OC 59.4 - Health-related quality of life, treatment burden and patient preference in patients with haemophilia A/B without inhibitors on concizumab prophylaxis: results from the phase 3 explorer8 study

Tuesday, June 27, 2023

15:30 – 15:45 ET

Room: 517

Session Available As: Live-Stream and On-Demand

Presenting Author(s)

Guy Young, MD

Director, Hemostasis and Thrombosis Center
Children's Hospital Los Angeles
Los Angeles, California, United States

Co-Author(s)

Gary M. Benson, BCh BAO FRCP FRCPath

Consultant Haematologist
Belfast Health and Social Care Trust
Bangor, Northern Ireland, United Kingdom
Hermann Eichler, MD (he/him/his)

Professor of Transfusion Medicine and Haemostaseology
Institute of Clinical Haemostaseology and Transfusion Medicine, University and University Hospital of the Saarland, Homburg, Germany
Homburg, Saarland, Germany
Johnny Mahlangu, MD (he/him/his)

Professor and Director
Haemophilia Comprehensive Care Centre, Charlotte Maxeke Johannesburg Academic Hospital, University of the Witwatersrand and National Health Laboratory Service, Johannesburg, South Africa
Johannesburg, Gauteng, South Africa
JO

Jan Odgaard-Jensen

Statistical Specialist
Novo Nordisk A/S, Søborg, Denmark
Søborg, Hovedstaden, Denmark
JN

Jesper S. Neergaard

COA Principal Scientist
Novo Nordisk A/S, Søborg, Denmark
Søborg, Hovedstaden, Denmark
JT

Jay Jay Thaung Zaw

International Medical Manager
Novo Nordisk A/S, Søborg, Denmark
Søborg, Hovedstaden, Denmark
JS

Jameela Sathar

Consultant Haematologist
Department of Hematology, Hospital Ampang, Ampang, Selangor, Malaysia
Ampang, Selangor, Malaysia
HT

Huyen Tran

Professor
Australian Centre for Blood Diseases, Monash University
Melbourne, Victoria, Australia
TM

Tadashi Matsushita

Professor
Department of Transfusion Medicine, Nagoya University Hospital, Nagoya, Japan.
Nagoya, Aichi, Japan

Background: Patients with haemophilia generally report impaired health-related quality-of-life (HRQoL) and a high treatment burden. Concizumab is an anti-tissue factor pathway inhibitor (TFPI) monoclonal antibody in development as a once-daily subcutaneous prophylaxis for bleeding episodes in haemophilia. The phase 3 explorer8 study (NCT04082429) evaluated efficacy, safety and patient-reported outcomes of concizumab prophylaxis in patients with haemophilia A/B without inhibitors (HA/HB).

Aims: To present data from explorer8 on HRQoL, treatment burden and treatment preference in patients with HA/HB.

Methods: Male patients aged ≥12 years were randomised 1:2 to no prophylaxis (arm 1) or concizumab prophylaxis (arm 2). Additional patients were directly allocated to concizumab prophylaxis (arms 3&4). HA and HB subgroups were analysed separately. Exploratory patient-reported outcome endpoints were evaluated for arm 1 vs arm 2 at baseline and Week 24 for HRQoL (36-item Short-Form Health Survey version 2 [SF-36v2] and Haemophilia Quality-of-Life Questionnaire for Adults [Haem-A-QoL]) and treatment burden (Haemophilia Treatment Experience Measure [Hemo-TEM]). Treatment preference was evaluated at Week 24 after baseline in patients who received concizumab (Haemophilia Patient Preference Questionnaire; arms 2–4). Relevant ethical approvals and informed consent were obtained.

Results: This study included 148 patients (arms 1–4). At Week 24, HRQoL, as measured by SF-36v2 and Haem-A-QoL, was significantly improved in patients treated with concizumab prophylaxis (arm 2) vs no prophylaxis (arm 1) in some, but not all, domains (Figure 1). Overall treatment burden was significantly reduced in arm 2 vs arm 1 (Figure 2). Among those who completed the patient preference questionnaire in arms 2–4, 53/62 (85.4%) and 37/40 (92.5%) patients with HA and HB, respectively, preferred concizumab to their previous treatment.

Conclusion(s): Overall, patient preference for concizumab over previous treatment, and improvements in aspects of HRQoL and treatment burden, indicate the potential value of concizumab for patients with haemophilia.

Learning Objectives:

Upon completion, participant will be able to review the patient-reported outcome data collected in the phase 3 explorer8 study in patients with haemophilia A or B without inhibitors.
Upon completion, participant will be able to understand the supporting evidence for improved health-related quality of life, reduction in treatment burden and patient preference in patients receiving once-daily, subcutaneous concizumab prophylaxis.
Upon completion, participant will be able to examine the limitations associated with the patient-reported outcome data.