Treatment burden, adherence, and quality of life in children with daily GH treatment in France

Abstract

Objective: To describe in a real-life setting the treatment burden and adherence, and quality of life of children treated with daily injections of growth hormone, and their relationship with treatment duration. Design: This non-interventional, multicenter, cross-sectional French study involved children aged 3 to 17 years treated with daily growth hormone injections. Methods: Based on a recent validated dyad questionnaire, the mean overall Life Interference total score (100=most interference) was described, with treatment adherence and quality of life (QOL) using the Quality of Life of Short Stature Youth questionnaire (100=best). All analyses were performed according to treatment duration prior to inclusion. Results: Among the 275/277 analyzed children, 166 (60.4%) had only growth hormone deficiency (GHD). In the GHD group, mean age was 11.7±3.2 years; median treatment duration was 3.3 years (IQR 1.8-6.4). The mean overall Life Interference total score was 27.7±20.7 (95% CI [24.2;31.2]), with non-significant correlation with treatment duration (p=0.1925). Treatment adherence was good (95.0% of children reported receiving >80% of planned injections over the last month); it slightly decreased with treatment duration (p=0.0364). Children overall QOL was good: 81.5±16.6 and 77.6±18.7 according to children and parents, respectively, but sub scores of the coping and treatment impact domains were <50. Similar results were observed in all patients independently of the condition requiring treatment. Conclusions: This real-life French cohort confirms the treatment burden of daily growth hormone injections, as previously reported in an interventional study.

Parents’ Experience of Administering Vosoritide: A Daily Injectable for Children with Achondroplasia

“…Parents also faced some challenges in terms of maintaining a consistent treatment schedule. This was especially true where parents wanted to travel, or with older children who took part in more activities evident in previous research amongst families administering growth hormone treatment [13][14][15][16]. This is something which will likely become more important in the future, as parents continue treatment over the course of years and seek to travel more and have a normal life.…”

“…This contrasts with previous research in similar areas [ 14 , 16 ]. Parents may be especially committed to persisting through challenges and identifying strategies for reducing burden to both themselves and their children given the lack of alternative treatment options [ 15 ]; however, this is a hypothesis which warrants further investigation. As in previous research, parents reported a reduction in barriers over time as the family adjusts to treatment [ 14 ].…”

“…Parents are impacted by watching their children experience fear or pain associated with an injection and worry about causing their child pain [13,14]. Research has also shown that growth hormone treatment interfered with daily activities and overnight travel, given the need to refrigerate the treatment, ultimately impacting dose timing and late or missed injections [13][14][15][16]. Whilst parents may report challenges with at-home injectables initially, these typically lessen over time and become part of daily life [14].…”