How is the response to therapy defined for amyloidosis?

Updated: May 09, 2019
  • Author: Robert O Holmes, Jr, DO; Chief Editor: Herbert S Diamond, MD  more...
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While amyloidosis is a very heterogeneous disease, there has been a focused effort to define what a meaningful response to therapy looks like. Each affected organ can be measured over time.

In cardiac amyloidosis, improvement is defined as a 2-mm decrease in mean interventricular septal thickness, 20% improvement in ejection fraction, improvement by 2 New York Heart Association (NYHA) classes without an increase in diuretic use, and no increase in wall thickness, and/or a reduction (≥30% and ≥300 ng/L) of N-terminal pro b-type natriuretic peptide (NT-proBNP) in patients in whom the estimated glomerular filtration rate (eGFR) is ≥45 mL/min/1.73 m2. [110]

For renal-related amyloidosis, a meaningful clinic response is defined as "50% decrease (at least 0.5 g/day) of 24-hr urine protein (urine protein must be >0.5 g/day pre-treatment) in the absence of a reduction in eGFR ≥25% or an increase in serum creatinine ≥ 0.5 mg/dL". [110]

For liver-related amyloid, a decrease in the size of the liver by 2 cm radiographically and a 50% drop in the alkaline phosphatase level is a meaningful clinical response goal. [110]

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