A Multi-center, Ambispective Cohort Study to Evaluate the Impact of Iptacopan for Adult Patients With PNH in China

A Multi-center, Ambispective Cohort Study to Evaluate the Impact of Iptacopan on Disease Management, Treatment-Related Outcomes and Healthcare Resource Utilization for Adult Patients With Paroxysmal Nocturnal Hemoglobinuria in China

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.

Inclusion criteria For Cohort 1,

Patient who meets all the following criteria can be included in this study:

1. Age ≥ 18 years at the time of signing the ICF;
2. Patient with a documented diagnosis of PNH;
3. Patient who has never received complement inhibitor therapy;
4. Patient who is initiating iptacopan therapy; Patient who is initiating iptacopan therapy must start the first dose within 60 days of signing the ICF;
5. Documented vaccination against Neisseria meningitidis and Streptococcus pneumoniae and the date of vaccination must be at least 2 weeks prior to the date of iptacopan initiation; If an urgent prescription for Iptacopan is needed, it is recommended that the antibiotic be used continuously according to the drug label until 14 days after the vaccination is completed, and that the vaccination be completed as soon as possible.
6. Patient who has signed the ICF.

For Cohort 2,

Patient who meets all the following criteria can be included in this study:

1. Age ≥ 18 years at the time of signing the ICF;
2. Patient with a documented diagnosis of PNH; Patients who have been receiving stable treatment with C5 complement inhibitors for at least three months prior to enrollment;
3. Patient who is initiating iptacopan therapy; Patient who is initiating iptacopan therapy must start the first dose within 60 days of signing the ICF;
4. Documented vaccination against Neisseria meningitidis and Streptococcus pneumoniae and the date of vaccination must be at least 2 weeks prior to the date of iptacopan initiation;
5. If an urgent prescription for Iptacopan is needed, it is recommended that the antibiotic be used continuously according to the drug label until 14 days after the vaccination is completed, and that the vaccination be completed as soon as possible.
6. Patient who has signed the ICF. Exclusion criteria

For Cohort 1 and Cohort 2, patients who meet any of the following criteria will meet the exclusion criteria for this study:

1. Participating in an interventional PNH clinical study;
2. Have an active systemic bacterial, viral (incl. COVID-19) or fungal infection within 14 days prior to first dose;
3. Documented with a history of recurrent invasive infections, e.g. active systemic bacterial, viral or fungal infection within 14 days prior to first dose;
4. Documented with a history of HIV infection;
5. Women who are pregnant or breastfeeding or intending to conceive during the study period;
6. Existence of bone marrow failure (reticulocytes < 100 × 109/L, platelets < 30 × 109/L, and neutrophils < 0.5 × 109/L) determined by the investigator;
7. Other conditions that are not suitable for participating in the study, in the judgment of the investigator.