文章：

经验证的复合器官和血液反应模型，用于早期评估轻链淀粉样变性治疗结果

A validated composite organ and hematologic response model for early assessment of treatment outcomes in light chain amyloidosis

原文发布日期：2020-04-14

DOI: 10.1038/s41408-020-0306-5

类型: Article

开放获取: 是

英文摘要：

Newly diagnosed AL amyloidosis patients were evaluated to develop a model for early assessment of treatment benefit at 6 months, integrating both hematologic (HR) and organ response (OR) assessment (testing cohort, Mayo: n = 473; validation cohort, Pavia: n = 575). Multiple OR were assessed as follows: All OR (AOR): response in all organs, mixed OR (MOR): response in some organs, no OR (NOR)]. AOR rates at 6 months improved with deepening HR; complete response (CR; 38%, 35%), very good partial response (VGPR; 30%, 26%), and partial response (PR; 16%, 21%), respectively. A composite HR/OR (CHOR) model was developed using incremental scoring based on hazard ratios with scores of 0–3 for HR (0—CR, 1—VGPR, 2—PR, 3—no response) and 0–2 for OR (0—AOR, 1—MOR, 2—NOR). Patients could be divided into two distinct CHOR groups (scores 0–3 and 4–5), with median OS in group 1 and group 2: Not reached vs. 34 months, p < 0.001 [Mayo] and 87 vs. 23 months, p < 0.001 [Pavia]. In conclusion, we developed a model that can assess multiple organs concurrently, and integrate both HR and OR assessments to determine early clinical benefit with treatment, which may be used as a surrogate end-point in trials and to compare outcomes with different therapies.
 

摘要翻译： 

新诊断的AL淀粉样变性患者被评估以开发一个模型，用于早期评估6个月时的治疗获益，整合了血液学反应（HR）和器官反应（OR）评估（测试队列，梅奥：n=473；验证队列，帕维亚：n=575）。多种器官反应评估如下：所有器官反应（AOR）：所有器官有反应，混合器官反应（MOR）：部分器官有反应，无器官反应（NOR）。6个月时的AOR率随着HR的加深而改善：完全缓解（CR；38%，35%）、非常好的部分缓解（VGPR；30%，26%）和部分缓解（PR；16%，21%）。开发了一个复合HR/OR（CHOR）模型，使用基于风险比的增量评分，HR评分为0-3（0—CR，1—VGPR，2—PR，3—无反应），OR评分为0-2（0—AOR，1—MOR，2—NOR）。患者可以分为两个不同的CHOR组（得分0-3和4-5），组1和组2的中位OS：未达到 vs. 34个月，p<0.001 [梅奥] 和87 vs. 23个月，p<0.001 [帕维亚]。总之，我们开发了一个可以同时评估多个器官、并整合HR和OR评估以确定治疗早期临床获益的模型，该模型可能用作试验中的替代终点，并用于比较不同疗法的结果。

原文链接：

A validated composite organ and hematologic response model for early assessment of treatment outcomes in light chain amyloidosis