Cytokine storm induced by COVID-19 infection has an increased risk for mortality and may lead to severe complications requiring intensive care, intubation, or mechanical ventilation. Ruxolitinib is a JAK1/JAK2 inhibitor currently approved for the treatment of myelofibrosis and acute graft-versus-host disease. The efficacy and safety of adding ruxolitinib to standard of care (SoC) for patients with COVID-19-associated cytokine storm versus placebo plus SoC has been under investigation in a phase III, randomized, multi-center RUXCOVID study (NCT04362137). The initial results of this trial, which is now completed, did not demonstrate a clinical benefit in favor of ruxolitinib.1
Eligible patients were given an oral dose of 5 mg ruxolitinib twice daily for 14 days and were followed for 29 days following randomization.
The study failed to meet its primary endpoint of improving the proportion of patients who experienced severe COVID-19-related complications such as death, respiratory failure requiring mechanical ventilation, or admission to intensive care unit. The decrease in rate was very similar among the two comparator arms by Day 29 (12.0% in the ruxolitinib arm versus 11.8% in the placebo arm; odds ratio, 0.91; 95% CI, 0.48–1.73; p = 0.769). There was no clinical benefit in terms of reduced mortality rate by end of the follow-up period or any improvement in time to recovery in the experimental arm. The safety profile of ruxolitinib in this setting was acceptable, with good tolerability.