Rationale and Design of PathfinderReal: A Prospective, Non-Interventional Study Assessing Joint Health in Adults With Haemophilia A after Switching to Prophylaxis with Turoctocog Alpha Pegol (N8-GP).

Ay, C.; Nagao A.; Liles D.; Matino D.; Gidley G.; Waller J.; Nemes L.; Mancuso M.; Benitez O.; Oldenburg J.

Rationale and Design of PathfinderReal: A Prospective, Non-Interventional Study Assessing Joint Health in Adults With Haemophilia A after Switching to Prophylaxis with Turoctocog Alpha Pegol (N8-GP).

All Authors

Ay, C.

Nagao A.

Liles D.

Matino D.

Gidley G.

Waller J.

Nemes L.

Mancuso M.

Benitez O.

Oldenburg J.

LTHT Author

Gidley, Gillian

LTHT Department

Haematology

Publication Date

2023

Item Type

Conference Abstract

Abstract

Background: Joint damage affects quality of life in people with haemophilia. The long-term safety and efficacy of turoctocog alfa pegol (N8-GP) prophylaxis in patients with haemophilia A (HA) has been well investigated in pivotal phase 3 trials in children, adolescents and adults (pathfinder programme). However, there is a paucity of long-term real-world data regarding joint health of people treated with N8-GP. Aim(s): To describe the rationale and design of the ongoing pathfinder-Real study investigating the joint health status in patients with HA after switching to N8-GP prophylaxis. Method(s): pathfinderReal is a multi-country, non-interventional, single-arm study (NCT05621746) of joint health in adult male patients (>=18 years) with HA who have switched to N8-GP prophylaxis. Patients enrolled in other interventional studies and those who have previously terminated N8-GP treatment will be excluded. Approximately 124 adults with HA will be enrolled and followed for a maximum of 24 months (Figure 1). Result(s): Data from routine clinical assessments of patients' joint health will be collected. The primary end point is maintenance in Hemophilia Joint Health Score (HJHS; defined as change in total score <=2 points) from initiation of N8-GP treatment until the end of the study. Secondary endpoints include patient-reported outcomes of problem joints, pain score, number of bleeding episodes, number and resolution of target joints, and change in physical activity levels. An exploratory end point is included to measure the number of patients achieving improved HJHS from initiation of N8-GP treatment until the end of the study. Conclusion(s): The pathfinderReal study will provide insights regarding longterm joint health in adult patients with HA treated with N8-GP in a real world setting. [Figure presented]