Disease burden of systemic light-chain amyloidosis: a systematic literature review. Curr Med Res Opin 2017 Jun;33(6):1017-1031
Date
03/10/2017Pubmed ID
28277869DOI
10.1080/03007995.2017.1297930Scopus ID
2-s2.0-85019629331 (requires institutional sign-in at Scopus site) 21 CitationsAbstract
INTRODUCTION: A systematic literature review on systemic light chain (AL) amyloidosis was conducted in order to understand the disease burden, and identify unmet medical needs and knowledge gaps.
METHODS: MEDLINE, Embase and Cochrane databases were searched for English language studies published in the last 10 years using search terms that focused on the clinical, economic, and patient-reported outcome (PRO) aspects of AL amyloidosis. There was a low yield of articles in the economic and PRO categories and additional searches were conducted in clinical conference proceedings, and using Google and Google Scholar. After review, there were 65 articles included for data extraction.
RESULTS: AL amyloidosis is a rare disorder without any FDA or EMA approved indications for drug therapy. Using off-label therapies, there is a high rate, 42-64%, of non-response or progression, and an associated high mortality. Toxicities during therapy are common with estimates of up to 30-40% of patients experiencing severity of grade 3 or higher. Patients with AL amyloidosis report severe psychological distress, anxiety and clinical depression.
CONCLUSIONS: There is a deficiency in the literature on the economic costs associated with AL amyloidosis, and information on costs has been derived from studies that examined multiple myeloma or other disease or treatment components common to AL amyloidosis.
Author List
Lin HM, Gao X, Cooke CE, Berg D, Labotka R, Faller DV, Seal B, Hari PAuthor
Parameswaran Hari MD Adjunct Professor in the Medicine department at Medical College of WisconsinMESH terms used to index this publication - Major topics in bold
AmyloidosisCost of Illness
Costs and Cost Analysis
Humans
Immunoglobulin Light-chain Amyloidosis
Multiple Myeloma