Weinshenker B G, Issa M, Baskerville J
Department of Neurology, Mayo Clinic and Mayo Foundation, Rochester, Minnesota, USA.
Arch Neurol. 1996 Apr;53(4):353-8. doi: 10.1001/archneur.1996.00550040093018.
The anticipated rate of short-term worsening of disability scores is the basis of power estimations in clinical trials of progressive multiple sclerosis (MS). While the clinician is most concerned in modifying the long-term outcome (eg, time to reach the Expanded Disability Status Scale [EDSS]6), the end points studied in clinical trials are those describing short-term outcome (eg, worsening of EDSS scores over 1 to 3 years). However, short-term outcome of MS may not be correlated with long-term outcome.
To validate previously published models predicting time to EDSS 6. To establish predictors of short-term outcome of MS.
The Ottawa, Ontario, Regional Multiple Sclerosis Clinic.
Two hundred fifty-nine patients were followed up prospectively by a single neurologist.
Actuarial analysis of time to reach EDSS 6 and change in EDSS scores over a follow-up period of 1 to 3 years.
The long-term outcome in the Ottawa population was more favorable than published data from London, Ontario. Predictions of time to EDSS 6 were not strongly correlated with the degree of short-term worsening over the follow-up period. Parameters associated with a higher probability of short-term worsening were proximity of the baseline EDSS score to 4.5 and duration of MS less than 20 years.
Baseline EDSS and duration of MS must be considered in the design of clinical trials of progressive MS.
在进行性多发性硬化症（MS）的临床试验中，残疾评分短期恶化的预期速率是功效估计的基础。虽然临床医生最关心的是改变长期结局（例如，达到扩展残疾状态量表[EDSS]6分的时间），但临床试验中研究的终点是描述短期结局的指标（例如，1至3年内EDSS评分的恶化情况）。然而，MS的短期结局可能与长期结局不相关。
验证先前发表的预测达到EDSS 6分时间的模型。确定MS短期结局的预测因素。
安大略省渥太华地区多发性硬化症诊所。
由一名神经科医生对259例患者进行前瞻性随访。
对达到EDSS 6分的时间进行精算分析，并在1至3年的随访期内分析EDSS评分的变化。
渥太华人群的长期结局比安大略省伦敦市公布的数据更有利。达到EDSS 6分时间的预测与随访期内短期恶化程度没有很强的相关性。与短期恶化可能性较高相关的参数是基线EDSS评分接近4.5以及MS病程少于20年。
在进行性MS的临床试验设计中，必须考虑基线EDSS和MS病程。
