Natural history and clinical features of the flail arm and flail leg ALS variants
Natural history and clinical features of the flail arm and flail leg ALS variants
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DOI：
10.1212/01.wnl.0000345041.83406.a2
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发表时间：
2009-03-24
期刊：
影响因子：
9.9
通讯作者：
Leigh, P. N.
中科院分区：
文献类型：
--
作者：
Wijesekera, L. C.;Mathers, S.;Leigh, P. N.
目的：明确肌萎缩侧索硬化症(ALS；运动神经元病)的臂肌型肌萎缩侧索硬化症和假性多发性神经炎的意义。方法：分析英国伦敦(1188例)和澳大利亚墨尔本(432例)的临床队列患者的生存情况。结果：在伦敦队列中，FA综合征占样本的11%，FL综合征占样本的6%。肢体肌萎缩侧索硬化症的中位生存期为35个月，延髓肌萎缩侧索硬化症的中位生存期为27个月，而FA综合征的中位生存期为61个月(p<；0.001)，FL综合征的中位生存期为69个月(p<；0.001)。在这个队列中，球起病的5年生存率为8.8%，肢体起病的5年生存率为20%，FA综合征的5年生存率为52%，FL综合征的5年生存率为%。在FA组，男性与女性的比例为4：1，而在其他肢体发病病例中，男性与女性的比例为2：1。排除下运动神经元FA和FL病例，进行性肌肉萎缩占样本的4%，其预后类似于典型的肢体ALS。在墨尔本队列中，肢体ALS的中位生存期为31个月，延髓ALS的中位生存期为27个月，FA综合征的中位生存期为66个月(p<；0.001)，FL综合征的中位生存期为71个月(p=0.001)。我们的研究结果强调了肌萎缩侧索硬化症FA和FL变异的临床和预后重要性。神经病学(R)2009；72：1087-1094
Objective: We sought to define the significance of brachial amyotrophic diplegia (flail arm syndrome [ FA]) and the pseudopolyneuritic variant (flail leg syndrome [ FL]) of amyotrophic lateral sclerosis (ALS; motor neuron disease).Methods: We analyzed survival in clinic cohorts in London, UK (1,188 cases), and Melbourne, Australia (432 cases). Survival from disease onset was analyzed using the Kaplan-Meier method and Cox proportional hazards model.Results: In the London cohort, the FA syndrome represented 11% and the FL syndrome 6% of the sample. Median survival was 35 months for limb onset and 27 months for bulbar onset ALS, whereas this was 61 months for FA syndrome (p < 0.001) and 69 months for FL syndrome (p < 0.001). Five-year survival in this cohort was 8.8% for bulbar onset, 20% for limb onset, 52% for FA syndrome, and 64% for FL syndrome. The ratio of men to women was 4: 1 in the FA group compared to 2: 1 in other limb onset cases. Excluding lower motor neuron FA and FL cases, progressive muscular atrophy comprised 4% of the sample and had a prognosis similar to typical limb onset ALS. In the Melbourne cohort, median survival for limb onset ALS was 31 months, bulbar onset 27 months, FA syndrome 66 months (p < 0.001), and FL syndrome 71 months (p = 0.001).Conclusions: The flail arm (FA) and flail leg (FL) syndromes had significantly better survival than typical amyotrophic lateral sclerosis (ALS) or progressive muscular atrophy cases that were not classified as FA or FL. Our findings underline the clinical and prognostic importance of the FA and FL variants of ALS. Neurology (R) 2009; 72: 1087-1094