Division of Nephrology And Hypertension, Mayo Clinic, Rochester, Minnesota.
Department of Biomedical Statistics and Informatics, Mayo Clinic, Rochester, Minnesota.
Am J Hematol. 2021 Apr 1;96(4):446-454. doi: 10.1002/ajh.26092. Epub 2021 Jan 28.
Three sets of criteria (International Society of Amyloidosis [ISA], Palladini and Kastritis) were independently developed for staging, progression and response criteria to predict renal survival in patients with AL amyloidosis. We evaluated these criteria using a cohort of 495 newly diagnosed AL amyloidosis patients with renal involvement using time to event competing risk analysis at baseline, 3, 6 and 12 months after treatment. Only Palladini and Kastritis had a staging system and both predicted a higher risk of end stage renal disease (ESRD) in the stage III vs stage I patients but only the Palladini model was predictive for stage II patients. At 3 months, risk of ESRD was significantly higher for Palladini and ISA renal progression (hazard ratio [HR] 2.8 [95% CI: 1.5-5.3, p = .001] and 2.5 [CI: 1.4-4.6, p = .004, respectively]), but renal response was not significantly protective; conversely, the risk of ESRD was not significantly higher for the Kastritis renal progression, but was significantly protective for the Kastritis renal responders (HR 0.38 [95% CI: 0.17-0.84], p = .017). Both progression and response with ISA, Palladini and Kastritis criteria were predictive of ESRD at 6 months and 12 months. While the Palladini staging criteria at baseline, and the ISA and Palladini criteria for progression at 3 months performed better than the Kastritis criteria at baseline and 3 months post-treatment, the Kastritis criteria performed better for response 3 months after treatment. All three sets of criteria performed well at and after 6 months post-treatment. These differences are important when choosing endpoints for clinical trials.
三套标准（国际淀粉样变性学会 [ISA]、Palladini 和 Kastritis）独立开发用于分期、进展和反应标准，以预测 AL 淀粉样变性患者的肾脏存活率。我们使用时间竞争风险分析在基线、治疗后 3、6 和 12 个月评估了这 495 名新诊断为 AL 淀粉样变性伴肾受累患者的队列中的这些标准。只有 Palladini 和 Kastritis 有分期系统，两者均预测 III 期患者比 I 期患者发生终末期肾脏疾病 (ESRD) 的风险更高，但只有 Palladini 模型对 II 期患者具有预测性。在 3 个月时，Palladini 和 ISA 肾脏进展的 ESRD 风险显著更高（危险比 [HR] 2.8 [95% CI：1.5-5.3，p =.001] 和 2.5 [CI：1.4-4.6，p =.004]），但肾脏反应没有显著保护作用；相反，Kastritis 肾脏进展的 ESRD 风险并不高，但 Kastritis 肾脏反应者的风险显著较低（HR 0.38 [95% CI：0.17-0.84]，p =.017）。ISA、Palladini 和 Kastritis 标准的进展和反应在 6 个月和 12 个月时均预测 ESRD。虽然 Palladini 基线分期标准以及 ISA 和 Palladini 标准在 3 个月时的进展标准优于 Kastritis 基线和治疗后 3 个月的标准，但 Kastritis 标准在治疗后 3 个月时的反应标准更好。这三组标准在治疗后 6 个月及以后均表现良好。在选择临床试验终点时，这些差异很重要。
