Inhibitors Significantly Impair Quality of Life for Hemophilia a Patients

Background: Hemophilia A is a rare, inherited bleeding disorder that can severely affect a patient's quality of life (QoL) across all age groups. The development of inhibitors, antibodies that neutralize clotting factor therapies, may further exacerbate the disease burden in this patient population. Aims: This study aims to assess the impact of inhibitors on key disease burden factors and their association with decreased QoL in hemophilia A patients. Methods: The Ipsos Hemophilia Therapy Monitor, a multi-country quantitative online survey, was conducted from November 2023 to February 2024.The study collected 805 hemophilia A patient records (US n=149; UK n=113; France n=110; Germany n=87; Italy n=144; Spain n=154; Canada n= 48) from participating physicians, including 113 patients with inhibitors and 692 patients without inhibitors. Physicians were screened for duration of practice in their specialty and caseload, and provided data on the presence of target joints (defined as joints in which spontaneous bleeding has occurred on 3 or more occasions within the past 12 months), chronic pain levels (0-10 scale), and treated bleeds (a bleeding event where coagulation factors are administered to treat signs or symptoms of bleeding (e.g., pain, swelling, etc.) in the past 12 months). Patients were considered to have decreased QoL if they reported the presence of target joints, chronic pain levels of 5 or above, and at least one treated bleed in the past 12 months. Results: In the study, reported patients with inhibitors were significantly more likely to have target joints (30% vs. 12%, p<0.01), chronic pain levels of 5 or above (45% vs. 21%, p<0.01), and had at least one treated bleed in the past 12 months (61% vs. 43%, p<0.01) compared to those without inhibitors. Inhibitor patients were also more likely to experience all 3 impairments simultaneously (19% vs. 6%, p<0.01), indicating a higher prevalence of decreased QoL. Overall, only 26% of the inhibitor patients reported none of the 3 disease burdens, compared to 48% of non-inhibitor patients (p<0.01). Interestingly, 40% of patients who had previously developed inhibitors but are now inhibitor-free reported an absence of the three impairments. This suggests that patients who have overcome inhibitors may experience an improvement in their QoL, potentially regaining functionality and reduced disease burden compared to those who continue to struggle with persistent inhibitors. Summary/Conclusion: In this study, hemophilia patients with inhibitors, across all age groups, experienced a severe, multifaceted disease burden, that significantly undermined their quality of life. However, eradicating inhibitors may lead to improved QoL outcomes. These findings highlight the critical need for enhanced treatment strategies and comprehensive care approaches to alleviate the unique challenges faced by inhibitor hemophilia A patients and optimize their overall well-being. Further research using comparator data is warranted.