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Poster Networking

Inherited Bleeding Disorders and Inhibitors

PB0237 - Prospective observational study to evaluate the quality of life in patients with Hemophilia A without inhibitors: HEMOLIFE study

Sunday, June 23, 2024
13:45 – 14:45 ICT
Room: Exhibition Hall
Background: Haemophilia A (HA) is a disease with an impact on the sufferer and their environment interfering with their daily lives.

Aims: To evaluate the impact of HA in quality of life in patients without inhibitors and in their caregivers’ burden. Herein, we report final results of HEMOLIFE prospective study.

Methods: Multicenter, prospective, non-interventonal study to describe changes in socio-occupational impact over a 12-month period in patients ≥ 12 years with moderate or severe HA without inhibitors and their caregivers. Informed consent and approval by ethics committee obtained. Maladjustment scale, Haemo-QoL, Haemo-QoL-SF for adolescents, functional assessment (HAL) and WPAI+CIQ were used beyond others. Patient complete self-questionnaires and outcomes in the study app.

Results: The study included 85 patients with a mean age of 33 years, 71(83,5%) had severe and 14(16,5%) moderate haemophilia. 77(90,6%) patients were receiving prophylactic treatment at study entry: 39(51%) standard half-life, 23(30%) extended half-life and 17(22%) emicizumab. Participant characteristics are shown in table 1. 12 caregivers were included in the study, mainly women 10(83.3%). A score greater than 12 points in maladjustment scale in 17(20.5%) patients at baseline and 13(25.5%) at the end of the study. Results of different questionnaires are shown in table 2.

Conclusion(s): Hemophilia affects many aspects of a patient's life. We analyzed the disease holistically to identify appropriate therapeutic approach and to achieve an active life, as recommended by the guidelines(1).
We highlight that 76% patients were given pain meds, and 38% used rescue medication. Anxiety and depression's impact on patients and caregivers, emphasizing the need for a multidisciplinary approach.
The sample is representative from our environment, with a mean treated ABR of 0,6.
The maladjustment scale found that 1 of each 4 scored above the cut-off(2) and almost half had work/study-related or overall maladjustment meaning a degree of adjustment needed to achieve an active life.