SveATTR Registry: Long-Term Real-World Data on Diflunisal for Hereditary Amyloidosis

Data from 118 patients in the longitudinal Swedish SveATTR registry through December 2022, with an estimated mean therapy duration of 3.4 years. The dataset includes clinical progression metrics and was analyzed by researchers using mixed effects modeling. It provides real-world evidence supporting the efficacy of diflunisal for hereditary transthyretin amyloidosis.

Use Cases

• Modeling disease progression over time based on trends in Kumamoto and PND scores mentioned in the description
• Assessing treatment efficacy in a real-world setting based on longitudinal registry data
• Conducting Bayesian meta-analyses by combining registry data with pivotal trial results
• Analyzing the relationship between different clinical metrics like mBMI and Karnofsky performance status

Strengths

• Includes data from 118 patients, providing a real-world cohort.
• Covers a long observation period with a mean therapy duration of 3.4 years (range 0.1 to 10.2 years).
• Analysis was combined with pivotal trial data for a Bayesian augmentation, showing high probabilities of treatment superiority.

Limitations

• Column-level documentation is absent; field semantics must be inferred after download.
• Row count is unknown, which may limit suitability assessment.
• The dataset is very small at 73.0 KB, indicating limited scope.

Provenance

Source

SveATTR, a longitudinal Swedish registry for patients with ATTR amyloidosis.

Collection Method

Data from diflunisal-treated patients registered in the SveATTR registry.

Time Range

Data included through December 2022.

Freshness

Last updated 2026-06-03 09:40:02; freshness should be verified

Geography

Sweden