Prognostic factors in ALS: A critical review.

Prognostic factors in ALS: A critical review.
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DOI:
10.3109/17482960802566824
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发表时间:
2009-10
期刊:
Amyotrophic lateral sclerosis : official publication of the World Federation of Neurology Research Group on Motor Neuron Diseases
影响因子:
--
通讯作者:
Eurals Consortium
Eurals Consortium
中科院分区:
其他
文献类型:
--
作者:
Chiò A;Logroscino G;Hardiman O;Swingler R;Mitchell D;Beghi E;Traynor BG;Eurals Consortium

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我们进行了一项系统回顾,总结了目前关于ALS生存相关因素的知识,并评估了这些数据对临床试验设计的影响。从发病到死亡的中位生存时间为20至48个月,但10-20%的ALS患者的生存期超过10年。年龄较大和球结膜炎的发病一直被报道为预后较差。关于性别、诊断延迟和埃尔埃斯科里亚标准的数据相互矛盾。症状进展率被认为是一个独立的预后因素。心理社会因素、FTD、营养状况和呼吸功能也与ALS结局相关。肠内营养对生存率的影响尚不清楚,而NIPPV已被发现可提高生存率。目前还没有成熟的进展生物标志物,尽管在不久的将来可能会出现一些。这些发现对未来试验的设计具有相关意义。除了发病类型外,随机化还应考虑入组时的年龄、呼吸状态和入组前疾病进展的指标。替代试验设计可包括使用自然史对照、所谓的治疗分配最小化方法和无效方法。
We have performed a systematic review to summarize current knowledge concerning factors related to survival in ALS and to evaluate the implications of these data for clinical trials design. The median survival time from onset to death ranges from 20 to 48 months, but 10–20% of ALS patients have a survival longer than 10 years. Older age and bulbar onset are consistently reported to have a worse outcome. There are conflicting data on gender, diagnostic delay and El Escorial criteria. The rate of symptom progression was revealed to be an independent prognostic factor. Psychosocial factors, FTD, nutritional status, and respiratory function are also related to ALS outcome. The effect of enteral nutrition on survival is still unclear, while NIPPV has been found to improve survival. There are no well established biological markers of progression, although some are likely to emerge in the near future. These findings have relevant implications for the design of future trials. Randomization, besides the type of onset, should take into account age, respiratory status at entry, and a measure of disease progression pre-entry. Alternative trial designs can include the use of natural history controls, the so-called minimization method for treatment allocation, and the futility approach.
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影响因子: 9.9
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发表时间: 2003-01-01
期刊: AMYOTROPHIC LATERAL SCLEROSIS AND OTHER MOTOR NEURON DISORDERS
影响因子: --
作者:
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