Less-frequent FVIII Dosing May Improve Treatment Adherence

Less-frequent and easier administration of factor VIII (FVIII) — the clotting protein that is missing or defective in people with hemophilia A — is likely to boost treatment adherence among patients with severe disease, a South Korean study has found.

Current treatments for hemophilia A are based on providing the missing FVIII, either during bleeding episodes as an on-demand treatment, or on a regular basis as a preventive measure (prophylaxis).

Patients’ adherence to their treatment plan is essential to keep the disease under control, most importantly because treatments usually are self-administered and long-lasting.

However, previous studies have reported that adherence drops when patients perceive high treatment burden, duration, and lack awareness about side effects.

In order to achieve the best treatment outcomes and enhance adherence it is important to know patients’ preferences and satisfaction with their treatment.

Here, a group of researchers in South Korea reported the findings of an observational study that sought to assess treatment satisfaction in patients with hemophilia A. The study was conducted from April 2018 to September 2019, and involved six nationwide hospitals and three Korea Hemophilia Foundation clinics.

Treatment satisfaction was assessed using the Treatment Satisfaction Questionnaire for Medication (TSQM), a tool that measures patients’ views on side effects, treatment effectiveness, convenience, and global satisfaction. TSQM scores range from zero to 100, with higher scores indicating greater satisfaction. The questionnaire was directed at patients with hemophilia A (ages 16 and older) or to the legal caregivers of children with the disease.

In total, 505 hemophilia A patients (99.6% men, mean age 31.5) participated in the study. More than half (87.7%) had severe disease, but less than 1% had developed inhibitors (neutralizing antibodies) against FVIII.

About half of the patients (53.5%) received preventive FVIII treatment and 22.2% were treated on-demand. Treatment was maintained for an average of 102.9 months (8.6 years). Most patients (72.9%) administered treatment at least twice weekly, and nearly half (49.1%) used two vials of FVIII per injection.

Overall, 264 patients (52.3%) experienced bleeds during the last month of treatment, at an average of 3.2 bleeding episodes per patient. In most of cases bleeding was mild (190 patients, 37.6%) and was considered to be severe in only four (0.8%) patients.

The global satisfaction score, as measured by the TSQM questionnaire, was 66.8. The highest score — indicative of greater satisfaction — was in the side effects domain (score of 97.9), followed by the effectiveness domain (score of 64.6). The lowest score — signaling lower satisfaction — was seen in the convenience domain (score of 57.1).

After adjusting for potential interfering factors like age, disease severity, and others, researchers found the number of vials per injection and the frequency of treatment were both linked significantly to treatment satisfaction.

Specifically, more-frequent dosing was associated with lower effectiveness and global satisfaction scores. Patients using four or more vials per injection had lower global satisfaction scores compared with patients using only one vial.

Also, simpler treatments requiring fewer doses and easier manipulation — for example, those using pre-filled, dual-chamber syringes — were the preferred choices of patients and caregivers.

“Patients with haemophilia A, or caregivers, were somewhat satisfied with their current treatment using FVIII concentrate in Korea,” the researchers wrote. “However, the study showed that they are less satisfied in terms of the convenience of treatment and that the preference is for simpler and easier characteristics of treatment.”

The researchers concluded: “Practices which enhance the treatment satisfaction of patients, considering their preference, should be tried in an effort to increase treatment adherence and compliance to attain the ultimate treatment goals.”