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[帕多瓦大学肾脏病科使用托伐普坦治疗常染色体显性多囊肾病患者:对生活质量、疗效及安全性的影响]

[Tolvaptan in ADPKD patients at the University of Padova Nephrology Unit: impact on quality of life, efficacy and safety].

作者信息

Cirella Irene, di Vico Valentina, Rigato Matteo, Bugnotto Beatrice, Baldini Anastasio Paola, Campo Daniela, Carraro Gianni, Calò Lorenzo A

机构信息

UOC Nefrologia, Dialisi e Trapianto, Dipartimento di Medicina (DIMED), Università di Padova.

出版信息

G Ital Nefrol. 2022 Jun 20;39(3):2022-vol3.

Abstract

Autosomal dominant polycystic kidney disease (ADPKD) is responsible of the 10% of the dialysis patients. Tolvaptan is a consolidate option for treatment of ADPKD patients; it slows renal deterioration rate and cysts' growth, although its acquaretic effects often impact on quality of life (QoL) and treatment adherence. Few studies have documented the tolvaptan long term efficacy and safeness profiles and, mostly, the impact of treatment with tolvaptan on patients' QoL. Our study aimed to investigate in 13 ADPKD patients of our cohort the differences in terms of QoL before and after the start of treatment via a questionnaire based on SF-36 and PSQI tests, integrated with other original questions. In addition we have also examined the tolvaptan long term efficacy and safeness profiles. The results of our study show that tolvaptan does not significantly reduce patients QoL notwithstanding its expected acquaretic effects, the only reported side effects. Finally, the average annual renal deterioration rate was lower in patients treated with tolvaptan than in the others. Relevant limits of our study are the small number of selected patients and the relative short study duration. However, on one hand, the results of our study provide further information to the few data available in literature; on the other hand, they may serve as a useful working hypothesis for further studies with a larger number of patients enrolled and an extended study duration. They would demonstrate the absence of significant impact of tolvaptan on patients' QoL.

摘要

常染色体显性多囊肾病(ADPKD)导致了10%的透析患者。托伐普坦是治疗ADPKD患者的一种巩固性选择;它能减缓肾脏恶化速度和囊肿生长,尽管其利水作用常常影响生活质量(QoL)和治疗依从性。很少有研究记录托伐普坦的长期疗效和安全性概况,而且大多未涉及托伐普坦治疗对患者QoL的影响。我们的研究旨在通过基于SF - 36和PSQI测试并结合其他原创问题的问卷,调查我们队列中的13名ADPKD患者在开始治疗前后的QoL差异。此外,我们还研究了托伐普坦的长期疗效和安全性概况。我们的研究结果表明,尽管托伐普坦有预期的利水作用且是唯一报告的副作用,但它并未显著降低患者的QoL。最后,接受托伐普坦治疗的患者的平均每年肾脏恶化率低于其他患者。我们研究的相关局限性在于所选患者数量少以及研究持续时间相对较短。然而,一方面,我们的研究结果为文献中现有的少量数据提供了更多信息;另一方面,它们可能为进一步纳入更多患者并延长研究持续时间的研究提供有用的工作假设。这些研究将证明托伐普坦对患者QoL没有显著影响。

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