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| Name | Class |
|---|---|
| The Belux Society for Pediatric Endocrinology and Diabetology (BELSPEED) | UNKNOWN |
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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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Belgian and Luxembourgish patients currently under rhGh treatment | Experimental | Patient population studied are 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 Number. |
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Questionnaire | Other | 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. |
| Measure | Description | Time Frame |
|---|---|---|
| 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. | Baseline |
| Measure | Description | Time Frame |
|---|---|---|
| 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. | Baseline |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Philippe Lysy, MD, PhD | Contact | 027641370 | +32 | philippe.lysy@saintluc.uclouvain.be |
| Laure Boutsen, MD | Contact | +3227641933 | laure.boutsen@saintluc.uclouvain.be |
| Name | Affiliation | Role |
|---|---|---|
| Philippe Lysy, MD, PhD | Cliniques universitaires Saint-Luc- Université Catholique de Louvain | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Cliniques Universitaires Saint-Luc | Recruiting | Brussels | Woluwe-saint-lambert | 1200 | Belgium |
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| ID | Term |
|---|---|
| D004393 | Dwarfism, Pituitary |
| ID | Term |
|---|---|
| D004392 | Dwarfism |
| D001848 | Bone Diseases, Developmental |
| D001847 | Bone Diseases |
| D009140 | Musculoskeletal Diseases |
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| ID | Term |
|---|---|
| D011795 | Surveys and Questionnaires |
| ID | Term |
|---|---|
| D003625 | Data Collection |
| D004812 | Epidemiologic Methods |
| D008919 | Investigative Techniques |
| D017531 | Health Care Evaluation Mechanisms |
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| D001849 |
| Bone Diseases, Endocrine |
| D007018 | Hypopituitarism |
| D010900 | Pituitary Diseases |
| D007027 | Hypothalamic Diseases |
| D001927 | Brain Diseases |
| D002493 | Central Nervous System Diseases |
| D009422 | Nervous System Diseases |
| D004700 | Endocrine System Diseases |
| D011787 | Quality of Health Care |
| D017530 | Health Care Quality, Access, and Evaluation |
| D011634 | Public Health |
| D004778 | Environment and Public Health |