Systemic light chain amyloidosis: an update for treating physicians

Blood. 2013 Jun 27;121(26):5124-30. doi: 10.1182/blood-2013-01-453001. Epub 2013 May 13.

Abstract

In immunoglobulin light chain amyloidosis a small, indolent plasma cell clone synthesizes light chains that cause devastating organ damage. Early diagnosis, based on prompt recognition of "red-flags" before advanced cardiomyopathy ensues, is essential for improving outcomes. Differentiation from other systemic amyloidoses may require advanced technologies. Prognosis depends on the extent of cardiac involvement, and cardiac biomarkers guide the choice of therapy. The protean clinical presentation requires individualized treatment. Close monitoring of clonal and organ response guides therapy changes and duration. Conventional or high-dose alkylator-based chemotherapy is effective in almost two-thirds of patients. Combinations of proteasome inhibitors, dexamethasone, and alkylators achieve high response rates, although controlled studies are needed. Risk-adapted stem cell transplant and consolidation with novel agents may be considered in selected patients. Immune-modulatory drugs are good options for refractory/relapsed patients. Novel agents and therapeutic targets are expected to be exploited, in an integrated, more effective and less toxic treatment strategy.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Amyloidosis / diagnosis*
  • Amyloidosis / therapy*
  • Combined Modality Therapy
  • Humans
  • Immunoglobulin Light Chains*
  • Physicians*
  • Practice Guidelines as Topic*

Substances

  • Immunoglobulin Light Chains