Healthcare professionals' perspectives towards the digitalisation of paediatric growth hormone therapies: expert panels in Italy and Korea.

Introduction: To analyse the perspectives of healthcare professionals (HCPs) regarding the acceptance of digital health solutions for growth hormone (GH) deficiency care. This study identified factors impacting HCPs' intent to use and recommend digital solutions supporting recombinant-human growth hormone (r-hGH) therapy in Italy and Korea with a use case of connected drug delivery system (Aluetta® with Smartdot™) integrated in a platform for GH treatment support (the Growzen™ digital health ecosystem). Methods: Participatory workshops were conducted in Rome, Italy, and Seoul, Korea, to collect the perspectives of 22 HCPs on various predefined topics. HCPs were divided into two teams, each moderated by a facilitator. The workshops progressed in five phases: introduction of the project and experts, capturing views on the current context of digitalisation, perceived usefulness and ease of use of Aluetta® with Smartdot™, exploration of the perception of health technology evolution, and combined team recommendations. Data shared by HCPs on technology acceptance were independently analysed using thematic analysis, and relevant findings were shared and validated with experts. Results: HCPs from both Italy and Korea perceived Aluetta® with Smartdot™ and the Growzen™ based digital health ecosystem as user-friendly, intuitive, and easy-to-use solutions. These solutions can result in increased adherence, a cost-effective healthcare system, and medication self-management. Although technology adoption and readiness may vary across countries, it was agreed that using digital solutions tailored to the needs of users may help in data-driven clinical decisions and strengthen HCP-patient relationships. Conclusion: HCPs' perspectives on the digitalisation in paediatric GH therapies suggested that digital solutions enable automatic, real-time injection data transmission to support adherence monitoring and evidence-based therapy, strengthen HCP-patient relationships, and empower patients throughout the GH treatment process.

Metabolic and Growth Outcome of Two-year Growth Hormone Treatment in Children Born Small for Gestational Age: A Retrospective Study.

BACKGROUND: Children born Small for Gestational Age (SGA) without early catch-up growth may show impaired growth rate, adult height, and metabolic profile [1]. Growth Hormone (GH) is recommended for their treatment, and it has been shown to have positive effects on growth and metabolic profile and good tolerability [2]. OBJECTIVE: The study aimed to evaluate the auxological and metabolic effects and safety of GH treatment in SGA children. METHODS: 34 SGA children (15 F, 19 M; mean age: 8.72 ± 2.48 yrs) treated with GH (starting dosage: 32.24 ± 2.88 mcg/kg/die) were evaluated every six months for 24 months with growth and metabolic parameters. RESULTS: After two years, SGA children showed a significant improvement in height, weight, and growth rate, already evident after six months (p < 0.001), with a constant, significant improvement in height throughout the treatment (p ≤ 0.03 T0 vs. T12, T12 vs. T24). Conversely, although significantly higher than baseline at each visit (p < 0.001), the growth rate significantly decreased from 6 to 18 months (p ≤ 0.015 T6 vs. T12, T12 vs. T18). During the follow-up, an increase in glycemia (p ≤ 0.042 vs. T12, T18) and urycemia (p ≤ 0.01 vs. T12, T18, and T24) and a decrease in AST (p ≤ 0.021 vs. T12, T18, and T24) and LDL cholesterol (p = 0.03 vs. T24) were observed. Overall, treatment was found to be well tolerated, with poor compliance being the most frequent adverse event (11.8%) and no reported hyperglycemia. CONCLUSION: In conclusion, GH can be considered an effective, safe treatment in SGA children, improving height and growth rate, although proper metabolic follow-up is required.

Auxological and metabolic effects of long-term treatment with recombinant growth hormone in children born small for gestational age: a retrospective study.

RATIONALE: Children born small for gestational age (SGA) not showing catch-up during the first two years of life reportedly show an impaired growth rate and adult height, as well as a worse metabolic outcome, mainly in terms of glycemic and lipid profile, compared to general population. In SGA children with short stature, treatment with recombinant growth hormone (GH) is currently recommended until adolescence; therefore, it may last long-term. STUDY METHODS: The aim of the current study was to evaluate the auxological and metabolic effects and the safety of long-term recombinant GH treatment in SGA children. The study included 15 SGA children (5 F, 10 M; mean age: 6.78 yrs) treated with GH for at least 48 months. Growth and metabolic parameters, including glycemic and lipid profile, transaminases, and urycemia, were collected every six months. RESULTS: Compared to baseline, SGA children showed a significant improvement in height, weight, and growth rate after four yaers of treatment with GH (p ≤ 0.002), being already evident after six months of treatment (p < 0.001). Noteworthy, patients showed a constant, significant improvement in height throughout the treatment, as it was significantly higher at each follow-up compared to the previous one, until 42 months of treatment, except at 30 months of treatment (p < 0.001 T6VST12; p < 0.01 T12VST18, T18VST24; p < 0.05 T30VST36, T36VST42). Considering metabolic parameters, compared to baseline, a recurring increase in glycemia (p ≤ 0.028 vs T30, T36, and T48) and decrease in AST (p ≤ 0.035 vs T36, T42, and T48) and an occasional decrease in LDL cholesterol (p ≤ 0.04 vs T24 and T42) and triglycerides (p = 0.008 vs T18) and increase in urycemia (p = 0.034 vs T42). Considering safety profile, treatment was well tolerated, as the most frequently reported adverse event was poor compliance (20%); no hyperglycemia, hypercholesterolemia or hyperstransaminasemia occurred throughout the treatment, CONCLUSIONS: Long-term GH treatment showed to be effective in improving height and growth rate in SGA children, with a positive impact of metabolic profile and a safety profile, although glycemia should be carefully monitored over time.