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Little is known about the treatment experience of patients with pediatric growth hormone deficiency (pGHD) and their caregivers in Japan after the approval of once-weekly growth hormone (GH) injections. This study aimed to assess the treatment experience, unmet needs, and treatment satisfaction of patients with pGHD and their caregivers who were administering once-daily or once-weekly GH injections. A cross-sectional online survey using Japanese consumer panels was conducted from 14th June 2024 to 24th June 2024. Caregivers who had administered GH injections for ≥ 3 months to patients with pGHD aged 3–14 years were surveyed. In addition to between-group comparisons by injection frequency, a multivariable logistic regression analysis adjusted for confounding factors was conducted to identify factors associated with the desire to change injection frequency. A total of 187 eligible caregivers (once-daily cohort, n = 137; once-weekly cohort, n = 50) participated in the study. The top three unmet needs of caregivers were injection pain (35.8%), effectiveness (29.4%), and injection frequency (26.2%). The once-daily cohort had higher proportions of caregivers who desired to change the injection frequency (47.4% versus 22.0%, p = 0.002) and who cited having no chance to switch as a reason for the current injection frequency (27.7% versus 10.0%, p = 0.011) compared with the once-weekly cohort. There were no significant differences between the once-daily cohort and the once-weekly cohort in terms of perceived injection pain or treatment satisfaction assessed by Treatment Satisfaction Questionnaire for Medication version II (TSQM v. II) from the caregiver’s perspective. Multivariable logistic regression analysis showed that current once-daily administration was an independent factor associated with the desire to change (adjusted odds ratio [aOR] 5.30, 95% confidence interval [CI] 2.22–13.7, p < 0.001). These findings suggest that the once-daily cohort may be missing opportunities to re-evaluate their current treatment regimen. To optimize the treatment experiences and quality of life for patients and caregivers, it is necessary to promote shared decision-making (SDM) for regular treatment reviews. However, given the cross-sectional design of this study, these findings represent associations rather than causal relationships. Children with growth hormone (GH) deficiency need regular GH injections. We wanted to understand how caregivers feel about their daily or weekly treatments after once-weekly GH injections became available in Japan. We surveyed 187 caregivers about their treatment experiences. Among caregivers using daily injections, nearly half (47.4%) expressed a desire to reduce the frequency of injections. However, some (27.7%) reported having no opportunity to switch their injection frequency. Meanwhile, from the caregivers’ perspective, there was no clear difference between the two groups in overall treatment satisfaction and perceived injection pain. Even after accounting for background factors such as the child’s school level, the analysis confirmed that receiving daily injections itself was a factor associated with the desire to reduce the injection frequency (adjusted odds ratio: 5.30). Promoting communication between doctors and families about available treatment options may improve the treatment experience and quality of life.
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Takasawa et al. (2026) studied this question.
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