Transplantation vs. conventional-dose therapy for amyloidosis

Abstract

Purpose of review: Multiorgan involvement renders patients with AL amyloidosis particularly susceptible to treatment toxicity. The introduction of autologous stem cell transplantation (ASCT) represented a major advancement, but was associated with unacceptable toxicity in high-risk patients. Thus, efforts were made to improve the eligibility criteria for ASCT and to design novel, more effective, conventional-dose regimens. This review focuses on the role of ASCT and conventional-dose therapy in light of advances in risk stratification and patient monitoring.

Recent findings: The possibility of directly measuring the amyloidogenic precursor, the circulating free light chain (FLC), improved monitoring response to therapy. Cardiac biomarkers, N-terminal pro-natriuretic peptide type-B (NT-proBNP) and troponins (cTn) allow the most accurate prognostic stratification and direct the choice of therapy. Serial measurement of NT-proBNP, cTn and FLC are used to rapidly assess treatment efficacy. Bortezomib and immune-modulatory drugs are going to play a major role in conventional-dose therapy and as adjuvant treatment after ASCT.

Summary: The choice between ASCT and conventional-dose chemotherapy is based on accurate risk assessment. Tight monitoring of hematologic and cardiac response is the cornerstone of treatment. Upcoming randomized trials will redefine the role of available therapies, assisting in the choice of the growing number of active regimens.