Real-world Study of Iptacopan Treatment in Italian Adults with Paroxysmal Nocturnal Hemoglobinuria

What this Trial Is About

This observational study focuses on adult patients with Paroxysmal Nocturnal Hemoglobinuria PNH in Italy who are treated with iptacopan. The research aims to evaluate the long-term hematological response to iptacopan, including changes in hemoglobin levels and avoidance of red blood cell transfusions over 12 months. The study uses real-world clinical data to better understand treatment outcomes in routine practice. Participants include those new to iptacopan treatment as well as patients transitioning from a Managed Access Program. The study collects data retrospectively, prospectively, and retro-prospectively, following patients from the start of iptacopan treatment for up to 24 months or until treatment discontinuation or other endpoints occur. No additional visits or procedures beyond routine care are required, though questionnaires may be collected during scheduled clinical visits. During the study, researchers will monitor hemoglobin levels, transfusion needs, adverse events, and patient-reported outcomes using validated questionnaires. Data on hospitalizations, infections, vascular events, medication adherence, and quality of life will also be collected. This approach allows assessment of iptacopans effects and safety in a real-world setting without altering standard medical care.

REAL-CARE: Real-world Effectiveness of Iptacopan in Italian Patients With Paroxysmal Nocturnal Hemoglobinuria