Real-world use of protein C concentrate for the treatment of patients with protein C deficiency: an international registry.

Abstract

Background: Published reports on the real-world use of protein C concentrate have been limited to case reports or small case series.

Objectives: To collect and assess data on the medical diagnoses, treatment regimens, safety outcomes, and treatment outcomes of patients receiving protein C concentrate in routine clinical practice.

Methods: This was a prospective, open-label, registry-based study conducted at 26 sites in Europe and the USA. Data were collected from the medical records of patients of all ages who received treatment with protein C concentrate. The primary endpoints were medical diagnoses associated with protein C concentrate treatment, treatment regimens, and the safety of protein C concentrate (incidence of related adverse events [AEs] and all serious AEs [SAEs]).

Results: Between June 23, 2010, and June 22, 2015, 43 patients, including 25 with severe congenital protein C deficiency (SCPCD) and 18 with severe acquired protein C deficiency, were enrolled in the study, all of whom were included in the analysis. In total, 306 treatment courses of protein C concentrate were documented during the study, for treatment of acute thrombotic episodes, short-term replacement, and long-term prophylaxis. Most patients (76%) with SCPCD were receiving long-term prophylaxis. Overall, 124 AEs were reported in 27 patients (63%); 17 patients experienced SAEs. Only 2 AEs (SAEs of abdominal pain and lower extremity pain) in 1 patient were considered possibly related to protein C concentrate.

Conclusion: The results of this study provide insights into the real-world use of protein C concentrate in clinical practice in Europe and the USA.