Chong Jiehan, Harris Tess, Ong Albert C M
Academic Nephrology Unit, Department of Infection, Immunity, and Cardiovascular Disease, Medical School, University of Sheffield, Sheffield, UK.
Sheffield Kidney Institute, Sheffield Teaching Hospitals NHS Foundation Trust, Sheffield, UK.
Clin Kidney J. 2022 Aug 26;16(1):61-68. doi: 10.1093/ckj/sfac190. eCollection 2023 Jan.
Tolvaptan, a vasopressin V2 receptor antagonist, was approved in 2015 by the UK National Institute for Health and Care Excellence for use in patients with autosomal dominant polycystic kidney disease (ADPKD) and rapid disease progression. Simultaneous guidance was issued by the UK Kidney Association (UKKA) to facilitate national implementation.
Data on tolvaptan prescribing in England was obtained through the National Health Service (NHS) Digital, a national survey of all 77 adult kidney units, and the implementation of UKKA guidance was evaluated at an expert PKD centre.
A regional variation of up to 4-fold for tolvaptan prescribing in England was found. Despite most kidney units following UKKA guidance, centre-based estimates of eligible or treated patient numbers were highly variable. Retrospective evaluation at an expert PKD centre revealed that in a cohort demonstrating rapid estimated glomerular filtration rate (eGFR) decline, 14% would not be eligible for tolvaptan by Mayo imaging classification and more than half (57%) would not be eligible by Predicting Renal Outcome in Polycystic Kidney Disease score. The 3-year discontinuation rate was higher than expected (56%), the majority (70%) due to aquaretic symptoms. In patients taking tolvaptan for at least 2 years, 81% showed a reduction in the rate of eGFR decline compared with baseline, with earlier disease associated with positive treatment response.
Real-world data have revealed a much higher regional variation in tolvaptan prescribing for ADPKD in England than expected. We propose further investigation into the factors responsible for this variation.
托伐普坦是一种血管加压素V2受体拮抗剂，2015年被英国国家卫生与临床优化研究所批准用于常染色体显性多囊肾病（ADPKD）且疾病进展迅速的患者。英国肾脏协会（UKKA）同时发布了指导意见以促进其在全国的应用。
通过英国国家医疗服务体系（NHS）数字化部门获取英格兰地区托伐普坦处方数据，对所有77个成人肾脏科室进行全国性调查，并在一家专业多囊肾病中心评估UKKA指导意见的实施情况。
发现英格兰地区托伐普坦处方量存在高达4倍的地区差异。尽管大多数肾脏科室遵循UKKA指导意见，但基于中心的符合条件或接受治疗患者数量的估计差异很大。一家专业多囊肾病中心的回顾性评估显示，在一个估计肾小球滤过率（eGFR）快速下降的队列中，根据梅奥成像分类，14%的患者不符合使用托伐普坦的条件，而根据多囊肾病肾脏预后预测评分，超过一半（57%）的患者不符合条件。3年停药率高于预期（56%），大多数（70%）是由于利水症状。在服用托伐普坦至少2年的患者中，81%的患者与基线相比eGFR下降速率降低，疾病越早与积极的治疗反应相关。
实际数据显示，英格兰地区ADPKD患者托伐普坦处方的地区差异比预期大得多。我们建议进一步调查造成这种差异的因素。
