Recent lung cGVHD prospective interventional trials
| Trial intervention . | Non-NIH BOS definition . | NIH BOS definition % . | Non-NIH BOS definition % . | N . | Trial design . | Response definition . | Year . |
|---|---|---|---|---|---|---|---|
| Etanercept9 | FEV1/FVC <0.75; trial amended after NIH criteria published | 88 (22/25 obstructive) | 22 | 34 | Single arm | FEV1% | 2012 |
| Montelukast7 | N/A | 100 | 25 | Single arm vs historical | Slope & FEV1% | 2022 | |
| FAM-inhaled fluticasone, azithromycin, montelukast8 | N/A | 100 | 36 | Single arm vs historical | Slope & FEV1% | 2016 | |
| Pirfenidone11 | CT with air trapping >28% or clinical improvement after treatment | 90.9 | 9.1 | 30 | Single arm | Slope of FEV1% | 2022 |
| Belumosudil12 | FEV1 ≤79%, clinician attribution to BOS | Unknown | 100 | 59 | Single arm | FEV1% | 2022 |
| Inhaled corticosteroid and long-acting beta agonist13 | FEV1 <80%, FEV1/FVC <5th percentile of CI, TLC ≥80% | Unknown | 100 | 32 | Randomized controlled | FEV1 (mL) | 2015 |
| Axatilimab10 | N/A | 100 | 16 | Single arm | FEV1% in 1 of 5 responders Symptoms in 4 of 5 responders | 2023 | |
| Ruxolitinib1 | FEV1 <80%, >10% decrease in <2 years, VC <80%, FEV1/VC >0.7, absence of infection | 66 | 33 | 49 | Single arm | FEV1 (mL) | 2024 |
| Trial intervention . | Non-NIH BOS definition . | NIH BOS definition % . | Non-NIH BOS definition % . | N . | Trial design . | Response definition . | Year . |
|---|---|---|---|---|---|---|---|
| Etanercept9 | FEV1/FVC <0.75; trial amended after NIH criteria published | 88 (22/25 obstructive) | 22 | 34 | Single arm | FEV1% | 2012 |
| Montelukast7 | N/A | 100 | 25 | Single arm vs historical | Slope & FEV1% | 2022 | |
| FAM-inhaled fluticasone, azithromycin, montelukast8 | N/A | 100 | 36 | Single arm vs historical | Slope & FEV1% | 2016 | |
| Pirfenidone11 | CT with air trapping >28% or clinical improvement after treatment | 90.9 | 9.1 | 30 | Single arm | Slope of FEV1% | 2022 |
| Belumosudil12 | FEV1 ≤79%, clinician attribution to BOS | Unknown | 100 | 59 | Single arm | FEV1% | 2022 |
| Inhaled corticosteroid and long-acting beta agonist13 | FEV1 <80%, FEV1/FVC <5th percentile of CI, TLC ≥80% | Unknown | 100 | 32 | Randomized controlled | FEV1 (mL) | 2015 |
| Axatilimab10 | N/A | 100 | 16 | Single arm | FEV1% in 1 of 5 responders Symptoms in 4 of 5 responders | 2023 | |
| Ruxolitinib1 | FEV1 <80%, >10% decrease in <2 years, VC <80%, FEV1/VC >0.7, absence of infection | 66 | 33 | 49 | Single arm | FEV1 (mL) | 2024 |
The non-NIH BOS patients accrued in the etanercept trial were accrued prior to the development of the 2005 NIH BOS consensus criteria. Of note, the axatilimab trial included patients with other cGVHD manifestations (not just BOS). For the belumosudil and inhaled corticosteroid and long-acting beta agonist trials, all patients met the atypical BOS definition; what is unknown is how many of these patients would also have met the NIH-BOS definition at study entry.
CI, confidence interval; CT, computed tomography; FAM, fluticasone, azithromcyin, montelukast therapy; FEV1, forced expiratory volume at 1 second; N, total number of patients with BOS enrolled; N/A, not applicable; NIH, National Institutes of Health; TLC, total lung capacity; VC, vital capacity.