Ann Pediatr Endocrinol Metab.  2018 Mar;23(1):43-50. 10.6065/apem.2018.23.1.43.

Design of the long-term observational cohort study with recombinant human growth hormone in Korean children: LG Growth Study

• Chung S
• Yoo JH
• Choi JH
• Rhie YJ
• Chae HW
• Kim JH
• Hwang IT
• Shin CH
• Kim EY
• Lee KH

• KMID: 2409790
• DOI: http://doi.org/10.6065/apem.2018.23.1.43

Abstract

PURPOSE
Regarding recombinant human growth hormone (rhGH) use in the pediatric population, no long-term follow-up data are available for Korean patients. To fill in the gap of knowledge, a registry study (LG Growth Study) was initiated to assess the safety and effectiveness of four types of rhGH products in real-life settings.
METHODS
A total of 4,000 children will be registered and prospectively followed up at 6-month intervals until 2 years after epiphyseal closure to collect data on treatment and adverse events, with primary interest in malignancies and growth outcomes.
RESULTS
As of 22 March 2017, approximately 50% (2,024) of the target number of patients have been included in the analysis set: growth hormone deficiency, 1,297 (64.1%); idiopathic short stature, 315 (15.6%); small for gestational age, 206 (10.2%); Turner syndrome, 197 (9.7%); and chronic renal failure, 9 (0.4%). At baseline, median age (years) was 8 (interquartile range [IQR], 5-11); 52% (1,048) were boys; and the majority were at Tanner stage I (83% based on breast/external genitalia, 97% on pubic hair). Median height standard deviation score was -2.26 (IQR, -2.69 to -2.0), and median bone age delay (years) was -1.46 (IQR, -2.26 to -0.78).
CONCLUSIONS
This registry study will provide the opportunity to assess the risk of malignancies as well as the general safety data in Korean pediatric patients receiving rhGH. In addition, the long-term effectiveness of rhGH and comparative data between different disease entities will provide practical insight on the standard rhGH treatment.

Keyword

Growth hormone deficiency; Turner syndrome; Chronic renal failure; Small for gestational age; Idiopathic short stature

MeSH Terms

Child*
Cohort Studies*
Follow-Up Studies
Genitalia
Gestational Age
Growth Hormone
Human Growth Hormone*
Humans*
Kidney Failure, Chronic
Prospective Studies
Turner Syndrome
Growth Hormone
Human Growth Hormone

Figure

• 

  Fig. 1. Study scheme. Study data collection is prospective in nature (A); however, if a patient is registered during the course of recombinant human growth hormone (rhGH) treatment, pretreatment (baseline) information and data obtained thereafter are collected retrospectively (B). For all patients including those who are withdrawn from the treatment before reaching adult height, the follow-up (F/U) continues until 2 years are passed after epiphyseal closure.

• 

  Fig. 2. Patient distribution. Since the initiation of the study on 9 November 2011, around 50% of the targeted number of patients’ data have been pooled into the database as of 22 March 2017.

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