Predictors of delay in the diagnosis and clinical trial entry of amyotrophic lateral sclerosis patients: a population-based study.
ABSTRACT The El Escorial and the revised Airlie House diagnostic criteria for amyotrophic lateral sclerosis (ALS) were introduced to select patients for clinical trials. Heterogeneity of clinical presentation at onset and delay in diagnosis may decrease the likelihood for trial entry.
Identify risk factors for delay in the diagnosis and trial exclusion.
ALS incident cases were identified with El Escorial (EEC) and Airlie House criteria (AHC) through a population-based registry established in Puglia, Southern Italy, in the years 1998-99.
130 ALS incident cases were diagnosed with a median interval between onset of symptoms and diagnosis of 9.3 months and not different across both EEC and AHC categories. Twenty percent of cases were not eligible for clinical trials according to the AHC. About 5% of subjects in this series died with only lower motor neuron signs. Predictors for delay in the diagnosis were age between 65 and 75 years and spinal onset while fasciculations and cramps as first symptoms were predictors of exclusion from trials.
In this population-based series, diagnostic delay was longer in subjects with spinal onset and age between 65 and 75 and fasciculation as first symptoms. About 80% of incident cases were trial eligible with AHC criteria. However, a significant number of subjects with ALS, characterized by a limited spread of signs, were not trial eligible while alive.
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ABSTRACT: Cases of adult-onset idiopathic motor neuron disease (MND) identified from January 1970 through December 1986 were studied in a defined area of California. The patients were followed prospectively throughout the illness in 99% of cases. Among 708 cases aged 25-74 years at onset, the most common type (86%) was typical, sporadic amyotrophic lateral sclerosis (SporALS). The risk of bulbar onset and shorter survival times increased with age in both men and women. About 4%, mainly younger men, experienced unusually long courses with milder paralysis, but could not be identified early in the illness. They probably represent one extreme of the ALS spectrum rather than a distinct subtype. Familial ALS (FamALS) was diagnosed in 7%. It developed earlier in life but ran a slightly longer course, which suggests a different disease process. Overall there was a statistically significant predominance of males, especially in 17 cases (2%) of progressive muscular atrophy (PMA). There were 26 cases (4%) classified as primary lateral sclerosis (PLS). Progressive bulbar palsy was not found; that diagnosis usually denotes merely the bulbar onset of ALS.Journal of the Neurological Sciences 09/1993; 118(1):48-55. · 2.24 Impact Factor
- Journal of the Neurological Sciences 06/1995; 129 Suppl:11-2. · 2.24 Impact Factor
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ABSTRACT: The Scottish Motor Neuron Disease Register (SMNDR) is a prospective, collaborative, population-based project which has been collecting data on incident patients since 1989. In this report we present the clinical features of 229 patients with motor neuron disease (218 sporadic and 11 familial) diagnosed in 1989 and 1990 and compare their prognosis with previous studies of survival. The overall 50% survival from symptom onset was 2.5 years (95% CI, 2.2-3.0) and 5-year survival 28% (95% CI, 20-36%). The presence of progressive bulbar palsy (PBP), either at presentation or developing during the course of the illness, significantly reduced survival and was the most important prognostic indicator. Patients who survived longer than 5 years from symptom onset did not have PBP as part of their presenting illness. The prognosis was worse for women, and this was in part related to the higher frequency of PBP in older women, but age was also an independent adverse risk factor. Differences in survival between this and previous series can probably be explained on the basis of variation in case definition and ascertainment methods.Journal of Neurology 07/1993; 240(6):339-46. · 3.58 Impact Factor