OBJECTIVES: Growth hormone deficiency (GHD) in children causes decreased growth and markedly reduced adult height. The treatment for GHD has been given as daily subcutaneous injections. However, new GH formulations can be given weekly. Injections are a burden for children and caregivers, but to our knowledge, the impact on quality of life (QoL) has not been investigated using time trade-off (TTO) methodology. This study evaluates the impact of type of injections on QoL using a TTO survey.

METHODS: TTO methodology was used to estimate utilities through two online surveys (S1 and S2) completed by the general population, as required by NICE and CADTH, in the UK and Canada. In S1, adults (18+ years) evaluated health states as if they took injections themselves, and in S2, adults evaluated health states as if they gave injections to a child with GHD. To increase relatability, only adults with a child under the age of 15 years were included in S2. The following treatment aspects were evaluated: device complexity, injection frequency, injection pain, needle visibility and storage possibilities.

RESULTS: 2,029 and 2,028 respondents completed S1 and S2, respectively. The results suggest that injection pain is important. Avoiding injection pain at weekly injections was associated with a significant utility gain of 0.030 (CI 95% 0.026–0.035, p<0.001) in S1 and 0.044 (CI 95% 0.038–0.051, p<0.001) in S2. Furthermore, less complex injection devices and lower injection frequency also had a significant impact on QoL in both S1 (0.020, CI 95% 0.016-0.025, p<0.001; 0.009, CI 95% 0.005-0.014, p<0.001) and S2 (0.008, CI 95% 0.002-0.014, p=0.006; 0.009, CI 95% 0.003-0.014, p=0.003).

CONCLUSIONS: Several aspects of GHD treatment have a significant impact on QoL. Less time-consuming and complex treatment options with reduced injection pain and frequency are expected to result in higher QoL which could lead to higher treatment compliance.