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| Name | Class |
|---|---|
| Leeds Cancer Centre at St. James's University Hospital | OTHER |
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The aim of this non-interventional secondary use of data study is to evaluate hematological response in patients with paroxysmal nocturnal hemoglobinuria and anemia in the 6-month period after initiation of anti-C5 antibody treatment using real-world data obtained from multiple datasets. The results will be used to contextualize results from the APPOINT-PNH (NCT04820530) trial with iptacopan.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Real-world cohort | The real-world cohort consists of patients with diagnosis of paroxysmal nocturnal hemoglobinuria and anemia initiating anti-C5 therapy. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Anti-C5 antibody treatment | Other | Anti-C5 antibody treatment (i.e., eculizumab and ravulizumab) |
|
| Measure | Description | Time Frame |
|---|---|---|
| Increase from baseline in hemoglobin | Increase from baseline in hemoglobin of ≥ 2 g/dL in the absence of transfusions occurring after Day 14 post index date | between Day 100 and Day 200 |
| Measure | Description | Time Frame |
|---|---|---|
| Hemoglobin Response | Achieving hemoglobin of ≥ 12 g/dL in the absence of blood transfusions occurring after Day 14 post index date | between Day 100 and Day 200 |
| Transfusion avoidance | Transfusion avoidance (dichotomous endpoint) defined proportion of subjects without transfusions during the initial 6-month treatment period starting from 2 Weeks after index date (i.e., date of first anti-C5 antibody treatment for PNH). |
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Inclusion Criteria:
Exclusion Criteria:
Patients who met any of the following criteria were not eligible for inclusion.
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The real-world cohort consists of patients with diagnosis of paroxysmal nocturnal hemoglobinuria and anemia initiating anti-C5 therapy. Patient data originate from the Hôpital Saint-Louis, France and St. James's University Hospital, Leeds, United Kingdom.
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| AP-HP Hôpital Saint Louis | Paris | France |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 40395624 | Derived | Holt M, Kelly RJ, Fermont JM, Ansari S, Dahlke M, Brindel I, Maafa L, de Fontbrune FS, de Latour RP. Effectiveness of Iptacopan Versus C5 Inhibitors in Complement Inhibitor-Naive Patients With Paroxysmal Nocturnal Haemoglobinuria. EJHaem. 2025 May 20;6(3):e270055. doi: 10.1002/jha2.70055. eCollection 2025 Jun. |
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| Occurrence between Day 15 and Day 200 |
| ID | Term |
|---|---|
| D006457 | Hemoglobinuria, Paroxysmal |
| ID | Term |
|---|---|
| D000743 | Anemia, Hemolytic |
| D000740 | Anemia |
| D006402 | Hematologic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
| D009190 | Myelodysplastic Syndromes |
| D001855 | Bone Marrow Diseases |
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