Amyloidosis Response Criteria

*Consecutive Assessment Requirements
The Hematologic Response requires two consecutive assessments made by the same method at any time before the institution of any new therapy. Organ responses (i.e., Hepatic, Peripheral and Autonomic Neuropathy, Cardiac, Renal) do not require confirmatory assessments.

Hematologic Response

Complete Response

Requires all of the following:

• Serum and urine negative for monoclonal proteins by immunofixation
• Normal serum free light chain ratio
  

Very Good Partial Response

• Reduction in the difference between the involved and uninvolved serum free light chain to < 40 mg/L.

Partial Response (PR)

Requires any of the following:

• ≥ 50% reduction in serum monoclonal protein levels (if > 0.5 g / dL or > 5 g / L at baseline)
• ≥ 50% reduction in urine light chains (if >100 mg / day at baseline)
• ≥ 50% decrease in difference between the involved and uninvolved serum free light chain (if >10 mg / dL or >100 mg / L at baseline)
  

No Response (NR)/Stable Disease (SD)

Does not meet the criteria for CR, PR, or progressive disease.

Progressive Disease (PD)

Requires any of the following:

• If progressing from CR, any detectable monoclonal protein or abnormal free light chain ratio (light chain must double)
• If progressive from PR or SD, ≥ 50% increase in the serum M protein to > 0.5 g/dL, or ≥ 50% increase in urine M protein to > 200mg/day with visible peak present.
• Serum free light chain increase of ≥ 50% to > 10 mg/dL (100 mg/L)
  

Cardiac Response

Cardiac Response

Requires any of the following:

• > 30% and > 300 ng /l decrease in recipients with baseline N-terminal prohormone of brain natriuretic peptide (NT-proBNP) ≥ 650 ng/L or
• ≥ 2 class decrease in recipients with baseline New York Heart Association functional class class of 3 or 4
  

No Response/Stable Disease

Does not meet the criteria for cardiac response or progressive disease

Progressive Disease

Requires any of the following:

• > 30% and > 300 ng/l increase in N-terminal prohormone of brain natriuretic peptide (NT-proBNP)¹ or
• ≥ 33% increase in cardiac troponin (cTn) or
• ≥ 10% decrease in ejection fraction
  

¹ Recipients with progressively worsening renal function cannot be score for NT-proBNP progression. Progressively worsening renal function is defined as 50% increase (at least 1 g / day) of 24-hour urine protein from baseline to > 1 g / day or 25% worsening of serum creatinine or creatinine clearance from baseline

Hepatic Response

Hepatic Response

Requires all of the following:

• ≥ 2 cm decrease in liver span if hepatomegaly present (liver > 15 cm)
• ≥ 50% decrease and/or normalization of serum alkaline phosphatase (ALP) level
  

No Response/Stable Disease

Does not meet the criteria for hepatic response or progressive disease

Progressive Disease

Requires the following:

• ≥ 50% increase in the serum alkaline phosphatase (ALP) level
  

Autonomic Neuropathy Response

Autonomic Neuropathy Response

Resolution of symptomatic orthostatic hypotension

No Response/Stable Disease

Does not meet the criteria for autonomic neuropathy response or progressive disease

Progressive Disease

Worsening of symptomatic orthostatic hypotension

Peripheral Neuropathy Response

Peripheral Neuropathy Response

Requires any of the following:

• Resolution of abnormal physical findings
• Resolution or improvement of abnormal electromyography (EMG) and/or Nerve Conduction Velocity (NCV) findings
  

No Response/Stable Disease

Does not meet the criteria for peripheral neuropathy response or progressive disease

Progressive Disease

Requires any of the following:

• Worsening of physical findings
• Worsening of EMG and/or NCV findings
  

Renal Response

Renal Response

• ≥ 50% decrease of at least 0.5 g/day (500mg/24hr) in 24-hour urine protein of > 0.5 g/day (500mg/24hr) pre-treatment and
• Creatinine clearance or serum creatinine must not have worsened by ≥ 25% over baseline

If only serum creatinine is obtained, an estimated creatinine clearance can be calculated using the following formula:

Estimated Creatinine Clearance = [(140 – Age (years)) * Weight (kg)] / [72 * Serum Creatinine (mg/dL)]
The calculation should be multiplied by 0.85 for women

No Response/Stable Disease

Does not meet the criteria for renal response or progressive disease

Progressive Disease

Requires any of the following:

• ≥ 50% increase of at least 1 g/day (1000mg/24hr) for urine protein to > 1g/day (1000mg/24hr)
• 25% worsening of serum creatinine or creatinine clearance
  

Manual Updates:
Sections of the Forms Instruction Manual are frequently updated. The most recent updates to the manual can be found below. For additional information, select the manual section and review the updated text.

