Efficacy and Safety of Tocilizumab for Acute Chest Syndrome Treatment in Pediatric and Adult Patients With Sickle Cell Disease
The purpose of this study is to determine whether a single infusion of tocilizumab is effective in reducing the time to successful weaning from both supplemental oxygen and any respiratory support, in pediatric and adult patients with sickle cell disease (SCD) during acute chest syndrome (ACS).
• SCD patient of all genotypes (SS, SC, S/β0 and S/β+ or other major SCD syndrome)
• Age ≥ 2 years old
• Hospitalized for ACS, defined by the WHO as the association of fever and/or acute respiratory symptoms with a new pulmonary infiltrate on chest imaging, (X-ray, lung ultrasound, or CT scan)
• Requiring supplemental oxygen ≥ 2 L/min for SpO2 ≥ 95% or non-invasive respiratory support (high flow nasal oxygen or continuous positive airway pressure or bilevel non-invasive ventilation) or invasive mechanical ventilation or ECMO, for less than 48 hours
• Negative pregnancy test for girls or women of childbearing age
• Freely given, informed and written consent of patient or legal representatives
• Affiliation to the social security (or health insurance)
• Effective contraception up to 3 months after the administration of treatment (tocilizumab or placebo)