Treatment of Pediatric Growth Hormone Deficiency With Oral Secretagogues Revisited
| Autor: | Mabel Yau, Robert Rapaport |
|---|---|
| Rok vydání: | 2021 |
| Předmět: |
medicine.medical_specialty
Growth hormone stimulation test business.industry Endocrinology Diabetes and Metabolism secretagogues Ibutamoren LUM-201 Growth hormone–releasing hormone medicine.disease Short stature Growth hormone deficiency GH Endocrinology predictive enrichment markers (PEM) Internal medicine receiver operating characteristic (ROC) medicine medicine.symptom growth hormone deficiency (GHD) business Clinical Research Articles AcademicSubjects/MED00250 |
| Zdroj: | Journal of the Endocrine Society |
| ISSN: | 2472-1972 |
| Popis: | Context We hypothesize, based on the degree of residual hypothalamic-pituitary function, that some, but not all, children with growth hormone deficiency (GHD) may have beneficial growth responses to the orally administered growth hormone (GH) secretagogue LUM-201. Objective To determine if pretreatment testing can identify predictive enrichment markers (PEM) for subjects with adequate residual function who are responsive to LUM-201. Methods We performed an analysis of a completed, randomized, placebo-controlled trial of LUM-201, a GH secretagogue receptor agonist, in which all randomized subjects had pretreatment testing. This international multicenter study conducted in pediatric endocrinology clinics included 68 naïve-to-treatment, prepubertal children with established diagnoses of GHD. Outcome measures included the sensitivity, specificity, and predictive accuracy of potential markers to predict 6-month growth responses to oral LUM-201 and daily rhGH. Results Two PEM were identified for use in defining PEM-positive status: (1) baseline insulin-like growth factor I (IGF-I) concentration >30 ng/mL and (2) peak GH response of ≥5 ng/mL upon administration of single-dose LUM-201. PEM-positive status enriches a population for better growth responses to LUM-201. PEM-negative status enriches a population for better growth responses to rhGH. Conclusion Combined, the peak GH response to single-dose LUM-201 and the baseline IGF-I concentration are effective PEMs for 6-month growth responses to LUM-201 and rhGH in prepubertal children with GHD. |
| Databáze: | OpenAIRE |
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