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Follow-Up Study of Growth Hormone Therapy in Children with Kabuki Syndrome: Two-Year Treatment Results.
- Source :
-
Hormone research in paediatrics [Horm Res Paediatr] 2021; Vol. 94 (7-8), pp. 285-296. Date of Electronic Publication: 2021 Oct 04. - Publication Year :
- 2021
-
Abstract
- Introduction: Kabuki syndrome (KS) is a genetic disorder with characteristic facial dysmorphisms, short stature, hypertension, and obesity later in life. The aim of this study was to evaluate catch-up growth and cardiovascular markers before and during growth hormone (rhGH) treatment in KS children.<br />Methods: This prospective study included 18 children whose KS was genetically established. Each KS subject received rhGH for a period of 2 years. Several measurements were performed before and during treatment: anthropometry, glucose metabolism, lipid profile, markers for endothelial function, and low-grade inflammation.<br />Results: This study found an increase in delta height standard deviation score (SDS) for the whole group of 1.1 SDS after 2 years of rhGH treatment. Baseline metabolic profiles showed no cardiometabolic abnormalities in these children. Although 4 out of 18 children were obese, there were no signs of the metabolic syndrome. During rhGH treatment, serum low-density lipoprotein cholesterol concentrations decreased significantly (2.16-1.91 mmol/L, p = 0.04). Apolipoprotein B100 concentrations also showed a reduction after 24 months of treatment, but the other lipid and (apo)lipoprotein parameters did not change. While other endothelial function markers were stable, only vascular cell-adhesion molecule-1 concentrations increased (1,084-1,161 pg/mL, p < 0.01) during rhGH therapy. Furthermore, BMI and waist circumference improved during treatment. There were no signs of hypertension.<br />Conclusions: At baseline and during rhGH therapy, there were no signs of the metabolic syndrome. This is the first study demonstrating that rhGH treatment in KS children is a safe and effective therapy and that it positively influences linear height without exerting adverse effects on a wide array of cardiovascular risk markers.<br /> (© 2021 The Author(s) Published by S. Karger AG, Basel.)
- Subjects :
- Abnormalities, Multiple genetics
Follow-Up Studies
Hematologic Diseases genetics
Human Growth Hormone deficiency
Humans
Metabolic Syndrome
Prospective Studies
Vestibular Diseases genetics
Waist Circumference
Abnormalities, Multiple drug therapy
Body Height drug effects
Face abnormalities
Hematologic Diseases drug therapy
Human Growth Hormone administration & dosage
Human Growth Hormone pharmacology
Obesity drug therapy
Vestibular Diseases drug therapy
Subjects
Details
- Language :
- English
- ISSN :
- 1663-2826
- Volume :
- 94
- Issue :
- 7-8
- Database :
- MEDLINE
- Journal :
- Hormone research in paediatrics
- Publication Type :
- Academic Journal
- Accession number :
- 34607328
- Full Text :
- https://doi.org/10.1159/000519963