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

• Published in: Haemophilia : the official journal of the World Federation of Hemophilia 2021-07, Vol.27 (4), p.563-573
• Main Authors: Park, Young Shil, Hwang, Tai Ju, Cho, Goon Jae, Kim, Soon Ki, Park, Sang Kyu, Kim, Ji Yoon, Baek, Hee Jo, Kim, Young‐Joo, Lee, Ho‐Jin, Shin, Ji‐Soo, Nam, Chung‐Mo, Sun, Jiyu, Yoo, Ki Young
• Format: Article
• Language: English
• Subjects: Caregivers; Child; Coagulation factors; Cross-Sectional Studies; factor VIII; haemophilia; Hemophilia; Hemophilia A - drug therapy; Humans; Infant, Newborn; Observational studies; Original; Parents; Patient Preference; Patient Satisfaction; Patients; Personal Satisfaction; preference; Preferences; Prophylaxis; satisfaction; treatment
• ISSN: 1351-8216; 1365-2516
• DOI: 10.1111/hae.14304

ABSTRACT Introduction Reports on patients’ satisfaction and preferred characteristics for treatments would be worthwhile when choosing an optimal treatment reflecting patients’ perspectives. Aim To identify the characteristics and treatment patterns of patients with haemophilia A, or their caregivers, in Korea and explore patient preferences and satisfaction with their treatment. Methods This cross‐sectional, multicentre, observational study was conducted from April 2018 to September 2019 at six nationwide hospitals and three Korea Hemophilia Foundation clinics. Patients aged ≥16 years, or legal caregivers of paediatric patients, who had used factor VIII (FVIII) concentrates for ≥1 month were enrolled. Satisfaction with treatment was measured using the Treatment Satisfaction Questionnaire for Medication (TSQM); preference was evaluated using discrete choice experiment (DCE), with 10 series of two hypothetical treatment options created from D‐efficient block design, which varied across five attributes. Results Overall, 505 patients (mean age 31 years) were enrolled in the study. Patients had received FVIII concentrate for an average of 102.9 months (prophylaxis: 53.5%; on‐demand: 22.2%). Mean TSQM scores were 64.6 (effectiveness domain), 97.9 (side effects), 57.1 (convenience) and 66.8 (global satisfaction). The number of vials per injection, and the frequency of drug administration, was significantly associated with treatment satisfaction. According to DCE, simpler treatment options were preferred by patients/caregivers. Conclusion The lowest satisfaction levels were shown in the treatment convenience domain. Patients/parents preferred simpler and easier treatment characteristics. In an attempt to enhance the overall satisfaction of patients and caregivers with treatment, consideration of more convenient characteristics is required in future decisions regarding treatment selection.