Table 3.
Summary of the clinical studies investigating efficacy and safety of ruxolitinib in COVID-19 patients.
| Study Reference | Study Design | No. Patients | Intervention Group | Comparator Group | Primary Outcome | Secondary Outcome | Safety Assessment |
|---|---|---|---|---|---|---|---|
| Cao et al., 2020 [31] | Randomized controlled, multicentre, single-blind (China) |
43 severe COVID-19 pneumonia |
Ruxolitinib 5 mg × 2/day + standard of care (antivirals + corticosteroids and supportive treatment) (n = 22) |
Placebo + standard of care (antivirals + corticosteroids and supportive treatment) (n = 21) |
Median time to clinical improvement: 12 days versus 15 days (p = 0.147) |
Significant improvement on chest CT scan at 14 days: 90% vs. 61.9% (p = 0.0495) 28-day mortality rate: 0.0% vs. 14.3% (p = 0.232) |
No difference in serious AEs |
| Giudice et al., 2020 [36] | Observational prospective, monocentric (Italy) |
17 severe COVID-19 pneumonia |
Ruxolitinib 10 mg × 2/day for 14 days + Eculizumab 900 mg/week (n = 7) |
Best available therapy (Hydroxychloroquine + Azithromycin + Heparin) (n = 10) |
Improvement in median PaO2 after 7 days: 94 versus 77 (p = 0.026) Improvement in median PaO2/FiO2 after 7 days: 370.5 versus 246 (p = 0.0395) |
No significant difference in mortality rate and duration of hospitalization | Increase in transaminase levels: 71.4% |
| Vannucchi et al., 2020 [41] | Observational prospective, monocentric (Italy) |
34 | Ruxolitinib 5–10 mg × 2/day | - | Clinical improvement in 85.3% of cases (reduction of at least 2 points in seven-point ordinal scale) Less frequent clinical improvement in patients with more severe respiratory impairment HR 0.31 (CI 0.1–1.0) |
CRP levels significantly decreased from a baseline median level of 72 mg/l (IQR, 39–111) to 26 mg/l (IQR, 5–76; p = 0.03) by day 7 and normalized by day 14 (12 mg/l, IQR, 6–21; p < 0.001) |
Discontinuation of treatment in 14.7% |
| Mortara et al., 2021 [42] | Observational prospective, monocentric (Italy) |
31 | Ruxolitinib 5 mg × 2/day for 15 days | - | Improvement in symptoms (Likert scale) at 7 and 15 days: 80.6% and 90.3% |
- | No AEs observed during treatment |
| Capochiani et al., 2020 [32] | Observational retrospective cohort, multicenter (Italy) |
18 ARDS due to COVID-19 |
Ruxolitinib 20 mg × 2/day in day 1–2, 5–10 mg × 2/day up to day 14 (n = 18) |
- | No evolution from NIV to mechanical ventilation: 88.9% Significant improvement in respiratory response within 48 h: 88.9% |
14-day complete respiratory function: 88.9% Rapid restoration within 48 h in PaO2/FiO2: 88.9% |
No AEs observed during treatment and at the follow-up |
| La Rosée et al., 2020 [39] | Observational retrospective, monocentric (Germany) |
14 severe COVID-19 pneumonia |
Ruxolitinib 7.5 mg × 2/day with subsequent reassessment for increase or decrease in dosage | - | Reduction by 25% in COVID-19 inflammatory score achieved after 5 days | - | One patient transient grade 3 liver toxicityTwo patients experienced grade 3 anaemia |
| Gaspari et al., 2020 [35] | Case report (Italy) |
2 | (a) Ruxolitinib 5 mg × 2/day on day 1–2 and 10 mg × 2/day on day 3–5 (b) Ruxolitinib 5 mg × 2/day on day 1–7 |
- | - | - | (a) Skin purpuric lesion associated with reduction in platelet count (b) Erythrodermic rash on whole body surface |
| Sammartano et al., 2020 [40] | Case report (Italy) |
1 | Ruxolitinib 20 mg × 2/day | Prior clinical failure with therapy including Hydroxychloroquine, Azithromycin, Corticosteroids, and Tocilizumab | COVID-19 related ARDS in a patient with diagnosis of Blastic Plasmocitoid Dendritic Cell Neoplasm Clinical improvement after 48 h with CPAP discontinuation |
- | - |
| Saraceni et al., 2021 [43] | Case report (Italy) |
1 | Ruxolitinib 5 mg × 2/day for chronic GVHD after allogeneic stem cell transplant, discontinued at hospital admission for COVID-19 pneumonia and re-started after clinical failure with standard of care | Hydroxychloroquine, Lopinavir-Ritonavir, and LMWH | Rapid improvement in respiratory function and hospital discharge at day 45 | - | No reported AEs |
| Innes et al., 2020 [37] | Case report (UK) |
1 | Ruxolitinib 5 mg × 2/day on day 1 × 3 and 10 mg × 2/day on day 4 × 21 | Prior clinical failure with intermediate dosage of LMWH and tocilizumab | Improvement in respiratory function and hospital discharge at day 28 | - | No reported AEs |
| Koschmieder et al., 2020 [38] | Case report (Germany) |
1 | Ruxolitinib 10 mg × 2/day chronic treatment (since 15 months) for myelofibrosis | - | ICU admission (no required mechanical ventilation) Rapid improvement in respiratory function and hospital discharge at day 15 |
- | No reported AEs |
| Caradec et al., 2020 [33] | Case report (France) |
1 | Ruxolitinib 10 mg × 2/day | Prior clinical failure with Hydroxychloroquine + Azithromycin | Improvement in respiratory function after 48 h and CRP normalization at day 8 | - | No reported AEs |
| Foss et al., 2020 [34] | Case report (USA) |
1 | Ruxolitinib 10 mg × 2/day for chronic GVHD after allogeneic stem cell transplant | - | Attenuated COVID-19 infection in an immunosuppressed patient in chronic treatment with ruxolitinib | - | - |
| Betelli et al., 2020 [30] | Case report (Italy) |
1 | Ruxolitinib 5 mg × 2/day for 14 days + Dexamethasone 20 mg/day for 5 day and subsequent decalage |
Prior clinical failure with standard of care (Hydroxychloroquine + Azithromycin) |
Oxygen supplementation suspended after 14 days Hospital discharge after 23 days |
- | - |
AE: adverse event; ARDS: acute respiratory distress syndrome; CI: confidence interval; CPAP: continuous positive airway pressure; CRP: C-reactive protein; CT: computed tomography; GVHD: graft-versus-host disease; HR: hazard ratio; IQR: interquartile range; LMWH: low-molecular weight heparin; and NIV: non-invasive ventilation.