PDF(Pubmed)
Abstract：
Tolvaptan is the first disease-modifying drug proven to slow eGFR decline in high-risk patients with ADPKD. However, barriers from the patient perspective to its use in real-life settings have not been systemically examined in a large cohort. This was a single-center, retrospective study of 523 existing or new patients with ADPKD followed at the Center for Innovative Management of PKD in Toronto, Ontario, between January 1, 2016 to December 31, 2018. All patients underwent clinical assessment including total kidney volume measurements and Mayo Clinic Imaging Class (MCIC). Those who were deemed to be at high risk were offered tolvaptan with their preference (yes or no) and reasons for their choices recorded. Overall, 315/523 (60%) patients had MCIC 1C-1E; however, only 96 (30%) of them were treated with tolvaptan at their last follow-up. Among these high-risk patients, those not treated versus treated with tolvaptan were more likely to have a higher eGFR (82 ± 26 vs. 61 ± 27 ml/min/1.73 m2), CKD stages 1-2 (79% vs. 41%), and MCIC 1C (63% vs. 31%). The most common reasons provided for not taking tolvaptan were lifestyle preference related to the aquaretic effect (51%), older age ≥ 60 (12%), and pregnancy/family planning (6%). In this real-world experience, at least 60% of patients with ADPKD considered to be at high risk for progression to ESKD by imaging were not treated with tolvaptan; most of them had early stages of CKD with well-preserved eGFR and as such, were prime targets for tolvaptan therapy to slow disease progression. Given that the most common reason for tolvaptan refusal was the concern for intolerability of the aquaretic side-effect, strategies to mitigate this may help to reduce this barrier to tolvaptan therapy.
摘要：
托伐普坦是第一种被证明可以减缓ADPKD高危患者eGFR下降的疾病改善药物。然而,从患者的角度来看，在现实生活中使用它的障碍尚未在大型队列中进行系统检查。这是一个单一的中心，在多伦多PKD创新管理中心对523名ADPKD现有或新患者进行回顾性研究，安大略省,2016年1月1日至2018年12月31日。所有患者均接受临床评估，包括总肾脏体积测量和梅奥诊所成像分类（MCIC）。那些被认为是高风险的人被提供托伐普坦与他们的偏好（是或否）和他们的选择记录的原因。总的来说,315/523(60%)患者患有MCIC1C-1E；然而，其中只有96例(30%)在最后一次随访时接受托伐普坦治疗.在这些高危患者中，与托伐普坦治疗相比，未治疗的患者更有可能具有较高的eGFR（82±26vs.61±27ml/min/1.73m2),CKD1-2期（79%vs.41%)，和MCIC1C(63%与31%）。不服用托伐普坦的最常见原因是与饮水效应相关的生活方式偏好（51％），年龄≥60岁（12%），怀孕/计划生育(6%)。在这个现实世界的经验，至少60%的ADPKD患者被认为有进展为ESKD的高危患者未接受托伐普坦治疗;他们中的大多数患有CKD的早期阶段,eGFR保存良好,是托伐普坦治疗减缓疾病进展的主要目标。鉴于托伐普坦拒绝治疗的最常见原因是担心水副作用的不能容忍，缓解这种情况的策略可能有助于减少托伐普坦治疗的障碍.
