Real-World Treatment of Patients with Severe Congenital Protein C Deficiency with Protein C Concentrate: A Physician Survey

Authors: Wang, M.; Hertfelder, H.-J.; Swallow, E.; Greatsinger, A.; Billmyer, E.; Fung, S.; Turecek, P. L.; Gazda, H. T.; Siffel, C.

Affiliations: Global Evidence and Outcomes (GEO) Lead, GEO, Data Sciences Institute, Takeda Pharmaceutical Company Limited, 650 East Kendall St, Cambridge, MA, United States.

Publication: Thrombosis Update. 2024. 14

ABSTRACT: INTRODUCTION: Replacement therapy with intravenous (IV) protein C concentrate (Ceprotin®; Baxalta US Inc., a Takeda company, Lexington, MA, USA; Takeda Manufacturing Austria AG, Vienna, Austria) is an approved treatment approach for patients with severe congenital protein C deficiency (SCPCD). Data on the real-world use of protein C concentrate are limited. MATERIALS AND METHODS: In this non-interventional real-world study, physicians in Europe and the United States (US) with experience using protein C concentrate to treat patients with SCPCD completed an internet-based survey. Information collected included physician clinical practice details, experience treating patients with SCPCD, and opinions on the subcutaneous (SC) administration of protein C concentrate. Physicians responded based on their best recall. RESULTS: The analysis included 19 physicians (Europe, n = 12; US, n = 7) who had used protein C concentrate to treat 32 patients with SCPCD. Sixteen patients received IV long-term prophylaxis (LTP; treatment duration ≥3 months) and 12 received SC LTP. Five patients received both IV and SC LTP. Eighteen physicians indicated an interest in adding SC administration to the approved administration routes. CONCLUSION: This survey shows that LTP with IV protein C concentrate is used in clinical practice by physicians in both Europe and the US for the treatment of patients with SCPCD. Protein C concentrate is also prescribed for SC administration by some physicians in Europe. Although only approved for IV administration, physicians in both Europe and the US indicated an interest in SC administration being an approved administration route for protein C concentrate.