Dataset Information

Incidence and geographical variation of amyotrophic lateral sclerosis (ALS) in Southern Germany--completeness of the ALS registry Swabia.

ABSTRACT: Objective of this paper was to investigate the incidence, potential geographical clusters and the completeness of the amyotrophic lateral sclerosis (ALS) registry in Southern Germany (Swabia). Age-standardized incidence rates (ASR) and ratios (SIR) as well as 95% confidence intervals (CI) were estimated at county level. Capture-recapture (CARE) procedures were applied taking data source dependency into account to estimate the quality of case ascertainment in the ALS registry Swabia. We identified 438 ALS cases (53% men, 47% women) in the target population of about 8.4 Mio inhabitants. The gender ratio (men?women) was 1.1?1. The mean age at onset of ALS was 63.8 (SD?=?11.9) years for men and 66.0 (12.2) for women. The age distribution peaked in the age group 70-74 years. The ASR of ALS was 2.5 per 100,000 person years (PY; 95% CI: 2.3-2.7). The mean SIR was 1.1 per 100,000 PY (95% CI: 1.0-1.2). High SIR suggesting geographical clusters were observed in two counties (Göppingen and Bodenseekreis), but the variation was not statistically significant (p-values?=?0.2 and 0.5). The percentage of CARE estimated missing cases was 18.9% in the registry yielding an ASR of 3.1 per 100,000 PY. The high coverage of the CARE estimated completeness of the ALS registry Swabia indicates excellent quality for future projects. Regional variations have to be investigated further.

SUBMITTER: Uenal H

PROVIDER: S-EPMC3983245 | biostudies-literature | 2014

REPOSITORIES: biostudies-literature

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Incidence and geographical variation of amyotrophic lateral sclerosis (ALS) in Southern Germany--completeness of the ALS registry Swabia.

Uenal Hatice H Rosenbohm Angela A Kufeldt Johannes J Weydt Patrick P Goder Katharina K Ludolph Albert A Rothenbacher Dietrich D Nagel Gabriele G

PloS one 20140410 4

Objective of this paper was to investigate the incidence, potential geographical clusters and the completeness of the amyotrophic lateral sclerosis (ALS) registry in Southern Germany (Swabia). Age-standardized incidence rates (ASR) and ratios (SIR) as well as 95% confidence intervals (CI) were estimated at county level. Capture-recapture (CARE) procedures were applied taking data source dependency into account to estimate the quality of case ascertainment in the ALS registry Swabia. We identifie ...[more]

PMID: 24722455

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Project description:Thoughtful clinical trial design is critical for efficient therapeutic development, particularly in the field of amyotrophic lateral sclerosis (ALS), where trials often aim to detect modest treatment effects among a population with heterogeneous disease progression. Appropriate outcome measure selection is necessary for trials to provide decisive and informative results. Investigators must consider the outcome measure's reliability, responsiveness to detect change when change has actually occurred, clinical relevance, and psychometric performance. ALS clinical trials can also be performed more efficiently by utilizing statistical enrichment techniques. Innovations in ALS prediction models allow for selection of participants with less heterogeneity in disease progression rates without requiring a lead-in period, or participants can be stratified according to predicted progression. Statistical enrichment can reduce the needed sample size and improve study power, but investigators must find a balance between optimizing statistical efficiency and retaining generalizability of study findings to the broader ALS population. Additional progress is still needed for biomarker development and validation to confirm target engagement in ALS treatment trials. Selection of an appropriate biofluid biomarker depends on the treatment mechanism of interest, and biomarker studies should be incorporated into early phase trials. Inclusion of patients with ALS as advisors and advocates can strengthen clinical trial design and study retention, but more engagement efforts are needed to improve diversity and equity in ALS research studies. Another challenge for ALS therapeutic development is identifying ways to respect patient autonomy and improve access to experimental treatment, something that is strongly desired by many patients with ALS and ALS advocacy organizations. Expanded access programs that run concurrently to well-designed and adequately powered randomized controlled trials may provide an opportunity to broaden access to promising therapeutics without compromising scientific integrity or rushing regulatory approval of therapies without adequate proof of efficacy.

Dataset Information

Incidence and geographical variation of amyotrophic lateral sclerosis (ALS) in Southern Germany--completeness of the ALS registry Swabia.

Publications

Incidence and geographical variation of amyotrophic lateral sclerosis (ALS) in Southern Germany--completeness of the ALS registry Swabia.

Similar Datasets

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