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Estimating the global prevalence of transthyretin familial amyloid polyneuropathy
Introduction
This study sought to estimate the global prevalence of transthyretin familial amyloid polyneuropathy (ATTR-FAP).
Methods
Prevalence estimates and information supporting prevalence calculations was extracted from records yielded by reference-database searches (2005-2016), conference proceedings, and nonpeer reviewed sources. Prevalence was calculated as prevalence rate multiplied by general population size, then extrapolated to countries without prevalence estimates but with reported cases.
Results
Searches returned 3,006 records; 1,001 were fully assessed and 10 retained, yielding prevalence for 10 “core” countries, then extrapolated to 32 additional countries. ATTR-FAP prevalence in core countries, extrapolated countries, and globally was 3,762 (range 3639-3884), 6424 (range, 1,887-34,584), and 10,186 (range, 5,526-38,468) persons, respectively.
Discussion
The mid global prevalence estimate (10,186) approximates the maximum commonly accepted estimate (5,000-10,000). The upper limit (38,468) implies potentially higher prevalence. These estimates should be interpreted carefully because contributing evidence was heterogeneous and carried an overall moderate risk of bias. This highlights the requirement for increasing rare-disease epidemiological assessment and clinician awareness. Muscle Nerve, 2018.
Authors
H H Schmidt, M Waddington-Cruz, M F Botteman, J A Carter, A S Chopra, M M Hopps, M Stewart, S Fallet, L Amass
Journal
Muscle & Nerve
Therapeutic Area
Neurology
Center of Excellence
Health Economic Modeling & Meta-analysis
Year
2017
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