Disease burden of systemic light-chain amyloidosis: a systematic literature review

Abstract

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.

Keywords:

• Drug therapy
• health economics
• patient-reported outcomes
• stem cell transplant
• unmet medical needs

Transparency

Declaration of funding

This study was sponsored by Millennium Pharmaceuticals Inc., a wholly owned subsidiary of Takeda Pharmaceutical Company Limited, Cambridge, MA, USA. X.G., C.E.C. are consultant employees of Pharmerit International. D.B., R.L., and D.V.F. are employees of Millennium Pharmaceuticals, Inc.

Declaration of financial/other relationships

P.H. has disclosed that he has acted as a consultant/advisor to Takeda. H.M.L. has disclosed that he is an employee and shareholder of Millennium Pharmaceuticals Inc., a wholly owned subsidiary of Takeda Pharmaceutical Company Limited. B.S. has disclosed that he is an employee of and shareholder in Takeda.

CMRO peer reviewers on this manuscript have no relevant financial or other relationships to disclose.

Notes

1 Kyprolis is a registered trade name of Amgen Inc, Thousand Oaks, CA.