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Lancet oncol:肌萎缩侧索硬化症患者预后的预测模型

2018-03-27 MedSci MedSci原创

肌萎缩侧索硬化症(ALS)是一种持续性进展的病史多变的致命性运动神经疾病。现尚无精确的模型可预测该疾病的病程和预后,使得对个体患者的风险评估和咨询、临床试验分层以及进行干预的时机均复杂化。现有研究人员尝试弥补这一空白。Henk-Jan Westeneng等人从多个国家的14个专业ALS中心(每一个来源作为一个队列)获得相关患者资料。所有患者在排除其他疾病后确诊ALS,并根据修订版El Escori

肌萎缩侧索硬化症(ALS)是一种持续性进展的病史多变的致命性运动神经疾病。现尚无精确的模型可预测该疾病的病程和预后,使得对个体患者的风险评估和咨询、临床试验分层以及进行干预的时机均复杂化。现有研究人员尝试弥补这一空白。

Henk-Jan Westeneng等人从多个国家的14个专业ALS中心(每一个来源作为一个队列)获得相关患者资料。所有患者在排除其他疾病后确诊ALS,并根据修订版El Escorial标准予以分类。研究人员对作为综合存活预后的潜在预测指标的16个患者特征进行评估,并在最大的基于人群的数据集上进行反向消除,来确定预测指标。选择在70%以上的样本中出现的预测指标来建立多变量Royston-Parmar模型来预测个体患者的复杂存活预后。

收集了1992年1月1日-2016年9月22日期间的数据,中位随访时间97.5个月(IQR 52.9-168.5)。预测模型纳入8个候选预测指标:是否是从延髓发病(HR 1.71,95% CI 1.63-1.79)、发病年龄(1.03,1.03-1.03)、确诊或疑诊ALS(1.47,1.39-1.55)、诊断延迟(0.52,0.51-0.53)、最大肺活量(HR 0.99,0.99–0.99)、进展率(6.33,5.92-6.76)、额颞叶痴呆(1.34,1.20-1.50)和C9orf72重复扩增的存在(1.45,1.31-1.61)(所有p<0.0001)。模型外部预测精确度的c统计值为0.78(95% CI 0.77-0.80;95% PI 0.74-0.82),标定斜率1.01(95% CI 0.95-1.07;95% PI 0.83-1.18)。该模型用于定义5组从症状开始到复杂存活预后的时间中最明显的中位预测(SE)和观察(SE)时间:极短17.7(0.20)、1.65(0.23);短25.3(0.06)、25.2(0.35);中等32.2(0.09)、32.8(0.46);长43.7(0.21)、44.6(0.74);极长91.0(1.84)、85.6(1.96)。

Henk-Jan Westeneng等人已开发出一种用于预测无气管造瘘或无创通气不超过23小时的欧洲ALS患者预后的外部预测模型。该模型可应用于个体化患者管理、咨询以及未来临床试验设计,但为了最大限度的提高效应和预防伤害,该模型现仅供医生使用。

原始出处:

Henk-Jan Westeneng,et al.Prognosis for patients with amyotrophic lateral sclerosis: development and validation of a personalised prediction model. The Lancet Oncology. March 26,2018.https://doi.org/10.1016/S1474-4422(18)30089-9

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    2019-01-18 minlingfeng
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    2019-03-08 chendoc252
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  5. 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  6. 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    2018-03-31 lofter

    学习了感谢分享

    0

  7. 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  8. 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  9. 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    2018-03-28 smartxiuxiu

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