An evaluation of current treatment options for immunoglobulin light-chain amyloidosis. (March 2014)

• Record Type: Journal Article
• Authors: Sachchithanantham, Sajitha; Wechalekar, Ashutosh D; Hawkins, Philip N
• Is Part Of: Expert opinion on orphan drugs. Volume 2:Number 3(2014:Mar.)
• Page Start: 229
• Page End: 244
• Publication Date: 2014-03
• Subjects: Orphan drugs -- Periodicals; Rare diseases -- Periodicals; Chemotherapy -- Periodicals; 615.1
• DOI: 10.1517/21678707.2014.881285
• Languages: English
• ISSNs: 2167-8707

Abstract

<abstract> <title> <x xml:space="preserve">Abstract</x> </title> <p> <bold> <italic>Introduction:</italic> </bold> The treatment of immunoglobulin light-chain (AL) amyloidosis parallels that of multiple myeloma. Particular challenges not present in myeloma relate to amyloid-related organ damage, which greatly increases treatment-related toxicity and mortality. The management of patients with advanced organ involvement remains a huge challenge even in the era of novel agents with improved supportive care. Further, the limited number of direct comparisons of treatment in AL amyloidosis has meant that current clinical practice stems from retrospective studies and case reports.</p> <p> <bold> <italic>Areas covered:</italic> </bold> Here, current treatment options and future potential therapies in the management of AL amyloidosis are reviewed. A literature review has been performed on PubMed database and abstracts from recent scientific meetings with the search terms: 'AL amyloidosis', 'melphalan', 'stem cell transplantation', 'proteasome inhibitors' and 'immunomodulatory drugs'.</p> <p> <bold> <italic>Expert opinion:</italic> </bold> Stem cell transplantation (SCT) should remain a preferred option for patients deemed eligible to undergo this technique safely. Unfortunately, only 20 – 25% of patients with AL amyloidosis are eligible for SCT with acceptably low mortality risk. Most patients, therefore, require cyclic multiple-agent chemotherapy, including immunomodulatory drugs, proteasome inhibitors and alkylators such as melphalan. Novel therapeutic approaches such as immunotherapy may soon be available with potential to reshape the treatment strategy of this serious disorder.</p> </abstract>