Real-World Prevalence and Outcomes of Patients with Paroxysmal Nocturnal Hemoglobinuria Treated with C5 Inhibitors in the US: A Retrospective Claims Database Analysis
Journal of Health Economics and Outcomes Research, 2025
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 index date until earliest of treatment discontinuation/end of data/end of continuous health plan coverage (follow-up period).
Results
The 5-year prevalence of PNH was 2.4 per 100 000 persons in commercial claims. A total of 371 patients treated with C5i (median age: 40 years; female: 55.3%; eculizumab: 53.9%; ravulizumab: 46.1%) were followed for a mean ± SD [median] of 19.3 ± 16.9 [14.7] months. Annual incidence rates of PNH-related blood transfusion and breakthrough hemolysis (BTH) among patients treated with C5i were 1.2 (eculizumab: 1.3; ravulizumab: 1.0) and 4.5 (eculizumab: 5.2; ravulizumab: 3.3) per person per year (PPPY), respectively. In patients treated with eculizumab and ravulizumab, respectively, PNH-related blood transfusion was required by 46.2% and 11.9% of patients in the first 6 months post-index, and over the follow-up period, transfusion avoidance was observed in 46.2% and 78.2% of patients. The 6- and 12-month rates of PNH-related thrombosis were 8.0% and 10.6% for eculizumab and 6.1% and 11.6% for ravulizumab, respectively. Among patients treated with C5i, estimated annual total PNH-related costs PPPY were $660,533 (eculizumab: $697,459; ravulizumab: $612,522) for the first year and $633,984 (eculizumab: $691,022; ravulizumab: $570,832) for subsequent years, with treatment costs accounting for 94.3% to 94.6% of total costs.
Discussion
Despite treatment with C5i, patients with PNH still exhibited BTH, required blood transfusions, and experienced thrombosis.
Conclusion
This study highlights the unmet need for more effective PNH treatments to address the economic and clinical burden associated with PNH and improve disease control among patients.
Authors
Tantravahi SK, Latremouille-Viau D, Desai R, Lee S, Paulose J, Geevarghese A, Guérin A, Seshasayee S, Chanpura M, Yen G