FDA benefit/risk assessment
| Dimension . | Evidence . | Conclusions . |
|---|---|---|
| Condition | SR-aGVHD potentially lethal | Poor prognosis of SR-aGVHD |
| Current option | No approved therapies; drugs often used off-label with low evidence of efficacy | Clinical medical need |
| Benefit | In NCT02953678, day 28 ORR was 57% and DOR 0.5 mo based on the analysis of 49 patients | The magnitude of ORR and DOR supports the use of ruxolitinib |
| Risks | Safety analysis included 71 patients; fatal infection occurred in 14% and fatal hemorrhage in 4%; the most common AEs were expected | Potential risks of cytopenias, infections, and hemorrhage can be mitigated |
| Dimension . | Evidence . | Conclusions . |
|---|---|---|
| Condition | SR-aGVHD potentially lethal | Poor prognosis of SR-aGVHD |
| Current option | No approved therapies; drugs often used off-label with low evidence of efficacy | Clinical medical need |
| Benefit | In NCT02953678, day 28 ORR was 57% and DOR 0.5 mo based on the analysis of 49 patients | The magnitude of ORR and DOR supports the use of ruxolitinib |
| Risks | Safety analysis included 71 patients; fatal infection occurred in 14% and fatal hemorrhage in 4%; the most common AEs were expected | Potential risks of cytopenias, infections, and hemorrhage can be mitigated |
Adapted from Table 7 in Przepiorka et al24 with permission.
AE, adverse event; DOR, duration of response.