Observational, PRO Study to Evaluate Quality of Life for Hemophilia Patients on Emicizumab, KSA Single Center Study

Background:

Emicizumab (Hemlibra) is a novel molecule, and its widespread prophylactic use is expected to dramatically reduce the bleeding frequency in the community of hemophilia A (HA) patients, significantly improving their clinical condition and quality of life.

Patient-reported outcome (PRO) measures are validated questionnaires self-completed by patients. These measures provide the patient's perspective on the physical, functional, and psychological consequences of treatment, as well as the degree and impact of disease symptoms.

HRQoL (Health-Related Quality of Life) is an important outcome in treating patients with hemophilia.

Objectives:

To evaluate how emicizumab affects the quality of life of pediatric patients with HA in real-world scenarios by examining changes in various areas such as mobility, self-care, leisure activities, pain and discomfort, emotional well-being, self-perception, school/work activities, and future outlook.

Methods:

The PRO study was designed for pediatric patients over the age of four with HA, with or without inhibitors, who have been using emicizumab for at least six months at Al Aziziyah Maternity and Children's Hospital. Validated Arabic versions of the HAL, Heam-A-Qol, and EQ-5D-5L questionnaires were prepared. All patients were asked to provide their responses before and after using Hemlibra regarding mobility, self-care, usual leisure and activities, pain and discomfort, bleeding, emotional impact, and their perceived health assessment.

Results:

A survey was conducted on ten male hemophilia patients who met the eligibility criteria out of 23 children with Hemophilia A who started using Emicizumab.

Before using Hemlibra, 10% of the patients had no mobility issues, and this increased to 80% after use, with significant improvements in walking, sitting, running, and fine hand movement. Additionally, 70% of the patients had no self-care problems before use, and this improved to 100% after use.

Before using Hemlibra, 30% of the patients participated in leisure activities such as cycling and contact sports like football. That percentage increased to 70% post-Hemlibra use.

Only 20% of the patients experienced no pain or discomfort before use; that percentage increased to 90% after use.

On an emotional level, positive feelings increased from 29% before use to 78% after use. However, negative emotions (feeling burdened, angry, worried, and/or excluded) increased from 38% before use to 80% after use.

Before Hemlibra use, only 38% of the patients had positive feelings about hemophilia, but this percentage improved to 78% after use. Positive feelings about self-perception increased from 22% before use to 66% after use.

Feelings about school/work improved from 13% before use to 83% after use, mainly due to the reduction in difficulty paying attention at work and school because of pain (a 70% decrease). Patients' positive feelings about prospects also increased from 45% before use to 92% after use.

Conclusion:

After six months of using Hemlibra, patients experienced improvements in their feelings, overall self-perception, ability to engage in daily activities, and feelings about hemophilia treatment. They reported being less worried that their condition would worsen and being better able to control their bleeding. However, there is still room for improvement in their ability to participate in work and school activities.

No relevant conflicts of interest to declare.