A Retrospective Non-Interventional Study to Evaluate the Impact of Eculizumab in Korean PNH Patients

Handok

Status: Completed

Conditions: PNH

Study type: Observational

Funder types: Industry

Identifiers

NCT05337683

HD_ECU_OS2020

Details and patient eligibility

About

This study is a retrospective non-interventional study, and the main objective of this study is to identify the impact of eculizumab treatment and the improvement of complications before and after eculizumab treatment, and to evaluate the safety during the treatment and the clinical unmet needs during the treatment in PNH patients in real-world.

Enrollment: 80 patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Patients with PNH aged 18 years or older who started eculizumab treatment in accordance with drug label between 2012 and January 31st, 2020. (However, patients who have received eculizumab as part of a clinical trial or an expanded access program/compassionate use program prior to 2012 are also eligible.)
• Patients who are vaccinated against Neisseria meningitidis at least 2 weeks prior to starting eculizumab treatment. Patients who have been treated with appropriate prophylactic antibiotic therapy for 2 weeks after vaccination if they received eculizumab within 2 weeks after the vaccination.

Exclusion criteria

• Pediatric patients with PNH aged under 18 years
• Patients with hypersensitivity to the active ingredients of eculizumab, murine protein, or other components
• Patients with untreated severe meningococcal (Neisseria meningitidis) infection
• Patients who received other complement inhibitors for PNH before or during eculizumab treatment