Patients receiving C5 inhibitors for paroxysmal nocturnal hemoglobinuria (PNH) continue to experience breakthrough hemolysis and require transfusional support, suggesting that these therapeutics are inadequate for disease control, according to a study published in the Journal of Managed Care + Specialty Pharmacy. 

The researchers sought to characterize real-world treatment patterns and outcomes associated with the use of C5 inhibitors in the context of PNH. They conducted a retrospective study by identifying patients from Prime Therapeutics’ database of commercially insured US members between January 1, 2018 and December 31, 2020, a period prior to the Food and Drug Administration (FDA) approval of pegcetacoplan.

The database allowed researchers to track real-world treatment patterns, including discontinuation and switching of medications. The research team identified 86 patients who fit the inclusion criteria; 34 were in the eculizumab cohort while 52 were prescribed ravulizumab.

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A total of 47.2% of patients receiving eculizumab were treatment-naïve and all patients in the ravulizumab cohort were treatment-naïve. Researchers also reported that the maintenance dose in some individuals exceeded that recommended by the FDA; this was true in 38.2% of patients treated with eculizumab and 32.7% of patients administered ravulizumab. 

However, both cohorts continued to experience adverse events. A total of 29.4% of individuals receiving eculizumab and 13.5% of patients in the ravulizumab cohort experienced at least 1 episode of breakthrough hemolysis. Transfusional support was needed in 52.9% of patients receiving eculizumab and 32.7% of patients receiving ravulizumab.

The mean total annual cost of treatment was $711,785 in the eculizumab group and $624,911 in the ravulizumab group, with C5 inhibitor costs accounting for most of the expenditure. 

“With these high PNH drug therapy costs and frequent inadequate clinical outcomes, it is imperative managed care pharmacists remain vigilant in monitoring these members to aid in optimizing cost-effective pharmacotherapy,” the authors of the study concluded. 

Disclaimer: The development of this manuscript was supported by Nicholas Redman and Rachel Mann, employees of Labcorp Drug Development, Inc.


Broderick KC, Burke JP, Fishman J, Gleason PP. Descriptive, real-world treatment patterns, resource use, and total cost of care among eculizumab- and ravulizumab-treated members with paroxysmal nocturnal hemoglobinuriaJ Manag Care Spec Pharm. 2023;29(8):941-951. doi:10.18553/jmcp.2023.29.8.941