This study aimed to evaluate the efficacy and safety of Lanoracopan Hydrochloride in the treatment of Paroxysmal Nocturnal Hemoglobinuria (PNH)
In Chinese multicenter phase 3 trials, lanoracopan 400 mg BID was evaluated over 24 weeks. In the active-controlled study (N=66, complement-naïve), the proportion achieving Hb ≥120 g/L was 50.0% (lanoracopan) versus 9.09% (eculizumab). In the single-arm study (N=20, C5 inhibitor-inadequate responders), 70% reached Hb ≥120 g/L, and 100% had Hb increase ≥20 g/L from baseline. No treatment-discontinuation due to AEs, no severe breakthrough hemolysis, no major vascular events, no encapsulated bacterial infections, and no deaths were reported across both studies. These data demonstrate superior hemoglobin correction and favorable short-term safety. However, long-term real-world effectiveness and safety profiles stratified by prior treatment (C5 inhibitors vs. other factor B inhibitors) remain uncharacterized. This observational study will enroll a diverse cohort to capture durability of Hb response, transfusion independence rate, incidence of breakthrough hemolysis and thrombosis, and adverse event patterns over extended follow-up, providing evidence to guide therapy switching and sequencing in clinical practice.
Study Type
OBSERVATIONAL
Enrollment
90
400mg bid
rate of adverse events (AEs)
According to CTCAE V5.0
Time frame: During treatment, an average of 24 weeks
Proportion of subjects maintaining Hb ≥120 g/L
During the treatment observation period, assess the proportion of subjects maintaining Hb ≥120 g/L every 12 weeks without red blood cell transfusion
Time frame: 12 weeks, 24 weeks
changes in hemoglobin (Hb) levels
During the treatment observation period, evaluate changes in Hb levels every 12 weeks without red blood cell transfusion.
Time frame: 12 weeks, 24 weeks
incidence of clinically significant hemolysis
During the treatment observation period, assess the incidence of clinically significant hemolysis every 12 weeks.
Time frame: 12 weeks, 24 weeks
proportion of subjects experiencing a major adverse vascular event (MAVE)
During the treatment observation period, assess the proportion of subjects experiencing a MAVE every 12 weeks.
Time frame: 12 weeks, 24 weeks
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