Judith L Ross1, Peter A Lee2, Robert Gut3, John Germak4. 1. Department of Pediatrics, Thomas Jefferson University, Philadelphia, PA, United States; Nemours/Alfred I. duPont Hospital for Children, Wilmington, DE, United States. Electronic address: judith.ross@jefferson.edu. 2. Department of Pediatrics, Milton S. Hershey Medical Center, Penn State College of Medicine, Hershey, PA, United States. Electronic address: plee@hmc.psu.edu. 3. Novo Nordisk Inc., Plainsboro, NJ, United States. Electronic address: rogu@novonordisk.com. 4. Novo Nordisk Inc., Plainsboro, NJ, United States. Electronic address: jgak@novonordisk.com.
Abstract
OBJECTIVE: This study aimed to assess attainment of genetic height potential after long-term growth hormone (GH) treatment in GH-naïve children diagnosed with isolated growth hormone deficiency (IGHD), multiple pituitary hormone deficiency (MPHD), born small for gestational age (SGA), or idiopathic short stature (ISS) enrolled in the American Norditropin® STUDIES: Web-enabled Research (ANSWER) Program. DESIGN: Children with IGHD (n=2884), MPHD (n=200), SGA (n=481), or ISS (n=733) with baseline height standard deviation score (HSDS)≤-2 were assessed over 5 years of GH treatment for mean HSDS, change in HSDS (ΔHSDS), and corrected HSDS (HSDS-target HSDS). RESULTS: Mean HSDS and corrected HSDS significantly increased to close to target height across all diagnostic groups after 5 years of GH treatment (P<0.0001). ∆HSDS at year 5 increased for all groups (IGHD: 1.8; MPHD: 2.1; SGA: 1.8; ISS: 1.6). Among patients who continued GH for 5 years, mean insulin-like growth factor-I (IGF-I) SDS increased to within normal range across all groups. Body mass index (BMI) SDS remained relatively stable in all diagnostic groups. Bone age (BA) increased, and the mean BA to chronological age (BA/CA) ratio reached or approached 1 across diagnostic groups over 5 years of GH treatment. CONCLUSIONS: Long-term GH therapy resulted in a significant increase in mean HSDS and corrected HSDS from baseline values in all diagnostic groups. The observed increase in mean corrected HSDS is consistent with growth that approached the patients' genetic height potential, although complete height gains will be evaluated at the attainment of final height.
OBJECTIVE: This study aimed to assess attainment of genetic height potential after long-term growth hormone (GH) treatment in GH-naïve children diagnosed with isolated growth hormone deficiency (IGHD), multiple pituitary hormone deficiency (MPHD), born small for gestational age (SGA), or idiopathic short stature (ISS) enrolled in the American Norditropin® STUDIES: Web-enabled Research (ANSWER) Program. DESIGN:Children with IGHD (n=2884), MPHD (n=200), SGA (n=481), or ISS (n=733) with baseline height standard deviation score (HSDS)≤-2 were assessed over 5 years of GH treatment for mean HSDS, change in HSDS (ΔHSDS), and corrected HSDS (HSDS-target HSDS). RESULTS: Mean HSDS and corrected HSDS significantly increased to close to target height across all diagnostic groups after 5 years of GH treatment (P<0.0001). ∆HSDS at year 5 increased for all groups (IGHD: 1.8; MPHD: 2.1; SGA: 1.8; ISS: 1.6). Among patients who continued GH for 5 years, mean insulin-like growth factor-I (IGF-I) SDS increased to within normal range across all groups. Body mass index (BMI) SDS remained relatively stable in all diagnostic groups. Bone age (BA) increased, and the mean BA to chronological age (BA/CA) ratio reached or approached 1 across diagnostic groups over 5 years of GH treatment. CONCLUSIONS: Long-term GH therapy resulted in a significant increase in mean HSDS and corrected HSDS from baseline values in all diagnostic groups. The observed increase in mean corrected HSDS is consistent with growth that approached the patients' genetic height potential, although complete height gains will be evaluated at the attainment of final height.
Keywords:
Adult height; Born small for gestational age; Human growth hormone; Idiopathic short stature; Isolated growth hormone deficiency; Multiple pituitary hormone deficiency; Short stature
Authors: Juan P López-Siguero; Maria J Martínez-Aedo; Jose Antonio Bermúdez de la Vega; Jordi Bosch-Muñoz; Alfonso M Lechuga-Sancho; Triana Villalobos Journal: Clin Endocrinol (Oxf) Date: 2021-12-09 Impact factor: 3.523