If you need to reference the historical Manual Change History for this form, please click here or reference the retired manual section on the Retired Forms Manuals webpage.

Date	Manual Section	Add/Remove/Modify	Description
5/18/22	Amyloidosis Response Criteria	Add	The Consecutive Assessment Requirements blue instruction box added: The Hematologic Response requires two consecutive assessments made by the same method at any time before the institution of any new therapy. Organ responses (i.e., Hepatic, Peripheral Neuropathy, Cardiac) do not require confirmatory assessments.
5/18/22	Amyloidosis Response Criteria	Modify	The Partial Response criteria for Hematologic Response was updated to be consistent with the Working Group Response criteria: ≥ 50% decrease in clonal serum free light chain levels difference between the involved and uninvolved serum free light chain (if >10 mg / dL or >100 mg / L at baseline)
5/18/22	Amyloidosis Response Criteria	Modify	Cardiac response criteria update to be consistent with Working Group Response criteria. Cardiac response: ≥ 2 mm decrease in mean intraventricular septal wall thickness by echocardiogram, ≥ 20% increase in left ventricular ejection fraction, ≥ 2 grade decrease in New York Heart Association functional class without increase in diuretic use and no increase in wall thickness, Reduction (≥ 30% and ≥ 300ng/L) of NT-proBNP in patients whom the eGFR is ≥ 45 ml/minute/1.73m2 > 30 % and > 300 ng /l decrease in recipients with baseline N-terminal prohormone of brain natriuretic peptide (NT-proBNP) ≥ 650 ng/L or ≥ 2 class decrease in recipients with baseline New York Heart Association class of 3 or 4 Progressive disease: ≥ 2 mm increase from baseline in the intraventricular wall thickness by echocardiogram, ≥ 10% decrease in the left ventricular ejection fraction, ≥ 1 grade increase in New York Heart Association functional class > 30 % and > 300 ng/l increase in N-terminal prohormone of brain natriuretic peptide (NT-proBNP) or ≥ 33 increase in cardiac troponin (cTn) or ≥ 10% decrease in ejection fraction%
5/26/21	Amyloidosis Response Criteria	Modify	The Partial Response criteria for Hematologic Response was updated to be consistent with the amyloid response criteria: * ≥ 50% reduction in current serum monoclonal protein levels (if > 0.5 g / dL or > 5 g / L at baseline) > 0.5 g/dL * ≥ 50 reduction in urine light chains (if >100 mg / day at baseline) ≥ 50% reduction in current urine m-protein levels > 100 mg/day with a visible peak * ≥ 50 decrease in clonal serum free light chain levels (if >10 mg / dL or >100 mg / L at baseline) ≥ 50% reduction in current free light chain levels > 10mg/dL
12/22/20	Amyloidosis Response Criteria	Add	Clarified in the Hematologic Response criteria that free light chain ratios are for the serum .
8/4/2020	Amyloidosis Response Criteria	Remove	Removed the following (struck out below) criteria from the Hematologic Complete Response Criteria: Requires all of the following: Serum and urine negative for monoclonal proteins by immunofixation Normal free light chain ratio Plasma cells in marrow < 5%
4/6/2020	Amyloidosis Response Criteria	Add	Added guidance on Free Light Chain Ratios.
4/19/19	Amyloidosis Response Criteria	Modify	Modified the following sections (removed text is struck out below, added text is in red) of the Amyloidosis Response Criteria: Partial Response: ≥ 50% reduction in current serum monoclonal protein levels > 0.5 g/dL ≥ 50% reduction in current urine light chain urine m-protein levels > 100 mg/day with a visible peak ≥ 50% reduction in current free light chain levels > 10mg/dL Renal Response: ≥ 50% decrease of at least 0.5 g/day (500mg/24hr) in 24-hour urine protein of > 0.5 g/day (500mg/24hr) pre-treatment and Creatinine clearance or serum creatinine must not have worsened by ≥ 25% over baseline If only serum creatinine is obtained, an estimated creatinine clearance can be calculated using the following formula: Estimated Creatinine Clearance = [(140 – Age (years)) * Weight (kg)] / [72 * Serum Creatinine (mg/dL)] The calculation should be multiplied by 0.85 for women.
12/3/18	Amyloidosis Response Criteria	Add	Added further clarification for the Renal Response criteria (indicated in red below): ≥ 50% decrease of at least 0.5 g/day (500mg/24hr) in 24-hour urine protein of > 0.5 g/day (500mg/24hr) pre-treatment and Creatinine clearance must not have worsened by ≥ 25% over baseline

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