Objectives: This study aimed to evaluate the long-term safety and effectiveness of recombinant human growth hormone (rhGH) (Eutropin® Inj.., Eutropin®Pen Inj., Eutropin®AQ Inj., and Eutropin®Plus Inj., LG Chem, Ltd.) based on the interim analysis of a 7-year accumulated data of the LG Growth Study (LGS) in Korean pediatric patients with growth disorders including growth hormone deficiency (GHD), Turner Syndrome (TS), idiopathic short stature (ISS), small for gestational age (SGA) and chronic renal failure (CRF).
Methods: The LGS, an open-label, multicenter (total 73 sites), observational study has begun in November 2011, and the data collected until February 2019 was used for an interim analysis. All adverse events (AEs) were reported for safety, and effectiveness was assessed by height velocity, height standard deviation score (SDS) and inulin like growth factor-1 (IGF-1).
Results: During the 7-year study, a total of 3,144 patients were enrolled, and 2,871 patients (male 52%, female 48%) were analyzed for safety. Of those patients, 1,853 (64.5%) had GHD, 426 (14.8%) were diagnosed with ISS, 348 (12.1%) were born SGA, 235 (8.2%) had TS and 9 (0.3%) had CRF. The mean age at the screening was 7.8 years and the mean treatment duration was 3.6 years. Adverse events (AEs) were reported in 894 (31.1%) patients, most AEs were mild, and 163 (5.7%) adverse drug reactions (ADRs) was reported by physicians including headache (0.9%), injection site pain (0.7%), scoliosis (0.6%), and hypothyroidism (0.3%). A total of 27 neoplasms were reported in 24 (0.8%) patients, most were benign and/or non-related to rhGH. When the effectiveness was assessed in 1,094 patients followed-up for 12 months, height velocity was 8.9±1.9, 8.7±1.6, 9.0±1.6, 7.3±1.8, and 8.3±2.1 cm/year in GHD, ISS, SGA, TS and CRF patients, respectively. When 4-year follow-up effectiveness was assessed in GHD and TS patients, height SDS significantly increased from baseline to 4 years in GHD patients (from -2.9±0.9 to -1.3±1.1, P<0.0001) and TS patients (from -3.2±0.8 to -2.2±0.8, P<0.0001), respectively. Total IGF-1 SDS significantly increased (from -0.7±1.1 to 0.8±1.7 at 12 months, P<0.0001) and maintained within 0 to 2 SDS throughout the study period.
Conclusions: The incidence of AEs was low, and rhGH therapy was well tolerated. During 4 years of rhGH treatment, significant improvement in height SDS was confirmed in Korean pediatric patients with GHD and TS.
19 - 21 Sep 2019
European Society for Paediatric Endocrinology