ECE2022 Eposter Presentations Adrenal and Cardiovascular Endocrinology (131 abstracts)
A phase III trial of recombinant human growth hormone in pediatric patients with growth failure caused by chronic kidney disease
Hong Xu 1 , Xiaoshan Tang 1 , Qian Shen 1 , Jing Chen 1 , Xiaoyan Fang 1 , Aihua Zhang 2 , Fei Zhao 2 , Qiuxia Chen 2 , Wenyan Huang 3 , Ping Wang 3 , Liwen Sun 3 , Huijie Xiao 4 , Ke Xu 4 , Xiaorong Liu 5 , Zhi Chen 5 , Chaoying Chen 6 , Juan Tu 6 , Yubing Wu 7 , Xiuli Wang 7 , Jianhua Mao 8 , Zhihong Lu 8 , Jingjing Wang 8 , Xiaojing Nie 9 , Zihua Yu 10 , Jun Huang 9 , Cuihua Liu 11 , Guanghai Cao 11 , Yufeng Li 12 , Yaju Zhu 12 , Jianjiang Zhang 13 , Miao Wang 13 , Mo Wang 14 & Haiping Yang 14
1Children’s Hospital of Fudan University, Shanghai Kidney Development and Pediatric Kidney Disease Research Center, National Children’s Medical Center, Department of Nephrology, Shanghai, China; 2Nanjing Children’s Hospital Affiliated to Nanjing Medical University, Department of Nephrology, Nanjing, China; 3Shanghai Children’s Hospital, Shanghai Jiao Tong University, Department of Nephrology and Rheumatology, Shanghai, China; 4Peking University First Hospital, Department of Pediatrics, Beijing, China; 5Beijing Children’s Hospital affiliated with Capital Medical University, Department of Nephrology, Beijing, China; 6Children’s Hospital Affiliated to Capital Institute of Pediatrics, Department of Nephrology, Beijing, China; 7Shengfing Hospital Affiliated to China Medical University, Department of Pediatric, Shenyang, China; 8The Children’s Hospital, Zhejiang University School of Medicine, Department of Nephrology, Hangzhou, China; 9The 900th Hospital of Joint Logistic Support Force, Department of Pediatrics, Fuzhou, China; 10Fujian Children’s Hospital, Affiliated Hospital of Fujian Medical University, Department of Nephrology, Rheumatology and Immunology, Fuzhou, China; 11Children’s Hospital Affiliaten of Zhenzhou University, Department of Nephrology and Rheumatology, Zhenzhou, China; 12Xinhua Hospital Affiliated to Shanghai Jiao Tong University School of Medicine, Department of Pediatric Nephrology, Shanghai, China; 13The First Affiliated Hospital of Zhengzhou University, Department of Pediatric, Zhengzhou, China; 14Children’s Hospital of Chongqing Medical University, Department of Nephrology and Rheumatology, Chongqing, China.
Background and objective: Children with chronic kidney disease (CKD) have impaired growth that leads to short stature in adulthood and treatment with recombinant human growth hormone (rhGH) is associated with improved growth. This study aimed to evaluate the efficacy and safety of daily rhGH (Jintropin®) in children in China with growth failure caused by CKD prior to transplantation.
Methods: Prepubertal patients (2–13 years old) with CKD-related short stature were enrolled in this multicenter, randomized, open-label, negative-controlled study (NCT03535415). Eligible patients were randomized 1:1 to receive daily rhGH 0.05 mg/kg/day administered subcutaneously or control (no rhGH given, only relevant examinations were performed) for up to 52 weeks. The primary endpoint was improvement in (Δ) height standard deviation score (HT SDS). Secondary endpoints included improvements in height velocity (HV), bone maturation (bone age [BA]/chronological age [CA]), insulin-like growth factor 1 standard deviation score (IGF-1 SDS), IGF-1/IGF binding protein-3 (IGFBP-3) molar ratio, and safety.
Results: A total of 68 patients were randomized to either treatment or control; 11 patients (treatment: 7; control: 4) dropped out, 8 (treatment: 5; control: 3) of whom due to transplantation. At week 52, ΔHT SDS from baseline was 0.747±0.579 (P<0.001) and 0.173±0.470 (P=0.039) in the treatment and control groups, respectively (intergroup P<0.001). Least-squares mean difference between both groups was 0.582 (95% confidence interval 0.323–0.842). Statistically significant improvements were also observed in those who received treatment compared with those in the control group for ΔHV (7.021±3.795 cm/year vs 2.566±3.577 cm/year; intergroup P<0.001), ΔIGF-1 SDS (1.697±2.098 vs –0.171±1.506; intergroup P<0.001), ΔIGF-1/IGFBP-3 molar ratio (0.046±0.073 vs 0.001±0.041; intergroup P<0.001), and Δheight (9.87±2.89 cm vs 6.43±2.72 cm; intergroup P<0.001). Δ(BA/CA) was 0.041±0.074 and 0.008±0.079 in the treatment and control groups, respectively (intergroup P=0.118). Most treatment-emergent adverse events (TEAEs) were mild to moderate; 19 patients (treatment: 10; control: 9) experienced serious adverse events, and 5 in the treatment group temporarily discontinued rhGH due to TEAEs. 7 reported drug-related TEAEs, which included elevated blood insulin, scoliosis, glycosuria, musculoskeletal discomfort, hyperinsulinemia, and abnormal liver function.
Conclusion: Daily rhGH 0.05 mg/kg per day for 52 weeks was effective and well tolerated in children with short stature caused by CKD. At the end of the study, significant improvements in ΔHT SDS, ΔHV, ΔIGF-1 SDS, ΔIGF-1/IGFBP-3 molar ratio, and Δheight were observed with treatment.
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Endocrine Abstracts
ISSN 1470-3947 (print) | ISSN 1479-6848 (online)
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