Filiopoulos Vassilis, Kofotolios Ioannis, Vallianou Kalliopi, Karavasilis Efstratios, Ntounas Georgios, Melexopoulou Christina, Marinaki Smaragdi
Clinic of Nephrology and Renal Transplantation, Laiko General Hospital, Medical School of Athens, National and Kapodistrian University, 11527 Athens, Greece.
Medical Physics Laboratory, Democritus University of Thrace, 69100 Alexandroupolis, Greece.
J Clin Med. 2025 Sep 17;14(18):6537. doi: 10.3390/jcm14186537.
Tolvaptan, a vasopressin V2 receptor antagonist, is the only approved disease-modifying therapy for Autosomal Dominant Polycystic Kidney Disease (ADPKD), yet real-world data on its long-term effectiveness remain limited. In this single-center retrospective study, we evaluated 30 patients with ADPKD who received tolvaptan therapy for at least three years between 2019 and 2024. All patients met standard inclusion criteria and underwent serial magnetic resonance imaging to assess total kidney volume (TKV), along with longitudinal monitoring of renal function using estimated glomerular filtration rate (eGFR). At the end of follow-up, the median annual TKV growth rate was 4.27% (IQR: 1.39-7.98), which did not differ significantly from the predicted without treatment growth rate of 5.3% (95% CI: -2.75 to 0.69, = 0.194). Although the impact on TKV was limited, tolvaptan notably slowed the decline in kidney function, with a median eGFR of 65 mL/min/1.73 m at follow-up, compared to a predicted value of 60.8 mL/min/1.73 m (95% CI: -14.60 to -6.18, < 0.001), reflecting a 33.9% relative benefit. In 80% of patients, renal function after three years was better than predicted. These findings suggest that tolvaptan provides significant functional benefit in ADPKD patients in routine clinical practice, even in the absence of marked suppression in TKV growth and support its continued use in carefully selected individuals.
托伐普坦是一种血管加压素V2受体拮抗剂，是唯一获批用于治疗常染色体显性多囊肾病（ADPKD）的疾病修饰疗法，但关于其长期疗效的真实世界数据仍然有限。在这项单中心回顾性研究中，我们评估了30例ADPKD患者，这些患者在2019年至2024年期间接受了至少三年的托伐普坦治疗。所有患者均符合标准纳入标准，并接受了系列磁共振成像以评估总肾体积（TKV），同时使用估计肾小球滤过率（eGFR）对肾功能进行纵向监测。随访结束时，TKV的年增长率中位数为4.27%（四分位间距：1.39 - 7.98），与未经治疗预测的增长率5.3%无显著差异（95%置信区间：-2.75至0.69，P = 0.194）。尽管对TKV的影响有限，但托伐普坦显著减缓了肾功能下降，随访时eGFR中位数为65 mL/min/1.73m²，而预测值为60.8 mL/min/1.73m²（95%置信区间：-14.60至-6.18，P < 0.001），相对获益为33.9%。80%的患者在三年后的肾功能优于预测值。这些发现表明，在常规临床实践中，托伐普坦对ADPKD患者具有显著的功能益处，即使在TKV增长未得到明显抑制的情况下，也支持在精心挑选的个体中继续使用。
