New concepts in the treatment and diagnosis of amyloidosis

Paolo Milani, Giovanni Palladini, Giampaolo Merlini

Research output: Contribution to journalReview article

Abstract

Introduction: The most common form of systemic amyloidosis in Western countries is light chain amyloidosis. It is characterized by the deposition of a misfolded light chain in target organs. This amyloid precursor is produced by a usually small but dangerous B-cell clone. Areas covered: This review examines the diagnostic workup of this disease and current knowledge of biomarker-based staging systems. In addition, a risk-adapted treatment approach is presented, as well as an overview of the new treatment strategies. Expert commentary: The cornerstone of treatment is rapid and effective chemotherapy targeting the underlying plasma cell clone. In the near future, this will probably be associated with novel approaches targeting other steps of the amyloid cascade that result in amyloid deposits. Currently available effective treatments can alter its natural history if an early diagnosis is made. The availability of novel, more powerful drugs, and identification of the cellular mechanisms of organ damage and of the characteristics of the amyloidogenic plasma-cell clone all give grounds to hope that a dramatic improvement in the treatment of this disease will be seen in the near future.

Original languageEnglish
Pages (from-to)117-127
Number of pages11
JournalExpert Review of Hematology
Volume11
Issue number2
DOIs
Publication statusPublished - Feb 1 2018
Externally publishedYes

Keywords

  • Amyloidosis
  • diagnosis
  • prognosis
  • response
  • therapy

ASJC Scopus subject areas

  • Hematology

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