APPEX: Hematological Response in Patients With Paroxysmal Nocturnal Hemoglobinuria Treated With Anti-C5 Antibody: an External Control Arm Study for Iptacopan Use in Anti-C5 naïve Patients
Study Details
Study Description
Brief Summary
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.
| Condition or Disease | Intervention/Treatment | Phase |
|---|---|---|
|
Study Design
Arms and Interventions
| Arm | Intervention/Treatment |
|---|---|
| Real-world cohort The real-world cohort consists of patients with diagnosis of paroxysmal nocturnal hemoglobinuria and anemia initiating anti-C5 therapy. |
Other: Anti-C5 antibody treatment
Anti-C5 antibody treatment (i.e., eculizumab and ravulizumab)
|
Outcome Measures
Primary Outcome Measures
- Increase from baseline in hemoglobin [between Day 100 and Day 200]
Increase from baseline in hemoglobin of ≥ 2 g/dL in the absence of transfusions occurring after Day 14 post index date
Secondary Outcome Measures
- Hemoglobin Response [between Day 100 and Day 200]
Achieving hemoglobin of ≥ 12 g/dL in the absence of blood transfusions occurring after Day 14 post index date
- Transfusion avoidance [Occurrence between Day 15 and Day 200]
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).
Eligibility Criteria
Criteria
Inclusion Criteria:
-
Incident users of anti-C5 antibody treatment (i.e., first use of anti-C5 antibody with no evidence of use prior to that)
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Confirmed clinical diagnosis of PNH (including PNH clone size >10% in RBC percent/white blood cell (WBC) percent)
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Adult patients ≥18 years of age
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Baseline Hb <10 g/dL.
Exclusion Criteria:
Patients who met any of the following criteria were not eligible for inclusion.
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Bone marrow transplantation within one year prior to initiating anti-C5 treatment
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Hereditary complement deficiency
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Reticulocyte count <60x10^9/L.
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Pregnancy
Contacts and Locations
Locations
| Site | City | State | Country | Postal Code | |
|---|---|---|---|---|---|
| 1 | AP-HP Hôpital Saint Louis | Paris | France |
Sponsors and Collaborators
- Assistance Publique - Hôpitaux de Paris
- Leeds Cancer Centre at St. James's University Hospital
Investigators
None specified.Study Documents (Full-Text)
None provided.More Information
Publications
None provided.- CLNP023N12002R