Department of Nephrology, Hannover Medical School, Hannover, Germany.
Department of Human Genetics, Hannover Medical School, Hannover, Germany.
Am J Nephrol. 2019;50(4):281-290. doi: 10.1159/000502634. Epub 2019 Aug 30.
Tolvaptan can slow down renal function decline in autosomal dominant polycystic kidney disease (-ADPKD). While there is consensus across international recommendations that the drug should only be used in patients with high risk of rapid progression, identification criteria for rapid progression vary. Here, we investigated different assessment strategies using a real-life ADPKD cohort.
Observational retrospective cohort analysis. The study included 131 ADPKD patients aged 19-78 years who were referred to the Hannover Medical School outpatient clinic for evaluation of tolvaptan treatment. Six different assessment strategies for tolvaptan eligibility were tested for each patient. Comparative analysis for different assessments was performed in the total study population, the subpopulation with available computed tomography/magnetic resonance imaging data, and the genotyped subpopulation.
Comparing 6 assessment strategies revealed strong variations in the individual selection processes resulting in treatment recommendations for 14.5-64.9% of patients. The highest patient number was selected by the Scottish and the lowest by the Japanese approach. Few patients had positive recommendations by all 6 systems, but strong congruency was observed between the Scottish, U.K. and Canadian patient selection. The lowest number of overlapping patients was found between the Japanese and the ERA-EDTA selection. Important discrepancies were also found between the ERA-EDTA and the U.S. system due to different emphases on parameters of kidney function versus kidney volume. Limitations of the study included the restricted sample size, heterogeneity in parameter availability and lack of outcome data.
The study draws attention to important discrepancies between different decision algorithms for tolvaptan eligibility in ADPKD patients.
托伐普坦可减缓常染色体显性多囊肾病（ADPKD）患者的肾功能下降速度。尽管国际建议一致认为该药物仅应在快速进展风险高的患者中使用，但快速进展的识别标准存在差异。在此，我们使用真实的 ADPKD 队列研究了不同的评估策略。
观察性回顾性队列分析。该研究纳入了 131 名年龄在 19-78 岁之间的 ADPKD 患者，他们因评估托伐普坦治疗而被转诊至汉诺威医学院门诊。对每位患者进行了 6 种不同的托伐普坦合格评估策略测试。在总研究人群、有可用计算机断层扫描/磁共振成像数据的亚组和基因分型亚组中，对不同评估方法进行了比较分析。
比较 6 种评估策略发现，个体选择过程存在较大差异，导致 14.5-64.9%的患者有不同的治疗建议。苏格兰方法选择的患者人数最多，而日本方法选择的患者人数最少。少数患者得到了所有 6 个系统的阳性推荐，但苏格兰、英国和加拿大的患者选择具有很强的一致性。日本和 ERA-EDTA 的选择之间发现的重叠患者最少。由于对肾功能与肾体积参数的重视程度不同，ERA-EDTA 和美国系统之间也存在重要差异。该研究的局限性包括样本量有限、参数可用性的异质性以及缺乏结局数据。
该研究提请注意 ADPKD 患者托伐普坦合格性的不同决策算法之间存在重要差异。
