Evaluation of Patient Willingness to Switch from Daily rhGH to Long-Acting GH Treatment in Pediatric Growth Hormone Deficiency

What this Trial Is About

Researchers are evaluating the willingness of patients currently receiving recombinant human growth hormone (rhGH) treatment to switch to long-acting growth hormone (LAGH) therapies. These newer LAGH analogues, including lonapegsomatropin, somatrogon, and somapacitan, have been approved by the FDA for pediatric growth hormone deficiency and offer the possibility of less frequent injections compared to daily rhGH. This study aims to understand patient and family perspectives on changing treatment to these longer-acting options, considering potential benefits and challenges related to monitoring and side effects. The study involves Belgian and Luxembourgish pediatric patients on rhGH treatment for various conditions such as growth hormone deficiency, Turner Syndrome, chronic renal insufficiency, Prader-Willi syndrome, small for gestational age, Noonan Syndrome, and SHOX gene deficiency. Participants will complete a questionnaire during routine endocrinology consultations to assess their interest in switching from daily rhGH injections to LAGH therapies. This intervention is observational and does not involve changes to current medications. Participants will be assessed at baseline through the questionnaire to evaluate their propensity to switch treatment and to explore how responses vary by age, diagnosis, underlying conditions, and treatment duration. The study will collect data via these questionnaires during clinic visits without additional invasive procedures. The total participation period corresponds with routine clinical care, and there are no exclusion criteria, making this accessible to all eligible patients in the BELGROW Registry.

Evaluation of the Propensity of Patients Under rhGH to Envision a Modification of Their Treatment Regimen Toward LAGH