PB1292 - 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)

Tuesday, June 27, 2023

18:30 – 19:30 ET

Room: Exhibition

Presenting Author(s)

Cihan Ay, MD (he/him/his)

Professor, Head of the Thrombosis and Haemostasis Research Group
Clinical Division of Haematology and Haemostaseology, Department of Medicine I, Medical University of Vienna, Vienna, Austria
Vienna, Wien, Austria

Co-Author(s)

Azusa Nagao, MD, MSc

MD
Department of Blood Coagulation, Ogikubo Hospital, Tokyo, Japan
Tokyo, Tokyo, Japan
DL

Darla Liles

Chief of the Division of Hematology and Oncology
East Carolina University
Greenville, South Carolina, United States
Davide Matino, MD (he/him/his)

Assistant Professor
Department of Medicine, Division of Hematology, McMaster University, Hamilton, ON
Hamilton, Ontario, Canada
GG

Gillian Gidley

Consultant Haematologist
Leeds Teaching Hospitals Trust
Leeds, England, United Kingdom
JW

John Waller

Senior director, Real World Evidence
Novo Nordisk
Zurich, Zurich, Switzerland
LN

Laszlo Nemes

Director of the National Haemophilia Center and Haemostasis Department
Medical Center of Hungarian Defence Forces
Budapest, Budapest, Hungary
Maria Elisa Mancuso, MD, PhD (she/her/hers)

Senior Consultant in Haematology
IRCCS Humanitas Research Hospital
Milan, Lombardia, Italy
OB

Olga Benítez, MD

Hematology Deparment
Vall d'Hebrón Barcelona Hospital Campus
Barcelona, Catalonia, Spain
Johannes Oldenburg, MD (he/him/his)

Professor, Chairman and Director
University Clinic Bonn AöR, Institute of Experimental Haematology and Transfusion Medicine, Bonn, Germany
Bonn, Nordrhein-Westfalen, Germany

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.

Aims: To describe the rationale and design of the ongoing pathfinderReal study investigating the joint health status in patients with HA after switching to N8-GP prophylaxis.

Methods: 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).

Results: 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 long-term joint health in adult patients with HA treated with N8-GP in a real-world setting.