<p>Patient satisfaction and acceptability of an on-demand and on-prophylaxis device for factor VIII delivery in patients with hemophilia A</p>

Abstract

BackgroundFuseNGO is a relatively new device consisting of a prefilled dual-chamber syringe (DCS) that was recently introduced for the reconstitution of recombinant factor VIII. Herein, the DCS device was assessed using five questionnaires with the primary aim of evaluating patient perceptions and preferences.MethodsAn observational, non-interventional, longitudinal study on 86 patients with a confirmed diagnosis of hemophilia A was carried out at 21 sites in Italy. Each patient underwent a baseline visit and final study visit within 3–6 months. Patients were administered five questionnaires: HemoPREF; Treatment Satisfaction Questionnaire for Medication (TSQM); VeritasPRO; Hemophilia Well-being Index (HWBI); Work Productivity and Activity Impairment Questionnaire (WPAI) + Classroom Impairment Questions (CIQ): Hemophilia Specific (HS).ResultsCompared to baseline, scores for HemoPREF were higher at follow-up; significant increases in the percentage of positive responses were seen for all questions regarding the ease of use (P<0.05). The mean time needed for the reconstruction of the device at baseline was 11 minutes (range 1–30 minutes), which decreased to 6 minutes (range 30 seconds to 25 minutes) at follow-up. All scores in the TSQM indicated good satisfaction with the device. Patients reported an adherence of >70% in the VeritasPRO questionnaire, and the majority of patients reported in the HWBI that hemophilia A did not affect their lives in a significant way. The perceived level of overall impairment was 30% as reported in the WPAI + CIQ: HS, indicating little impairment. There were no safety concerns.ConclusionConsidering patient-reported outcomes, the DCS device was associated with easier preparation, storage, disposal of equipment, and overall use. Of particular note, preparation times were reduced by around 50%. The majority of patients were satisfied with the device and overall adherence scores were high. Considering these results, the device has the potential to increase adherence to therapy and, possibly, reduce healthcare costs.

Patients’ and parents’ satisfaction with, and preference for, haemophilia A treatments: a cross‐sectional, multicentre, observational study

“…postmenopausal osteoarthritis (PMO) reported treatment satisfaction as 56 in effectiveness, 64 in side effects and 54 in the global domain, and patients with atrial fibrillation (AF) in this study which is similar to that reported by patients with AF treated with vitamin K (58), and slightly lower than the mean score (63) reported by patients with PMO 18,19. Surprisingly, satisfaction with treatment convenience in our study was remarkably lower than that reported in patients with haemophilia using PDC syringes in Italy 20. …”

“… 18 , 19 Surprisingly, satisfaction with treatment convenience in our study was remarkably lower than that reported in patients with haemophilia using PDC syringes in Italy. 20 …”

Clinical attributes and treatment characteristics are associated with work productivity and activity impairment in people with severe haemophilia A

“…[20][21][22][23] Few studies are published using the WPAI in people with haemophilia. 15,24,25 A recent study of young adult PWSHA across Europe found high WPL even with lifelong prophylaxis. 15 In a Dutch cohort of young adults with congenital coagulation disorders (CCDs), WPL was not statistically associated with clinical characteristics, physical functioning or illness cognitions (helplessness, acceptance); AI was not evaluated.…”

Validation of a new instrument to measure disease‐related distress among patients with haemophilia

“…WPAI asks participants to rate how much each of 9 items interfered with work or school during the past 7 days. 22,23 Higher scores indicate greater activity impairment.…”