Real-World Tocilizumab Use in Pediatric Inpatients

Primary objective: to quantify tocilizumab (TCZ) use in pediatric inpatients. Secondary objectives: to explore safety and clinical outcomes.

This retrospective cohort study took place in a free-standing, 564-bed children's hospital. Pediatric inpatients who received intravenous TCZ from January 2016 to May 2021 were included. Data collected included demographics, indication, dose, number of administrations, safety events on days 0 to 7 after TCZ, use of extracorporeal support (ES), presence of concurrent infection, and survival to discharge. Exploratory analyses assessed characteristics associated with mortality.

A total of 103 TCZ courses (n = 87 patients) were analyzed. Median age was 14 years. Tocilizumab indications included cytokine release syndrome (CRS; 56%), autoimmune disease (27%), graft-versus-host disease (GVHD; 5%), and COVID-19 (4%). The median TCZ dose was 8 mg/kg (IQR, 7.9-11.9), 18% of courses were administered during active infection, and ES was used in 15% of courses. New-onset alanine transaminase (ALT) or aspartate transaminase (AST) levels >3 times upper limit of normal (ULN) occurred in 53% and 60% of courses, respectively. Of 29 courses with evaluable hematologic data, 10% resulted in new-onset neutropenia and 3% in severe thrombocytopenia. Overall survival to discharge was 83%. In multivariable analyses, independent associations with mortality were found for the use of ES (OR, 8.68; 95% CI, 1.85-4.87), oncologic diagnosis (OR, 7.07; 95% CI, 1.14-89.29), and post-TCZ infection (OR, 11.17; 95% CI, 1.50-138.13).

Tocilizumab is used for many pediatric inpatient indications, most commonly CRS. Newly identified transaminitis was common following TCZ administration. Risk factors for mortality are likely confounded by illness severity. Administration during active infection was not independently associated with increased mortality.