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

Assistance Publique - Hôpitaux de Paris92 enrolled

Overview

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.

Study Type

OBSERVATIONAL

Enrollment

Conditions

Paroxysmal Nocturnal Hemoglobinuria

Interventions

Anti-C5 antibody treatmentOTHER

Anti-C5 antibody treatment (i.e., eculizumab and ravulizumab)

Eligibility

Sex: ALLMin age: 18 Years

Medical Language ↔ Plain English

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

Outcomes

Primary Outcomes

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

Time frame: between Day 100 and Day 200

Secondary Outcomes

Hemoglobin Response

Achieving hemoglobin of ≥ 12 g/dL in the absence of blood transfusions occurring after Day 14 post index date

Time frame: 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).

Time frame: Occurrence between Day 15 and Day 200