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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.
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| Measure | Description | Time Frame |
|---|---|---|
| Decrease of hemolysis after eculizumab treatment | The change in LDH levels before and after eculizumab treatment will be compared to determine whether hemolysis decreased after the treatment. | The results of LDH tests performed before eculizumab treatment, at 6, 12, 24 months after the start of eculizumab treatment, and every 12 months thereafter will be collected. |
| The Improvement/occurrence of complications and related clinical symptoms before and after eculizumab treatment | The major complications due to PNH include thromboembolism, pulmonary hypertension, renal failure, and smooth muscle spasm, and their occurrence will be determined by the investigator. In addition, PNH-related symptoms (fatigue, anemia, dysphagia, shortness of breath/dyspnea, chest pain, abdominal pain, erectile dysfunction, hemoglobinuria, others) before and after eculizumab administration will be compared. | 6 months before eculizumab administration to the end of the administration (It is up to subject's treatment period. Through study completion, an average of 51months) |
| Severity of anemia | Hb levels will be compared to identify the severity of anemia. | Before the administration, at 6, 12, 24 months after the start of the treatment and every and 12 months thereafter |
| The required Unit of pRBC transfusion before and after eculizumab treatment | From 1 year before the treatment to the end of the administration (It is up to subject's treatment period. Through study completion, an average of 51months) | |
| Effectiveness Evaluation at Last Follow-Up | The investigator will perform the final evaluation of the efficacy for entire observation period based on the collected data. The investigator will comprehensively judge and evaluate the decrease of hemolysis occurrence, the improvement and occurrence of complications, and the severity of anemia, etc. |
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Inclusion Criteria:
Exclusion Criteria:
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Patients with PNH aged 18 years or older who started receiving eculizumab treatment accordance with the drug label until January 31, 2020 will be enrolled in this study.
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Handok | Seoul | South Korea |
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| At the last follow-up (At the time of data collection retrospectively) |