The implementation of new standards for the management of PNH and the use of iptacopan in patients with PNH are expected to change the treatment landscape and improve the overall prognosis of patients.
Based on these backgrounds, we plan to conduct a real-world study of iptacopan to further evaluate its impact on treatment-related outcomes, disease management, and healthcare resource utilization in Chinese patients with PNH.
Age ≥ 18 years at the time of signing the ICF;
Patient with a documented diagnosis of PNH;
Patient who has never received complement inhibitor therapy;
Patient who is initiating iptacopan therapy; Patient who is initiating iptacopan therapy must start the first dose within 60 days of signing the ICF;
Participating in an interventional PNH clinical study;
Have an active systemic bacterial, viral (incl. COVID-19) or fungal infection within 14 days prior to first dose;
Documented with a history of recurrent invasive infections, e.g. active systemic bacterial, viral or fungal infection within 14 days prior to first dose;
Documented with a history of HIV infection;
Adult PNH patients receiving iptacopan for the first time, including those who are complement inhibitor naive and treated
Cohort
Cross-sectional
LNP023
DrugCapsules for oral administration
Change from baseline in hemoglobin (Hb) levels at designated time points
Assessment of the Hematological Response to iptacopan.
Number of participants having Hb normalization (Hb level ≥ 12 g/dL) after iptacopan initiation.
Assessing the Impact of iptacopan on Treatment-Related Outcomes
Duration of Hb level ≥ 12 g/dL within a 12-month
Assessing the Impact of iptacopan on Treatment-Related Outcomes
Change from baseline in LDH Levels after iptacopan initiation
Assessing the Impact of iptacopan on Treatment-Related Outcomes
Number of participants with LDH levels ≤ 1.5 x ULN before vs. after iptacopan initiation
Assessing the Impact of iptacopan on Treatment-Related Outcomes
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