GvHD and involvement of ≥ 4 organs effectively stratified FFS at 12 months: 100% with no risk factors, 75.8% with one, and 30% with two risk factors (HR 3.91, 95% CI 1.58-9.67, p = 0.003). BEL demonstrated durable efficacy and acceptable safety in heavily pretreated cGvHD. BEL treatment was associated with a high probability of corticosteroid withdrawal. Risk stratification by disease burden and prior aGvHD identified distinct prognostic groups, informing patient selection and future therapeutic strategies.
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How this classification was reachedexpand
Full frame distilled prediction
Teacher imitationNot calibrated prevalence, not ground truth. Human validation pending. Learned from the 10,348 direct Codex labels and 10,348 direct Gemma labels. Candidate is the union of thresholded teacher heads; consensus is their intersection. These outputs are machine_predicted_unvalidated and are not human labels or direct frontier model labels.
Codex and Gemma teacher scores by category
| Category | Codex | Gemma |
|---|---|---|
| Metaresearch | 0.000 | 0.000 |
| Meta-epidemiology (narrow) | 0.000 | 0.000 |
| Meta-epidemiology (broad) | 0.001 | 0.000 |
| Bibliometrics | 0.001 | 0.000 |
| Science and technology studies | 0.000 | 0.000 |
| Scholarly communication | 0.000 | 0.000 |
| Open science | 0.000 | 0.000 |
| Research integrity | 0.000 | 0.000 |
| Insufficient payload (model declined to judge) | 0.000 | 0.000 |
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.
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 teacher head, 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".