Amyloidosis: Pathogenesis and new therapeutic options

Giampaolo Merlini, David C. Seldin, Morie A. Gertz

Research output: Contribution to journalArticle

292 Citations (Scopus)

Abstract

The systemic amyloidoses are a group of complex diseases caused by tissue deposition of misfolded proteins that results in progressive organ damage. The most common type, immunoglobulin light chain amyloidosis (AL), is caused by clonal plasma cells that produce misfolded light chains. The purpose of this review is to provide up-to-date information on diagnosis and treatment options for AL amyloidosis. Early, accurate diagnosis is the key to effective therapy, and unequivocal identification of the amyloidogenic protein may require advanced technologies and expertise. Prognosis is dominated by the extent of cardiac involvement, and cardiac staging directs the choice of therapy. Treatment for AL amyloidosis is highly individualized, determined on the basis of age, organ dysfunction, and regimen toxicities, and should be guided by biomarkers of hematologic and cardiac response. Alkylator-based chemotherapy is effective in almost two thirds of patients. Novel agents are also active, and trials are ongoing to establish their optimal use. Treatment algorithms will continue to be refined through controlled trials. Advances in basic research have led to the identification of new drug targets and therapeutic approaches, which will be integrated with chemotherapy in the future.

Original languageEnglish
Pages (from-to)1924-1933
Number of pages10
JournalJournal of Clinical Oncology
Volume29
Issue number14
DOIs
Publication statusPublished - May 10 2011

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Amyloidosis
Therapeutics
Immunoglobulin Light Chains
Amyloidogenic Proteins
Drug Therapy
Alkylating Agents
Plasma Cells
Early Diagnosis
Biomarkers
Technology
Light
Research
Pharmaceutical Preparations
Proteins

ASJC Scopus subject areas

  • Cancer Research
  • Oncology

Cite this

Amyloidosis : Pathogenesis and new therapeutic options. / Merlini, Giampaolo; Seldin, David C.; Gertz, Morie A.

In: Journal of Clinical Oncology, Vol. 29, No. 14, 10.05.2011, p. 1924-1933.

Research output: Contribution to journalArticle

Merlini, Giampaolo ; Seldin, David C. ; Gertz, Morie A. / Amyloidosis : Pathogenesis and new therapeutic options. In: Journal of Clinical Oncology. 2011 ; Vol. 29, No. 14. pp. 1924-1933.
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