Prognostic factors in ALS: A critical review

被引:802
作者
Chio, Adriano [1 ,2 ]
Logroscino, Giancarlo [3 ]
Hardiman, Orla [4 ,5 ]
Swingler, Robert [6 ]
Mitchell, Douglas [7 ]
Beghi, Ettore [8 ]
Traynor, Bryan G. [9 ]
机构
[1] Univ Turin, Dept Neurosci, I-10126 Turin, Italy
[2] San Giovanni Battista Hosp, Turin, Italy
[3] Harvard Univ, Dept Epidemiol, HSPH, Boston, MA 02115 USA
[4] Beaumont Hosp, Dept Neurol, Dublin 9, Ireland
[5] Trinity Coll Dublin, Inst Neurosci, Dublin, Ireland
[6] Univ Dundee, Ninewells Hosp & Med Sch, Dept Neurol, Dundee DD1 9SY, Scotland
[7] Royal Preston Hosp, Preston MND Care & Res Ctr, Preston, Lancs, England
[8] Ist Ric Farmacol Mario Negri, Milan, Italy
[9] NIMH, Sect Dev Genet Epidemiol, NIH, Bethesda, MD 20892 USA
来源
AMYOTROPHIC LATERAL SCLEROSIS | 2009年 / 10卷 / 5-6期
关键词
Amyotrophic lateral sclerosis; survival; prognostic factors; trials; AMYOTROPHIC-LATERAL-SCLEROSIS; MOTOR-NEURON DISEASE; PERCUTANEOUS ENDOSCOPIC GASTROSTOMY; PREDICTS SURVIVAL-TIME; GROWTH-FACTOR-I; NATURAL-HISTORY; CLINICAL-TRIAL; NONINVASIVE VENTILATION; PROLONGED SURVIVAL; MND/ALS PATIENTS;
D O I
10.3109/17482960802566824
中图分类号
R74 [神经病学与精神病学];
学科分类号
摘要
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.
引用
收藏
页码:310 / 323
页数:14
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