Evaluation of efficacy and safety of long-acting PEGylated recombinant human growth hormone (Jintrolong) for patients with growth hormone deficiency

Hanze Du; Danning Wu; Pengfei Yi; Xi Bai; Yunyun Luo; Hongbo Yang; Shi Chen; Hui Pan; Huijuan Zhu

doi:10.1515/jpem-2021-0735

Article

Evaluation of efficacy and safety of long-acting PEGylated recombinant human growth hormone (Jintrolong) for patients with growth hormone deficiency

Hanze Du , Danning Wu , Pengfei Yi , Xi Bai , Yunyun Luo , Hongbo Yang , Shi Chen , Hui Pan and Huijuan Zhu

Published/Copyright: March 3, 2022

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From the journal Journal of Pediatric Endocrinology and Metabolism Volume 35 Issue 4

Abstract

Objectives

This study aimed to compare the efficacy, safety and metabolic effects of once-weekly pegylated human growth hormone (PEG-rhGH) with daily rhGH in patients with growth hormone deficiency (GHD).

Design

48 patients enrolled in this 12-month single-center, open-label, prospective, randomized controlled trial is allocated to PEG-rhGH 0.12 mg/kg/w and 0.20 mg/kg/w treatment. Besides, the control group allocated 23 patients treated with daily rhGH 0.28 mg/kg/w matched with sex, age, and baseline IGF-1 levels. The primary endpoint included height velocity (HV) and IGF-1 increase at the end of treatment. Other parameters associated with growth, metabolism and safety were also monitored.

Results

In terms of HV increase, the efficacy of PEG-rhGH dosed at 0.12 mg/kg/w and 0.20 mg/kg/w was comparable to that of daily rhGH dosed at 0.28 mg/kg/w after 3, 6 and 12 treatment (p>0.05). IGF-1 concentration and IGF-1 SDS were both elevated significantly at 3, 6 and 12 months (p=0.000) into normal range following PEG-rhGH treatment. BMI SDS elevated after PEG-rhGH treatment for 3, 6 and 12 months (p=0.000). HbA1c elevated after 3 and 12 months (p=0.009) and METS-IR elevated after 6 months (p=0.019) compared with baseline. The differences of other metabolic indexes (such as blood glucose, blood lipid, etc.) have no statistical significance (p>0.05). No severe adverse event was observed among the three groups.

Conclusions

The efficacy and safety were promising and comparable between once-weekly PEG-rhGH and daily rhGH injection within 12 months. The negative influence on glucose homeostasis needed attention and monitoring.

Keywords: GHD; PEG-rhGH; rhGH replacement therapy

Corresponding authors: Professor Hui Pan, Department of Endocrinology, Key Laboratory of Endocrinology of National Health Commission, State Key Laboratory of Complex Severe and Rare Diseases Peking Union Medical College Hospital, Chinese Academy of Medical Science and Peking Union Medical College, 100730, Beijing, China E-mail: panhui20111111@163.com; and Professor Huijuan Zhu, Department of Endocrinology, Key Laboratory of Endocrinology of National Health Commission, Translation Medicine Centre, Peking Union Medical College Hospital, Peking Union Medical College, Chinese Academy of Medical Sciences, 100730, Beijing, China E-mail: shengxin2004@163.com

Hanze Du and Danning Wu contributed equally. Hui Pan and Huijuan Zhu contributed equally.

Funding source: the National Key Program of Clinical Science

Award Identifier / Grant number: WBYZ2011-873

Funding source: the CAMS Innovation Fund for Medical Science

Award Identifier / Grant number: 2016-I2M-1-008

Research funding: This work was supported by the National Key Program of Clinical Science (Grant No: WBYZ2011-873) and the CAMS Innovation Fund for Medical Science (Grant No: 2016-I2M-1-008).
Author contribution: All authors have accepted responsibility for the entire content of this manuscript and approved its submission.
Competing interests: Authors state no conflict of interest.
Informed consent: Informed consent was obtained from all individuals included in this study.
Ethical approval: The local Institutional Review Board deemed the study exempt from review.

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Received: 2021-12-07

Accepted: 2022-02-07

Published Online: 2022-03-03

Published in Print: 2022-04-26

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Articles in the same Issue

https://doi.org/10.1515/jpem-2021-0735

Keywords for this article

GHD; PEG-rhGH; rhGH replacement therapy