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 Louvain
500 participants
Apr 15, 2024
INTERVENTIONAL
Conditions
Summary
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.
Eligibility
Inclusion Criteria4
- 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
- years
- Free written or e-consent and oral consent
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Interventions
Recruitment will take place through routine paediatric endocrinology consultations with physicians registered with BELSPEED. These doctors will take the time to explain the questionnaire. And allow the patient to complete it.
Locations(1)
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NCT06542809