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

Authors Di Minno G, Santagostino E, Morfini M, Ettorre C, Cultrera D, Baldacci E, Russo E, Gallucci C

Received 24 May 2018

Accepted for publication 9 October 2018

Published 31 January 2019 Volume 2019:13 Pages 233—240


Checked for plagiarism Yes

Review by Single anonymous peer review

Peer reviewer comments 4

Editor who approved publication: Dr Johnny Chen

Giovanni Di Minno,1 Elena Santagostino,2 Massimo Morfini,3 Cosimo Ettorre,4 Dorina Cultrera,5 Erminia Baldacci,6 Eleonora Russo,7 Cristiano Gallucci7

On behalf of the Italian FusENGO B1831081 Study Group

1Department of Clinical Medicine and Surgery, Azienda Universitaria Policlinico Federico II, Naples, Italy; 2Hemophilia and Thrombosis Center, IRCCS Fondazione Ca’ Granda, Ospedale Maggiore Policlinico, Milan, Italy; 3Scientific Committee, Italian Association of Haemophilia Centres (AICE), Florence, Italy; 4Haemophilia and Thrombosis Center, Policlinico Giovanni XXIII, Bari, Italy; 5Hematology Unit, Regional Center for Hemophilia, Italy Ospedaliera-Universitaria “Policlinico – Vittorio Emanuele”, Catania, Italy; 6Hemophilia, Thrombosis, and Hematology Center, Dipartimento Biotecnologie cellulari ed Ematologia, Università la Sapienza, Rome, Italy; 7Medical Department, Pfizer srl Rome, Italy

Background: FuseNGO 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.
Methods: An 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).
Results: Compared 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.
Conclusion: Considering 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.

Keywords: factor VIII delivery, device, hemophilia A, patient satisfaction, adherence

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