OC 12.2 - Decreased quality of life in non-severe hemophilia patients with an increased bleeding phenotype

Sunday, June 25, 2023

11:00 – 11:15 ET

Room: 710A

Session Available As: On-Demand

Presenting Author(s)

Marieke Verhagen, MD

PhD student, MD
Department of Hematology, Radboud University Medical Center, Nijmegen, the Netherlands. Hemophilia Treatment Center Nijmegen-Eindhoven-Maastricht, Nijmegen, the Netherlands. Laboratory of hematology, Radboud university medical center, Nijmegen, the Netherlands
Nijmegen, Gelderland, Netherlands

Co-Author(s)

Ev

Erna van Balen

Epidemiologist
Department of Clinical Epidemiology, Leiden University Medical Center, Leiden, the Netherlands
Leiden, Zuid-Holland, Netherlands
NB

Nicole M.A. Blijlevens

Internist-hematologist, Professor
Department of Hematology, Radboud university medical center, Nijmegen, the Netherlands
Nijmegen, Gelderland, Netherlands
Wv

Waander L. van Heerde

Biochemist, Coagulation Physiologist, Chief Scientific Officer Enzyre BV
Department of Hematology, Radboud university medical center, Nijmegen, The Netherlands. Hemophilia Treatment Center Nijmegen – Eindhoven – Maastricht, The Netherlands. Enzyre BV, Novio Tech Campus, Nijmegen, The Netherlands.
Nijmegen, Gelderland, Netherlands
Samantha Gouw, MD, PhD (she/her/hers)

Pediatric Hematologist
The University of Amsterdam
Amsterdam, Noord-Holland, Netherlands
SS

Saskia E.M. Schols

Internist-hematologist
Department of Hematology, Radboud university medical center, Nijmegen, The Netherlands. Hemophilia Treatment Center Nijmegen – Eindhoven – Maastricht, The Netherlands
Nijmegen, Gelderland, Netherlands

Background: Patients with moderate hemophilia form a heterogeneous group with varying bleeding phenotypes. A substantial number of patients are prone to severe bleeding complications, joint damage, and a decreased quality of life.

Aims: To analyse the difference in joint status and health related quality of life (HrQoL) between moderate hemophilia patients with a severe bleeding phenotype and those with a mild bleeding phenotype.

Methods: For this sub-study of the 6th Hemophilia in the Netherlands study, data of adults with moderate hemophilia A or B were used (FVIII 1-5%). Data on bleeding tendency, joint status, and HrQoL were obtained from electronic patient files and self-reported questionnaires (PROMIS-29). A severe phenotype was defined as an annual bleeding rate (ABR) ≥5, an annual joint bleeding rate (AJBR) ≥3, or the use of prophylaxis, and a mild phenotype vice versa. Outcomes on QoL were adjusted for age, BMI, and the presence of a comorbidity.

Results: In total, 145 moderate hemophilia patients were included (122 hemophilia A, 23 hemophilia B). Of these patients, 22% had a severe phenotype of whom 60% used prophylaxis. Patients with a severe phenotype treated on demand reported a higher ABR (7 vs 2 vs 0), AJBR (2 vs 0 vs 0), and a higher percentage of joint impairment (82% vs 57% vs 52%) compared to patients with a severe phenotype using prophylaxis or patients with a mild phenotype (figure 1).
Patients with a severe phenotype, treated on demand, scored worse on all HrQoL domains, except for sleep disturbance. Patients with a severe phenotype, who used prophylaxis, had similar scores as patients with a mild phenotype (except for physical function and fatigue) (table 1).

Conclusion(s): A quarter of moderate hemophilia patients experience a severe bleeding tendency, with related joint impairment and reduced quality of life, which advocates towards a more intensive treatment strategy with prophylaxis.

Learning Objectives:

create awareness that moderate hemophilia patients can suffer different bleeding phenotypes
understand that moderate hemophilia patients with a severe bleeding phenotype who are treated on demand have worse bleeding outcomes and a reduced quality of life
emphasize that a severe bleeding phenotype advocates towards a more intensive treatment strategy with prophylaxis