PB1031 - Real-world treatment of severe congenital protein C deficiency with protein C concentrate including prophylaxis: A physician survey in Europe and the US

Tuesday, July 12, 2022

6:30 PM – 7:30 PM

Location: Exhibition

Presentation Format: In-Person

Presenting Author(s)

Michael Wang, MD

HTC Clinical Director
University of Colorado Denver Hemophilia and Thrombosis Center
Aurora, Colorado, United States

Co-Author(s)

CS

Csaba Siffel

Director Pharmacoepidemiology
Takeda Pharmaceutical Company Limited
Lexington, Massachusetts, United States
Peter L. Turecek, PhD, PharmD

Professor
Baxalta Innovations GmbH, a Takeda company
Vienna, Wien, Austria
HG

Hanna Gazda

Medical Director
Takeda Pharmaceutical Company Limited
Lexington, Massachusetts, United States
SF

Samson Fung

Managing Director
Fung Consulting
Eching, Bayern, Germany
ES

Elyse Swallow

Vice President
Analysis Group, Inc.
Boston, Massachusetts, United States
AG

Alexandra Greatsinger

Senior Analyst
Analysis Group, Inc.
Boston, Massachusetts, United States
EB

Emma Billmyer

Senior Analyst
Analysis Group, Inc.
Boston, Massachusetts, United States
HH

Hans-Jörg Hertfelder

Deputy Medical Director
Center for Blood Coagulation and Transfusion Medicine, University of Bonn
Bonn, Nordrhein-Westfalen, Germany

Background: Severe congenital protein C deficiency (SCPCD) is a rare, autosomal recessive disorder associated with thromboembolic events. Protein C concentrate (Baxalta US Inc., a Takeda company, Lexington, MA, USA) administered intravenously is approved for long-term prophylaxis (LTP) in the US and short-term prophylaxis (STP) in Europe for patients with SCPCD. Data on the real-world use of protein C concentrate for LTP are limited.

Aims: To describe the real-world use of protein C concentrate for LTP and STP administered either intravenously or subcutaneously in patients with SCPCD in Europe and the US.

Methods: Physicians with experience using protein C concentrate to treat patients with SCPCD completed an online survey. Information collected included physicians’ clinical practice, experience treating SCPCD (based on their best recollection), treatment strategies, and opinions on the subcutaneous use of protein C concentrate.

Results: This analysis included responses from 19 physicians (US, n=7; Europe, n=12) who reported using protein C concentrate to treat 32 patients (US, n=8; Europe, n=24), of whom 23 (71.9%) received LTP (Table 1). Among those receiving LTP, 16 patients (69.6%) received intravenous LTP and 12 (52.2%) received subcutaneous LTP. Five patients (21.7%) received both intravenous and subcutaneous LTP. The administration of subcutaneous LTP was reported only by physicians in Europe. Overall, 18 physicians indicated that they would like subcutaneous administration to be approved for protein C concentrate. Reasons for this included its convenience relative to intravenous administration, and the difficulty associated with intravenous administration when venous access is limited.

Conclusion(s): LTP with intravenous protein C concentrate is used in clinical practice in both Europe and the US for the treatment of patients with SCPCD. Subcutaneous administration of LTP is also practiced by some physicians in Europe. Physicians in both Europe and the US indicated their interest in having subcutaneous administration as an approved route of administration for protein C concentrate.