Tocilizumab for patients with COVID-19 pneumonia. The single-arm TOCIVID-19 prospective trial

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Perrone Francesco, Carmela Piccirillo Maria, Antonio Ascierto Paolo, Salvarani Carlo, Parrella Roberto, Maria Marata Anna, Popoli Patrizia, Ferraris Laurenzia, Marrocco-Trischitta Massimiliano M., Ripamonti Diego, Binda Francesca, Bonfanti Paolo, Squillace Nicola, Castelli Francesco, Lorenza Muiesan Maria, Lichtner Miriam, Calzetti Carlo, Duccio Salerno Nicola, Atripaldi Luigi, Cascella Marco Massari Marco, Montesarchio Vincenzo, Mussini Cristina, Alberto Negri Emanuele, Botti Gerardo, Costantini Massimo, Dolci Giovanni, Cosimo Facciolongo Nicola, Fraganza Fiorentino, Cardone Claudia, Gargiulo Piera, Gravina Adriano, Schettino Clorinda, Arenare Laura, Chiodini and Ciro Gallo17 on behalf of the TOCIVID-19 investigators Paolo, Italy, Rocco Monica
ISSN: 1479-5876

Background: Tocilizumab blocks pro-inflammatory activity of interleukin-6 (IL-6), involved in pathogenesis of pneu- monia the most frequent cause of death in COVID-19 patients.
Methods: A multicenter, single-arm, hypothesis-driven trial was planned, according to a phase 2 design, to study the effect of tocilizumab on lethality rates at 14 and 30 days (co-primary endpoints, a priori expected rates being 20 and 35%, respectively). A further prospective cohort of patients, consecutively enrolled after the first cohort was accom- plished, was used as a secondary validation dataset. The two cohorts were evaluated jointly in an exploratory multi- variable logistic regression model to assess prognostic variables on survival.
Results: In the primary intention-to-treat (ITT) phase 2 population, 180/301 (59.8%) subjects received tocilizumab, and 67 deaths were observed overall. Lethality rates were equal to 18.4% (97.5% CI: 13.6–24.0, P = 0.52) and 22.4% (97.5% CI: 17.2–28.3, P < 0.001) at 14 and 30 days, respectively. Lethality rates were lower in the validation dataset, that included 920 patients. No signal of specific drug toxicity was reported. In the exploratory multivariable logistic regres- sion analysis, older age and lower PaO2/FiO2 ratio negatively affected survival, while the concurrent use of steroids was associated with greater survival. A statistically significant interaction was found between tocilizumab and respira- tory support, suggesting that tocilizumab might be more effective in patients not requiring mechanical respiratory support at baseline.
Conclusions: Tocilizumab reduced lethality rate at 30 days compared with null hypothesis, without significant toxic- ity. Possibly, this effect could be limited to patients not requiring mechanical respiratory support at baseline.

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