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J Endocrinol Invest. 2018 Apr;41(4):475-483. doi: 10.1007/s40618-017-0786-8. Epub 2017 Nov 4.

Recombinant growth hormone therapy for prepubertal children with idiopathic short stature in Korea: a phase III randomized trial.

Author information

1
Department of Pediatrics, Samsung Medical Center, Sungkyunkwan University School of Medicine, 81 Irwon-ro, Gangnam-gu, Seoul, 06351, Republic of Korea.
2
Department of Pediatrics, Seoul St. Mary's Hospital, The Catholic University of Korea, Seoul, Republic of Korea.
3
Department of Pediatrics, Kyungpook National University Hospital, Daegu, Republic of Korea.
4
Department of Pediatrics, Korea University Anam Hospital, Seoul, Republic of Korea.
5
Department of Pediatrics, Seoul National University Children's Hospital, Seoul, Republic of Korea.
6
Department of Pediatrics, Ajou University Hospital, Suwon, Republic of Korea.
7
Department of Pediatrics, Severance Hospital, Onsei University Health System, Seoul, Republic of Korea.
8
Department of Pediatrics, Inje University Busan Paik Hospital, Busan, Republic of Korea.
9
Department of Pediatrics, Chonnam National University Hospital, Gwangju, Republic of Korea.
10
Department of Pediatrics, Chungbuk National University Hospital, Cheongju, Republic of Korea.
11
Data Management and Clinical Statistics Team, Dong-A ST Co., LTD, Seoul, Republic of Korea.
12
Department of Pediatrics, Samsung Medical Center, Sungkyunkwan University School of Medicine, 81 Irwon-ro, Gangnam-gu, Seoul, 06351, Republic of Korea. nadri1217@naver.com.
13
Department of Pediatrics, Medical Genetics Clinic and Laboratory, Asan Medical Center Children's Hospital, University of Ulsan College of Medicine, 88 Olympic-ro 43-gil, Songpa-gu, Seoul, 05505, Republic of Korea. hwyoo@amc.seoul.kr.

Abstract

PURPOSE:

Several studies have evaluated the effects of growth hormone (GH) on auxological and biochemical parameters in children with non-GH-deficient, idiopathic short stature (ISS). This study evaluated the efficacy and safety of Growtropin®-II (recombinant human GH) in Korean patients with ISS.

METHODS:

This was a 1-year, open-label, multicenter, phase III randomized trial of Growtropin®-II in Korean patients with ISS. In total, 70 prepubertal subjects (39 males, 31 females) between 4 and 12 years of age were included in the study. All patients were naive to GH treatment.

RESULTS:

Annual height velocity was significantly higher in the treatment group (10.68 ± 1.95 cm/year) than the control group (5.72 ± 1.72, p < 0.001). Increases in height and weight standard deviation scores (SDSs) at 26 weeks were 0.63 ± 0.16 and 0.64 ± 0.46, respectively, for the treatment group, and 0.06 ± 0.15 and 0.06 ± 0.28, respectively, for the control group (p < 0.001). Serum insulin-like growth factor (IGF-1) and insulin-like growth factor binding protein-3 (IGFBP-3) increased significantly in the treatment group at week 26 compared to baseline. However, the SDS for body mass index (BMI) at 26 weeks did not change significantly in either group. Growtropin®-II was well tolerated and safe over 1 year of treatment.

CONCLUSIONS:

One-year GH treatment for prepubertal children with ISS demonstrated increased annualized velocity, height and weight SDSs, and IGF-1 and IGFBP-3 levels, with a favorable safety profile. Further evaluations are needed to determine the optimal dose, final adult height, and long-term effects of ISS treatment.

KEYWORDS:

Clinical trial; Growth hormone; Idiopathic short stature

PMID:
29103133
PMCID:
PMC5852196
DOI:
10.1007/s40618-017-0786-8
[Indexed for MEDLINE]
Free PMC Article

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