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

Cliniques universitaires Saint-Luc- Université Catholique de Louvain500 enrolled

Overview

Daily subcutaneous injections of rhGH can be burdensome for patients, leading to poor adherence and reduced growth outcomes. This has spurred the development of long-acting GH (LAGH) analogues that allow for weekly, biweekly, or monthly injections. Previous studies on LAGH analogues have demonstrated their non-inferiority compared to daily rhGH in terms of increasing growth velocity and improving body composition in children and adults with growth hormone deficiency (GHD), respectively, without significant and unexpected adverse events. Since 2020, three molecules have received approval from the Food and Drug Administration (FDA) for the treatment of pediatric GHD: lonapegsomatropin, somatrogon, and somapacitan. These LAGH analogues may offer better patient acceptance, improved tolerance, and greater therapeutic flexibility. However, these LAGH analogues could also be associated with potential clinical issues in terms of therapeutic monitoring, incidence and duration of side effects, and long-term safety due to a non-physiological GH profile. The introduction of these new LAGH products will require clinicians to identify optimal candidates for LAGH therapy and gain knowledge on monitoring and adjusting treatment.

Study Type

INTERVENTIONAL

Allocation

Purpose

SCREENING

Masking

NONE

Enrollment

500

Conditions

Growth Hormone Deficiency

Interventions

Eligibility

Sex: ALLMin age: 1 DayMax age: 18 Years

Medical Language ↔ Plain English

Inclusion Criteria: * Belgian and Luxembourgish patients currently under rhGh treatment in its various official indications (as per the Belgian RIZIV/INAMI monitor : growth hormone deficiency, Turner Syndrome, chronic renal insufficiency, Prader-Willi syndrome, Small for gestational age, Noonan Syndrome, SHOX gene deficiency) and included in the BELGROW Registry * Male -female * 0-18 years * Free written or e-consent and oral consent Exclusion Criteria: * No exclusion criteria

Outcomes

Primary Outcomes

Evaluate families and patients propensity with a questionnaire to envision a switch from rhGH treatment to LAGH

By distributing a questionnaire designed by the investigators, they are looking to know if patients and families are already aware of these new treatments, likelihood to improve adherence with LAGH or other benefits they might expect from it, potential to increase/decrease risk of treatment-related side effects, concerns they might have in changing the current treatment, what information would they want to receive from their practitioner.

Time frame: Baseline

Secondary Outcomes

Evaluate if families/patients response varies according to age, indication of rhGH treatment, any underlying pathology, and/or duration of rhGH treatment.

By distributing a questionnaire designed by the investigators.