Systematic literature review of evidence in amyloid light-chain amyloidosis

J Comp Eff Res. 2022 Apr;11(6):451-472. doi: 10.2217/cer-2021-0261. Epub 2022 Feb 21.

Abstract

Introduction: Treatment of amyloid light-chain (AL) amyloidosis, a rare disease with a <5-year lifespan, remains challenging. This systematic literature review (SLR) aimed to evaluate the current evidence base in AL amyloidosis. Methods: Literature searches on clinical, health-related quality of life, economic and resource use evidence were conducted using the Embase, MEDLINE and Cochrane databases as well as gray literature. Results: This SLR yielded 84 unique studies from: five randomized controlled trials; 54 observational studies; 12 health-related quality of life studies, none with utility values; no economic evaluation studies; and 16 resource use studies, none with indirect costs. Conclusion: This SLR highlights a paucity of published literature relating to randomized controlled trials, utility values, economic evaluations and indirect costs in AL amyloidosis.

Keywords: AL amyloidosis; clinical trials; cost; health-related quality of life; resource use; systematic review.

Publication types

  • Research Support, Non-U.S. Gov't
  • Review
  • Systematic Review

MeSH terms

  • Cost-Benefit Analysis
  • Databases, Factual
  • Humans
  • Immunoglobulin Light-chain Amyloidosis* / therapy
  • Publications
  • Quality of Life