NATURAL-HISTORY OF AMYOTROPHIC-LATERAL-SCLEROSIS IN A DATABASE POPULATION - VALIDATION OF A SCORING SYSTEM AND A MODEL FOR SURVIVAL PREDICTION
NATURAL-HISTORY OF AMYOTROPHIC-LATERAL-SCLEROSIS IN A DATABASE POPULATION - VALIDATION OF A SCORING SYSTEM AND A MODEL FOR SURVIVAL PREDICTION
复制标题
DOI：
10.1093/brain/118.3.707
复制
发表时间：
1995-06-01
期刊：
影响因子：
14.5
通讯作者：
APPEL, SH
中科院分区：
文献类型：
--
作者：
HAVERKAMP, LJ;APPEL, V;APPEL, SH
超过1200名运动神经元疾病患者已被仔细诊断，随访，并纳入详细的数据库描述疾病的特征。在这些患者中，831例被确定为表现出典型的散发性肌萎缩侧索硬化症（ALS）。这些患者的疾病进展已经用我们的评分系统进行了随访，ALS评分被证实是生存率的重要协变量。首次出现症状时的年龄、从首次出现症状到进入ALS诊所的时间延迟以及呼吸功能的变化率也被确定为生存率的显著协变量。这些措施，适用于考克斯比例风险模型，被用来开发一个数学模型，预测生存时间的ALS，这被证明是高度准确的80%的患者在中等风险。对于这些患者，开发了第二个模型，该模型在最初的观察期后准确预测ALS患者在ALS总评分中下降一定数量点的时间。这种验证允许通过比较相对较小的治疗患者组与该历史对照组来进行ALS中药物治疗的初始试验，该历史对照组基于胎儿ALS评分的特定递减的预测时间模型。
Over 1200 patients with motor neuron disease have been carefully diagnosed, followed, and included in a detailed database delineating characteristics of the disease. Of these patients, 831 were identified as exhibiting typical sporadic amyotrophic lateral sclerosis (ALS). The progression of the disease in these patients has been followed with our scoring system and the ALS score was verified as a significant covariate of survival. Age at first symptom, delay from first symptom to entering ALS clinic, and rate of change of respiratory function were also identified as significant covariates of survival. These measures, applied to the Cox proportional hazards model, were used to develop a mathematical model for prediction of survival time in ALS, which proved highly accurate for the 80% of patients at intermediate risk. For those patients, a second model was developed which accurately predicts, after an initial period of observation, the time over which ALS patients will decline a set number of points in total ALS score. Such validation permits initial trials for drug therapies in ALS by comparison of relatively small groups of treated patients to this historical control group, based on the model of predicted time to a particular decrement in fetal ALS score.