Zhou Li, Wei Xiansen, Wang Boya, Xu Qianqian, Li Wenge
Department of Nephrology, China-Japan Friendship Hospital, Beijing, China.
Department of Ultrasound, China-Japan Friendship Hospital, Beijing, China.
Transl Androl Urol. 2024 Oct 31;13(10):2307-2321. doi: 10.21037/tau-24-448. Epub 2024 Oct 28.
Tolvaptan has been shown to be effective in the treatment of autosomal dominant polycystic kidney disease (ADPKD). However, there is limited evidence regarding optimal dosing and its application within the Chinese population. In this study, we aimed to determine whether a lower tolvaptan dose could effectively control ADPKD in Chinese patients.
This retrospective, single-center cohort study was conducted in a real-world setting and included all patients newly diagnosed with rapidly progressive ADPKD who initiated tolvaptan treatment and maintained it for at least 12 months. Data were collected at baseline and at 1, 2, 4, 8, and 12 months after treatment initiation. Patients began with morning/evening tolvaptan doses of 7.5 mg/7.5 mg, and the dose was subsequently adjusted based on effectiveness and tolerability. The patients were categorized by baseline estimated glomerular filtration rate (eGFR) and final daily tolvaptan dose. Changes in eGFR and other key physiological indicators after treatment were compared within each group.
The study included 43 patients with ADPKD, of whom 20 were female, with a median age of 34.3 years (range, 16-85 years). At 12 months, eGFR improved by 5.48 mL/min/1.73 m [95% confidence interval (CI): 2.68-8.29] (P<0.001) compared to baseline. Significant improvements were observed in patients with baseline eGFR levels of 30-59, 60-89, and ≥90 mL/min/1.73 m (P=0.007, 0.045, and 0.02, respectively), as well as in medium and high dose groups (P=0.002 and 0.02, respectively). At 12 months, the annual height-adjusted total kidney volume (HtTKV) growth slope decreased by -0.17 %/year (95% CI: -0.33 to -0.01) (P=0.04). Significant decreases were observed in patients with an eGFR of 30-59 mL/min/1.73 m (P=0.008) and in the medium dose group (P=0.03). Thirst was reported in 22 (51.2%) patients, all of whom experienced mild symptoms. No liver-associated adverse events were noted.
Tolvaptan is well tolerated at low initial doses in Chinese patients with ADPKD. Significant improvements in eGFR and reduced HtTKV growth were observed in the overall population and across various baseline eGFR and final dose groups.
托伐普坦已被证明可有效治疗常染色体显性遗传性多囊肾病（ADPKD）。然而，关于最佳剂量及其在中国人群中的应用，证据有限。在本研究中，我们旨在确定较低剂量的托伐普坦是否能有效控制中国患者的ADPKD。
本回顾性单中心队列研究在真实环境中进行，纳入所有新诊断为快速进展性ADPKD并开始接受托伐普坦治疗且持续至少12个月的患者。在治疗开始时的基线以及治疗开始后1、2、4、8和12个月收集数据。患者起始的托伐普坦剂量为早上/晚上各7.5毫克，随后根据疗效和耐受性进行调整。根据基线估计肾小球滤过率（eGFR）和最终每日托伐普坦剂量对患者进行分类。比较每组治疗后eGFR和其他关键生理指标的变化。
该研究纳入了43例ADPKD患者，其中20例为女性，中位年龄为34.3岁（范围16 - 85岁）。与基线相比，12个月时eGFR改善了5.48毫升/分钟/1.73平方米[95%置信区间（CI）：2.68 - 8.29]（P<0.001）。基线eGFR水平为30 - 59、60 - 89和≥90毫升/分钟/1.73平方米的患者以及中高剂量组均观察到显著改善（分别为P = 0.007、0.045和0.02）。12个月时，年度身高校正后的总肾体积（HtTKV）生长斜率下降了-0.17%/年（95% CI：-0.33至-0.01）（P = 0.04）。eGFR为30 - 59毫升/分钟/1.73平方米的患者以及中剂量组观察到显著下降（分别为P = 0.008和P = 0.03）。22例（51.2%）患者报告有口渴，所有患者症状均较轻。未观察到与肝脏相关的不良事件。
在中国ADPKD患者中，低初始剂量的托伐普坦耐受性良好。在总体人群以及不同基线eGFR和最终剂量组中均观察到eGFR显著改善且HtTKV生长减缓。
