Paroxysmal Nocturnal Hemoglobinuria
Conditions
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.
Interventions
OTHERAnti-C5 antibody treatment
Anti-C5 antibody treatment (i.e., eculizumab and ravulizumab)
Sponsors
Leeds Cancer Centre at St. James's University Hospital
Assistance Publique - Hôpitaux de Paris
Study design
Observational model
COHORT
Time perspective
RETROSPECTIVE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
1. Incident users of anti-C5 antibody treatment (i.e., first use of anti-C5 antibody with no evidence of use prior to that) 2. Confirmed clinical diagnosis of PNH (including PNH clone size \>10% in RBC percent/white blood cell (WBC) percent) 3. Adult patients ≥18 years of age 4. Baseline Hb \<10 g/dL.
Exclusion criteria
Patients who met any of the following criteria were not eligible for inclusion. 1. Bone marrow transplantation within one year prior to initiating anti-C5 treatment 2. Hereditary complement deficiency 3. Reticulocyte count \<60x10\^9/L. 4. Pregnancy
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| 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
| Measure | Time frame | Description |
|---|---|---|
| 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). |
Countries
France
Outcome results
None listed