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. 2022 Jul 19;17(1):278.
doi: 10.1186/s13023-022-02414-6.

Global epidemiology of amyloid light-chain amyloidosis

Nishant Kumar  1 , Nicole J Zhang  1 , Dasha Cherepanov  2 , Dorothy Romanus  3 , Michael Hughes  1 , Douglas V Faller  3
Affiliations

Global epidemiology of amyloid light-chain amyloidosis

Nishant Kumar et al. Orphanet J Rare Dis. .

Abstract

Background: Amyloid light-chain (AL) amyloidosis is an ultra-rare disease associated with significant morbidity and mortality. Few studies have examined the global epidemiology of this condition.

Methods: This study estimated the diagnosed incidence and 1-year, 5-year, 10-year, and 20-year period prevalence of AL amyloidosis in 2018 for countries in and near Europe, and in the United States (US), Canada, Brazil, Japan, South Korea, Taiwan, and Russia. A systematic literature review (SLR) was conducted to identify country-specific, age- and gender-specific diagnosed incidence of AL amyloidosis and observed survival data-point inputs for an incidence-to-prevalence model. Extrapolations were used to estimate incidence and prevalence for countries without registry or published epidemiological data.

Results: Of 171 publications identified in the SLR, 10 records met the criteria for data extraction, and two records were included in the final incidence-to-prevalence model. In 2018, an estimated 74,000 AL amyloidosis cases worldwide were diagnosed during the preceding 20 years. The estimated incidence and 20-year prevalence rates were 10 and 51 cases per million population, respectively.

Conclusions: Orphan medicinal product designation criteria of the European Medicines Agency or Electronic Code of Federal Regulations indicate that a disease must not affect > 5 in 10,000 people across the European Union or affect < 200,000 people in the US. This study provides up-to-date epidemiological patterns of AL amyloidosis, which is vital for understanding the burden of the disease, increasing awareness, and to further research and treatment options.

Keywords: Amyloid light-chain amyloidosis; Epidemiology; Global; Incidence; Prevalence; Rare disease.

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Conflict of interest statement

Nishant Kumar, Nicole Zhang, and Michael Hughes declare no conflict of interest. Dasha Cherepanov, Dorothy Romanus, and Douglas Faller are employees of Takeda Development Center Americas, Inc. and may own stock.

Figures

Fig. 1
Fig. 1
PRISMA diagram of identified and included records. PRISMA Preferred Reporting Items for Systematic Reviews and Meta-Analyses
Fig. 2
Fig. 2
Age-specific incidence of AL amyloidosis, by sex. AL amyloid light-chain
Fig. 3
Fig. 3
Estimating 5-year prevalent cases of AL amyloidosis in 2018. AL amyloid light-chain
Fig. 4
Fig. 4
Calculated crude incidence in 2018, by country. PMP per million population
Fig. 5
Fig. 5
Relative survival of AL amyloidosis, by year since diagnosis. AL amyloid light-chain
See this image and copyright information in PMC

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