PB0203 - NON-INTERVENTIONAL study on health-related quality of life, physical activity and bleeds in severe non-inhibitor hemophilia A patients with a bleeding phenotype on FVIII prophylaxis in the Nordics

Sunday, June 23, 2024

13:45 – 14:45 ICT

Room: Exhibition Hall

Co-Author(s)

JB

Jenny Björkqvist

Scientific Responsible
F. Hoffmann-La Roche Ltd, Basel, Switzerland
Huddinge, Stockholms Lan, Sweden
JA

Jan Astermark

Professor, Senior Consultant
Department of Translational Medicine, Lund University, Malmö, Sweden
Malmo, Skane Lan, Sweden
PH

Pal André Holme

Professor
Department of Hematology, Oslo University Hospital, Oslo, Norway and Institute of Clinical Medicine, University of Oslo, Oslo, Norway
Oslo, Oslo, Norway
Riitta Lassila, MD, PhD

Professor of Coagulation Medicine
Department of Hematology, Coagulation Disorders Unit, and Research Program Unit in Systems Oncology Oncosys, Medical Faculty, University of Helsinki, Finland
Helsinki, Uusimaa, Finland
SR

Susanna Ranta

Dr.
Astrid Lindgren's Children's Hospital, Karolinska University Hospital, and Department of Women's and Children's Health, Karolinska Institutet, both in Stockholm, Sweden
Stockholm, Stockholms Lan, Sweden
LM

Linda Myrin-Westesson

Nurse, PhD
Region Västra Götaland, Sahlgrenska University Hospital, Department of Medicine and Sahlgrenska Academy, Institution for Nursing Science and Health at Gothenburg University, both in Gothenburg, Sweden
Gothenburg, Vastra Gotaland, Sweden
MH

Marianne Hoffmann

Dr.
Department of Pediatrics and Adolescent Medicine, University Hospital Rigshospitalet, Copenhagen, Denmark.
Copenhagen, Hovedstaden, Denmark
SQ

Sandrine Quere

Study Lead
F. Hoffmann-La Roche, Ltd., Basel, Switzerland
Oslo, Oslo, Norway
AC

Amparo Yovanna Castro Sanchez

Data Sciences
F. Hoffmann-La Roche Ltd, Basel, Switzerland
Basel, Basel-Stadt, Switzerland

Presenting Author(s)

Tünde Czirok

Global Medical Director
F. Hoffmann-La Roche, Ltd., Basel, Switzerland
Basel, Basel-Stadt, Switzerland

Background: Physical activity (PA) has positive effects on health-related quality of life (HRQoL) in severe hemophilia A (sHA) patients but requires adequate prophylaxis. Some patients still experience bleeds which indicate unmet medical need.

Aims: To observe sHA patients from the Nordics in a real-world setting and describe HRQoL, PA, bleeds and safety. This cohort included patients with at least 1 annual bleed (ABR) in joints/muscles and considered for an alternative regime.

Methods: The primary objective was to describe HRQoL by CATCH questionnaire at week 24 while PA was monitored using Fitbit in two 8-week periods along with the international PA questionnaire IPAQ-SF. Bleeds and safety were self-reported. Patients were observed for at least 24 weeks (maximum 32 weeks) between Aug 2021 and Jan 2023 (Note: this study precedes 2020-003256-32 interventional study).

Results: Thirty patients were recruited, and 29 patients aged between 12 and 60 years were included in the analysis. Almost all reported high HRQoL based on low risk-perception and impact.
The Fitbit data showed a mean average daily step count of 8731 and 8596, and daily moderate to vigorous PA of 66 and 71 min (irrespective of wearing time). Of the 22 evaluable patients, 16 (73%) were classified in the highest or moderate level of PA by IPAQ-SF, Figure 1 present MET-minutes/week by activity-type.
The model-based ABR was 8.3 (median=6.6) for all bleeds and 5.1 (median=2.2) for treated bleeds (Table 1).
No AEs/SAEs were considered treatment-related.

Conclusion(s): The patients in this cohort with a bleeding phenotype report relatively good HRQoL and PA levels despite a high self-reported bleed rate, suggesting lack of bleed recognition and various levels of sub-optimal bleed control under different FVIII replacement regimens. The study findings indicate the need to evaluate alternative prophylactic options to improve bleed control in sHA patients with similar characteristics.

The study sponsor was F.Hoffmann-La Roche Ltd