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Table 2 Objectives and epidemiological measures obtained from the clinical ALS-registry and additional information derived from the attached casecontrol study

From: Implementation of a population-based epidemiological rare disease registry: study protocol of the amyotrophic lateral sclerosis (ALS) - registry Swabia

a. Clinical ALS registry (patient centered analyses)
Descriptive measures: -ALS prevalence (proportion)
-Symptoms at disease manifestation
-Distribution of localization at onset
-Time from first symptoms to clinical diagnosis (diagnostic delay)
-Distribution of diagnostic procedures in routine clinical care of ALS
-Application of therapeutic means and measures during follow-up
-Background rate of adverse events in ALS population
-Rate and risk factors of comorbid diseases in ALS population
-Natural history of disease (progression free survival, overall survival, case fatality rate)
-Quality of life
Analytical measures: -(Long-term) efficacy of therapies under routine clinical care conditions on various outcomes (progression free survival, overall survival) and occurrence of adverse events (safety outcomes)
-Identification of prognostic markers of disease progression (includes assessment of potential new drug targets
b. Epidemiological ALS Registry (includes geographical analyses)
Descriptive measures: -All of the above mentioned (see 2.a), additionally
  plus incidence
  plus mortality
  plus geographic and frequency distribution of risk factors
Analytical measures: -see 2.a
  plus spatial changes
  plus cluster analysis
-in nested case – control study
  plus investigation of risk factors and potential causes of disease
Molecular epidemiology: -in biobanked material
  plus investigation of pathogenetic pathways (blood samples)