Patient preferences for growth hormone treatment in Japanese children

Background There are not clear evidence to date evaluating patients' and caregivers' preferences for the recombinant-human growth hormone (r-hGH) injection in children in Japan. This study aimed to quantitatively evaluated the factors driving preferences for daily r-hGH injections among Japanese children with growth hormone deficiency (GHD) or their caregivers and to determine the relative importance of treatment delivery factors. Methods This study was performed among Japanese children with GHD or their caregivers who visited a specialized clinic in Japan as part of their routine care. The participants were asked to complete a web-based discrete choice experiment (DCE) questionnaire. Results Choice-based conjoint analysis was used to evaluate the relative importance of the attributes of the choice predictors and determine utility scores for each attribute. Of the 47 respondents who participated in this study, 41 were caregivers who responded on behalf of the patients, the remaining six were patients who completed the DCE themselves. The injection schedule was found to be the most important factor for both patients and caregivers; a once-weekly injection schedule was preferred over a daily injection schedule. Storage and preparation was deemed more important to patients than it was to caregivers, with patients preferring storage at room temperature even if it required additional mixing (reconstitution). Both patients and caregivers showed a clear preference for devices that offered a dose-setting memory. Conclusions A less frequent injection schedule may enhance adherence to r-hGH treatment and expected improve quality of life for GHD patients over the long term.