Treatment Adherence, Satisfaction and Quality of Life of Adult Patients with Severe Haemophilia a: Results from a Real-World Survey of Physicians and Patients

Introduction:

Hemophilia A (HA) is a rare, inherited disease affecting clotting factor; severity is determined by baseline clotting factor activity. Hemophilia often impacts daily living, and this worsens with increased severity. Current standard of care includes extended half-life (EHL), standard half-life (SHL), and non-factor therapies (NFT). These treatments require frequent infusions which may impact overall adherence and patient quality of life. There is limited literature available on how treatment choice affects patient-reported outcomes; this study aimed to assess patient treatment adherence and self-reported quality of life by treatment type.

Methods:

Data were drawn from the Adelphi Real World Disease Specific Programme™, a cross-sectional survey, with retrospective data collection of physicians and their male patients with Hemophilia A (PwHA), in France, Germany, Italy, Spain, the United Kingdom, and the United States of America. Data were collected between September 2023 and April 2024. Hematologists, hematologist-oncologists, and the pediatric equivalents were eligible for inclusion if they were actively involved in the management of at least one PwHA. These patients were invited to complete a voluntary patient self-completion form. Patients included in this analysis were adults with severe HA (baseline clotting factor <1%) who received prophylactic treatment at the time of data collection. Physicians reported data on demographics, treatment history/ adherence and quality of life. Patients voluntarily reported impairment to their health-related quality of life via the Haem-A-Qol, highlighting three domains: sports and leisure (S&L), Treatment (Tx), Future (Ft) and the total score (TS). The three domains and TS are transformed to a scale of 0 to 100; higher scores indicating higher impairment. Analyses were descriptive.

Results:

Overall, 94 physicians reported data on 374 PwHA that met the analysis criteria. Of these patients, 37% were on SHL, 22% EHL and 42% NFT. Mean (standard deviation [SD]) age was 32.5 (13.3) years (SHL: 31.3 [13.4], EHL: 32.5 [13.2], NFT: 33.4 [13.2]).

Physicians reported 45% of patients do not always take their treatment on time (SHL: 52%, EHL: 63%, NFT: 31%) and 54% were not completely satisfied with their patients' treatment (SHL: 73%, EHL: 72%, NFT: 28%). Of the physicians who were not completely satisfied, 53% reported their patient's treatment required too many infusions/injections (SHL: 59%, EHL: 57%, NFT: 39%). Of all PwHA, 75% had switched treatments from a previous prophylactic treatment, with the most recent regimen being discontinued due to “too many infusions/injections”. Physicians reported 33% of patients had difficulty with activities due to their hemophilia, the most reported impaired activity was the patient's ability to take part in sport or hobbies (SHL: 15%, EHL: 25%, NFT: 28%).

Patient self-reported data was provided by 54 patients (SHL: 9%, EHL: 31%, NFT: 59%). Overall, 82% of patients reported they avoided physical activity due to their hemophilia (SHL: 100%, EHL: 88%, NFT: 76%). Patients mean [SD] score for the Haem-A-QoL TS was 38.8 [17.5] points (SHL: 42.4 [23.6], EHL: 35.5 [18.2], NFT: 40.1 [16.5]). Within the domains, patients scored 55.0 [21.4] points for S&L (SHL: 55.9 [24.1], EHL: 51.5 [23.0], NFT: 56.8 [20.7]), 41.3 [22.6] points for Tx (SHL: 36.2 [28.4], EHL: 39.5 [28.0], NFT: 43.1 [18.9]) and 45.9 [22.7] points for Ft (SHL: 49.0 [40.4], EHL: 40.6 [20.5], NFT: 48.3 [20.8]). For the domain S&L, 54% avoided sports either often or all the time (SHL: 33%, EHL: 60%, NFT: 54%). For Tx, 33% were often afraid of complications (SHL: 20%, EHL: 29%, NFT: 38%) and for Ft, 24% reported they often thought it would be difficult to lead a normal life (SHL: 20%, EHL: 29%, NFT: 22%).

Conclusion:

Physicians reported that nearly half of their patients (45%) were not always adherent to their prophylaxis treatment; reporting dissatisfaction with the high frequency of infusions/injections, in their current regimen. A large proportion (82%) of patients reported they were avoiding physical activity, indicating a burden on patients' quality of life. Similarly, this study found poor overall scores on different aspects of the Haem-A-Qol, i.e. sports and leisure, treatment, and future. This highlights the need for better treatment options to address patients' overall impairment due to hemophilia, in turn improving patient standard of quality of life.

Merla:Pfizer: Current Employment, Current equity holder in publicly-traded company. Thakkar:Pfizer: Current Employment, Current equity holder in publicly-traded company. Healy:Pfizer: Current Employment, Current equity holder in publicly-traded company. Morton:Adelphi Real World: Current Employment. Wynne-Cattanach:Adelphi Real World: Current Employment. Ball:Adelphi Real World: Current Employment. Lai:Adelphi Real World: Current Employment. Blazos:Adelphi Real World: Current Employment. Salehi:Adelphi Real World: Current Employment.