Claims Data Show Problematic Treatment Patterns, Resource Utilization in PNH

The majority of newly diagnosed patients with paroxysmal nocturnal hemoglobinuria (PNH) did not receive a PNH-specific therapy at diagnosis, and even patients who were treated had delays between diagnosis and treatment initiation, according to a claims database analysis published in Clinical and Applied Thrombosis/Hemostasis.

“Nearly one in five newly diagnosed PNH patients still require at least one blood transfusion, signaling some unmet medical need in PNH,” reported the study’s authors, led by Denise Clayton, PhD, from the Center for Healthcare Economics and Policy at FTI Consulting in Washington, DC.

The analysis enrolled 271 patients from the MarketScan health insurance claims database. The cohort was 57.9% female and had an average age of 46.6 years. At the time of diagnosis, only 25.1% (n=68) of patients received a C5 inhibitor with an average time from diagnosis to treatment of 4.7 months.

Among treated patients, the medication possession ratio was 97.0%, but 58.8% of patients discontinued treatment. Despite availability of treatments, 39.9% of the cohort had an inpatient stay and 50.9% had an emergency department visit. Researchers estimated the average per-patient-per-month cost in the cohort was $18,978, largely due to pharmacy and infusion costs at $11,182, followed by outpatient costs at $4,086, and inpatient costs at $3,318.

Overall, “better care management and the introduction of new treatment options are needed to address delays between diagnosis and treatment, and high rates of hospitalization and emergency department use among patients with PNH,” suggested Dr. Clayton and colleagues.

Reference

Clayton D, Shafrin J, Yen G, et al. Treatment patterns and healthcare resource utilization of patients with paroxysmal nocturnal hemoglobinuria: a retrospective claims data analysis. Clin Appl Thromb Hemost. 2024;30:10760296231213073. doi:10.1177/10760296231213073