Real-World Prevalence and Outcomes of Patients with Paroxysmal Nocturnal Hemoglobinuria Treated with C5 Inhibitors in the US: A Retrospective Claims Database Analysis.

ABSTRACT:

Background

Paroxysmal nocturnal hemoglobinuria (PNH) is a rare blood disorder with C5 inhibitors (C5i), eculizumab and ravulizumab, being part of current treatment options.

Objectives

To estimate the 5-year prevalence of PNH and describe the healthcare resource utilization and direct healthcare costs associated with C5i among commercially insured patients with PNH treated with C5i in the US.

Methods

The 5-year prevalence of adults with PNH in IQVIA PharMetrics® Plus was estimated (2018-2022). A retrospective cohort study (2011-2022) was also conducted in adults with PNH treated with C5i and ≥3 months of continuous health plan coverage following the first claim for C5i (index date). PNH-related health resource utilization and direct healthcare costs were assessed from

SUBMITTER: Tantravahi SK 

PROVIDER: S-EPMC12358179 | biostudies-literature | 2025

REPOSITORIES: biostudies-literature