According to hemophilia A patients, FuseNGo, Pfizer‘s pre-filled syringe device used for both the treatment and prevention of bleeding, is easy to use and has a wide range of benefits, a study reports.
These results support the potential for FuseNGO to increase patient adherence to therapy.
The study, “Patient satisfaction and acceptability of an on-demand and on-prophylaxis device for factor VIII delivery in patients with hemophilia A,” was published in the journal Patient Preference and Adherence.
Preventive, or prophylactic, treatment with therapies such as coagulation factor VIII (FVIII) is linked to a significant decrease in bleeding episodes. However, despite its benefits, adherence to prophylactic treatment is highly variable, ranging between 44% and 87% among hemophilia A patients worldwide.
Strategies to improve adherence include increasing the half-life of FVIII, reducing the need for more frequent dosing, and the development of devices to ease administration and reconstitution, which involves mixing with a liquid called a diluent.
Among these devices, those that simplify the reconstitution process of recombinant FVIII and are quick and easy to use are particularly attractive.
FuseNGo, developed by Pfizer, is a relatively new device and consists of a pre-filled dual-chamber syringe that includes both the recombinant factor VIII and diluent all in a single syringe. Specifically, it delivers the compound moroctocog alfa, which is marketed as Xyntha in the U.S. and ReFacto aF in Europe.
“The [dual-chamber syringe] was developed with the specific aim of rendering the treatment less burdensome, and therefore, increase adherence to the prescribed treatment regimen,” the researchers wrote.
In this observational study (NCT01959919) conducted in Italy, researchers asked patients with a confirmed diagnosis of hemophilia A to answer five questionnaires — HemoPREF, which assesses their experiences with the clotting-factor treatment; Treatment Satisfaction Questionnaire for Medication (TSQM); VeritasPRO, a measure of treatment adherence; Hemophilia Well-being Index (HWBI); and the Work Productivity and Activity Impairment Questionnaire (WPAI) + Classroom Impairment Questions (CIQ): Hemophilia Specific (HS).
The goal was to evaluate patient perceptions and preferences of the FuseNGo device after a trial period that included at least 10 infusions.
The study enrolled 86 male patients, ages 18–65 years, who were on treatment with FVIII, either prophylactically or on-demand. Of these patients, 84 completed the study and were included in the analysis. Patients’ responses were evaluated at the beginning of the study before they began using the device and after three to six months of use.
Results of the HemoPREF questionnaire, which evaluated ease of preparation, storage, disposal of needles and syringes, and use of treatment, showed that patients reported a significant improvement with the new device over what they were previously using. Additionally, the mean time spent reconstituting recombinant FVIII was, on average, cut almost in half — from 11 minutes to six minutes.
More than 95% of the participants said they were satisfied with the device, according to the results of the TSQM. In addition, more than 70% of them had positive responses in the VeritasPRO questionnaire, meaning that adherence to the prophylactic therapy was high.
More than 65% of the participants reported in the HWBI that hemophilia A had not affected their lives in a significant manner, also confirmed by an overall impairment of 30% in patients’ ability to work, according to the responses in the WPAI + CIQ: HS.
There were no severe adverse events, and the minor adverse events were not linked with the device.
“The vast majority of patients were satisfied with the device, and overall adherence to it was high,” the researchers wrote.
Moreover, use of the device was linked with a decrease in the overall burden of hemophilia on work and daily activities without raising safety concerns.
“Given its positive patient-rated evaluation, the device has the potential to increase adherence to therapy and reduce healthcare costs and, thus, warrants further investigation in a broad patient population to assess its benefits in routine settings,” they concluded.