Department of Pediatrics, Tongji Hospital, Tongji Medical College, Huazhong University of Science and Technology, Wuhan, China.
Department of Endocrinology, Metabolism, and Genetics, Children's Hospital of Nanchang University and Jiangxi Provincial Children's Hospital, Nanchang, China.
Adv Ther. 2023 Oct;40(10):4574-4588. doi: 10.1007/s12325-023-02617-8. Epub 2023 Aug 16.
Triptorelin is available as 1- and 3-month prolonged-release (PR) formulations; at the time of the study, only the former was approved for central precocious puberty (CPP) in China. This study assessed the efficacy and safety of the triptorelin 3-month PR formulation in Chinese children with CPP.
In this 12-month, prospective, open-label, multicentre, single-arm study (NCT04736602), Chinese children (mean age [standard deviation (SD)], 7.6 ± 0.8 years) with CPP received triptorelin pamoate 15 mg on day 1 and at months 3, 6 and 9. The primary endpoint was the proportion with luteinizing hormone (LH) suppression (stimulated peak LH ≤ 3 IU/L after gonadotropin-releasing hormone [GnRH] stimulation) at month 3. Secondary endpoints included changes from baseline in hormone levels and clinical parameters, as well as safety assessments.
Overall, 32 children were enrolled, including three boys. LH suppression to prepubertal levels (≤ 3 IU/L) after GnRH stimulation was observed in 100%, 93.5% and 93.5% of participants at months 3, 6 and 12, respectively. Basal and peak LH and follicle-stimulating hormone levels were substantially suppressed at months 3, 6 and 12, and most participants showed sex hormone suppression. At months 6 and 12 respectively 92.9% and 89.3% of girls had stable breast development, and all boys had stable genital development. There was a decrease in mean growth velocity from baseline (8.96 cm/year) to months 3, 6 and 12 (8.07, 5.24 and 6.94 cm/year, respectively). The mean difference between bone and chronological age decreased from baseline (2.85 years) to month 12 (2.39 years). In girls, uterine length was stable or reduced at month 12; in boys, testicular volume was reduced. Triptorelin was well tolerated.
The triptorelin 3-month PR formulation demonstrated similar efficacy to that previously reported in non-Chinese patients with CPP and had an acceptable safety profile. This supports triptorelin 3-month PR as a viable option for Chinese children with CPP.
曲普瑞林有 1 个月和 3 个月的缓释（PR）制剂；在研究时，中国仅批准前者用于中枢性性早熟（CPP）。本研究评估了曲普瑞林 3 个月 PR 制剂在中国 CPP 儿童中的疗效和安全性。
在这项为期 12 个月、前瞻性、开放标签、多中心、单臂研究（NCT04736602）中，中国儿童（平均年龄[标准差]，7.6±0.8 岁）在第 1 天和第 3、6 和 9 个月接受曲普瑞林棕榈酸酯 15mg。主要终点是第 3 个月时 LH 抑制（促性腺激素释放激素[GnRH]刺激后 LH 激发峰值≤3IU/L）的比例。次要终点包括基线时激素水平和临床参数的变化，以及安全性评估。
总体而言，32 名儿童入组，包括 3 名男孩。在第 3、6 和 12 个月时，分别有 100%、93.5%和 93.5%的参与者在 GnRH 刺激后 LH 抑制至青春期前水平（≤3IU/L）。第 3、6 和 12 个月时，基础和峰值 LH 和卵泡刺激素水平均显著抑制，大多数参与者表现出性激素抑制。分别有 92.9%和 89.3%的女孩在第 6 和 12 个月时乳房发育稳定，所有男孩的生殖器发育均稳定。从基线（8.96cm/年）到第 3、6 和 12 个月，平均生长速度分别下降（8.07、5.24 和 6.94cm/年）。骨龄与实际年龄的平均差值从基线（2.85 岁）下降至第 12 个月（2.39 岁）。在女孩中，子宫长度在第 12 个月时稳定或减小；在男孩中，睾丸体积减小。曲普瑞林耐受性良好。
曲普瑞林 3 个月 PR 制剂在中国 CPP 儿童中的疗效与非中国患者先前报告的疗效相似，安全性可接受。这支持曲普瑞林 3 个月 PR 制剂作为中国 CPP 儿童的一种可行选择。
