Effectiveness and safety of tocilizumab, an anti-interleukin-6 receptor antibody, in 342 Japanese patients with multicentric Castleman disease: a 3-year, prospective, observational study.

Abstract

Objectives: Multicentric Castleman disease (MCD) is a rare lymphoproliferative disorder characterized by multifocal lymphadenopathy and systemic inflammatory symptoms. This single-arm, prospective, observational, postmarketing surveillance (UMIN000023071) assessed the real-world effectiveness and safety of tocilizumab in Japanese patients with MCD.

Methods: Patients with MCD received tocilizumab intravenous infusion 8 mg/kg every 2 weeks. Effectiveness outcomes included enlarged lymph node regression; improvement in laboratory findings, associated symptoms, and organ involvement; and change in corticosteroid doses. Safety was assessed through adverse events (AEs).

Results: A total of 342 patients were included. Tocilizumab was continued for > 152 weeks in 70.1% of patients. After treatment, 58.9% of patients showed lymph node regression. Laboratory test values were improved, and most evaluable patients reported improvements in associated symptoms, including night sweats, anorexia, fever, and fatigue. Organ involvement, including pulmonary disease, rash, and hepatosplenomegaly, was 'improved' or 'stable' in more than half of evaluable patients. Tocilizumab treatment resulted in corticosteroid dose reduction. The most commonly reported AEs were infections and infestations; respiratory, thoracic, and mediastinal disorders; and gastrointestinal disorders.

Conclusions: The effectiveness and safety of tocilizumab in Japanese patients with MCD was confirmed for up to 3 years, without new safety concerns.