The response time for amyloidosis tends to occur well after transplant, so the “best response” to transplant may not occur within the first 100 days. The intent of these questions is to determine the best overall response to HCT or cellular therapy, which could include any response to planned therapy post-HCT or post-cellular therapy, or to therapy given for maintenance or prophylaxis. DO NOT include any response to treatment given for relapsed or progressive disease. This is assessed in each reporting period. When evaluating the best response, determine the disease status within the reporting period and compare it to all previous post-HCT or post-cellular therapy reporting periods. If the response in the current reporting period is the best response to date, report the disease status established within this reporting period. If a better response was established in a previous reporting period, report the previously established disease status.

Question 54: Specify the recipient’s best cardiac response to the HCT:

Indicate the recipient’s best cardiac response to HCT to date. See Amyloidosis Response Criteria for disease status definitions.

If the recipient’s cardiac status was not assessed during the reporting period, select “not assessed,” and continue with question 88. If the recipient never had evidence of cardiac involvement in their disease, select “not applicable,” and continue with question 88.

Questions 55-56: Date assessed:

Indicate if the date the best cardiac response to transplant was assessed is “known,” “unknown,” or “previously reported.” If the cardiac response is known, report the date in question 56. If the date is unknown, select “unknown” and continue with question 57. If the best response to transplant was already reported in a previous reporting period, select “previously reported” and continue with question 88.

Question 57: Was the left ventricular ejection fraction measured?

The left ventricular ejection fraction (LVEF) is a percentage that represents the volume of blood pumped from the left ventricle into the aorta (also known as stroke volume) compared to the volume of blood in the ventricle just prior to the heart contraction (also known as end diastolic volume). Indicate if the left ventricular ejection fraction (LVEF) was measured. If “yes,” continue with question 58. If “no,” continue with question 60.

Question 58: Specify the left ventricular ejection fraction:

Indicate the left ventricular ejection fraction at the time of best response. Most imaging reports will report the LVEF. If the LVEF is not explicitly documented it should be determined by dividing the stroke volume (SV, the volume of blood pumped into the aorta from the left ventricle) by the end diastolic volume (EDV, the volume of blood in the left ventricle just prior to contraction) of the left ventricle . For example, if the stroke volume was 75 ml and the end diastolic volume was 150ml, the ejection fraction would be 50%.

Question 59 : Specify the method used to determine the left ventricular ejection fraction:

Indicate the method used to determine the LVEF value.

Question 60: Was diastolic dysfunction present?

Diastole is the period in which chambers of the heart fill with blood. Diastolic dysfunction may be characterized by the difficulty of the ventricles to expand and contract appropriately due to stiffening of the heart walls by amyloid deposits. Indicate if diastolic dysfunction was present. Specify “yes,” “no,” or “unknown,” and continue with question 61.

Questions 61-62: Specify the intraventricular septal wall thickness measured by echocardiogram:

The heart is divided into the right and left sides by the septum. The area between the left and right ventricles is the intraventricular septum. Indicate if the intraventricular septal thickness was measured at the time of best response. If “yes,” report the value as documented on the echocardiogram in question 62. If “no,” or not measured by echocardiogram, continue with question 63.

Questions 63-64: Specify left ventricular (LV) strain percentage:

A strain pattern, as determined by electrocardiography, is a well-recognized marker of hypertrophy of the left ventricular (LVH) and is characterized by ST depression and T wave inversion on a resting ECG / EKG. The LV strain percentage is typically a negative percentage. The normal range for the LV global longitudinal strain (LV GLS) is -15.9% to -22.1%. Indicate if the left ventricular strain percentage was measured at the time of best response. If “yes,” based on evaluation by electrocardiogram, indicate the strain percentage in question 64. If “no,” or not measured by electrocardiogram, continue with question 65.

Questions 65-66: Were any serum cardiac biomarkers assessed?

Assessment of cardiac biomarkers helps determine if injury to cardiac tissue has occurred. Cardiac biomarkers include brain natriuretic peptide (BNP), N-terminal prohormone brain natriuretic peptide (NT-proBNP), troponin I, troponin T, and high-sensitivity troponin T. Indicate if serum cardiac biomarkers were assessed at the time of best response. If “yes,” report the date assessed in question 66 and continue with question 67. If “no” or “unknown,” continue with question 82.

Questions 67-68: Brain natriuretic peptide (BNP):

Indicate if the BNP was assessed at the time of best response. If “yes,” report the value (in pg/mL) in question 68 and continue with question 69. If “no,” continue with question 70.

Question 69: Upper limit of normal for BNP:

Indicate the upper limit of normal for BNP (in pg/mL) found on the laboratory report.

Questions 70-71: N-terminal prohormone brain natriuretic peptide (NT-proBNP):

Indicate if the NT-proBNP was assessed at the time of best response. If “yes,” report the value (in pg/mL) in question 71 and continue with question 72. If “no,” continue with question 73.

Question 72: Upper limit of normal for NT-proBNP:

Indicate the upper limit of normal (in pg/mL) for NT-proBNP found on the laboratory report.

Questions 73-74: Troponin I:

Indicate if the Troponin I was assessed at the time of best response. If “yes,” report the value (in µg/L) in question 74 and continue with question 75. If “no,” continue with question 76.

Question 75: Upper limit of normal for troponin I:

Indicate the upper limit of normal (in µg/L) for Troponin I found on the laboratory report.

Questions 76-77: Troponin T:

Indicate if the Troponin T was assessed at the time of best response. If “yes,” report the value (in µg/L) in question 77 and continue with question 78. If “no,” continue with question 79.

Question 78: Upper limit of normal for Troponin T:

Indicate the upper limit of normal (in µg/L) for Troponin T found on the laboratory report.

Questions 79-80: High-sensitivity troponin T:

Indicate if the high-sensitivity troponin T was assessed at the time of best response. If “yes,” report the value (in ng/L) in question 80 and continue with question 81. If “no,” continue with question 82.

Question 81: Upper limit of normal for high-sensitivity troponin T:

Indicate the upper limit of normal (in ng/L) for high-sensitivity troponin T found on the laboratory report.

Questions 82-83: Was a 6-minute walk test performed?

A 6-minute walk test is used to assess total distance walked within 6 minutes to determine aerobic capacity and endurance. Indicate if a 6-minute walk test was performed at the time of best response. If “yes,” report the total distance walked and specify the unit of measure in question 83. If “no,” continue with question 84.

Question 84: Specify the recipient’s New York Heart Association functional classification of heart failure: (Symptoms may include dyspnea, chest pain, fatigue, and palpitations; activity level should be assessed with consideration for patient’s age group)

Indicate the recipient’s New York Heart Association functional classification at the time of best response using the following guidelines:

  • Class I – Able to perform ordinary activities without symptoms; no limitation of physical activity
  • Class II – Ordinary physical activity produces symptoms; slight limitation of physical activity
  • Class III – Less-than-ordinary physical activity produces symptoms; moderate limitation of physical activity
  • Class IV – Symptoms present even at rest; severe limitation of physical activity

If the recipient’s NYHA functional classification it not known, select “unknown.”

Question 85: Recipient blood pressure:

Indicate if the recipient’s blood pressure was assessed at the time of best response. If “known,” continue with question 86. If “unknown,” continue with question 88.

Questions 86-87: Recipient blood pressure results:

Report the recipient’s blood pressure at the time of best response in question 86 and indicate in which body position the measurement was taken in question 87.

Question 88: Specify the recipient’s best renal response:

Indicate the recipient’s best renal response to HCT to date. See the Amyloidosis Response Criteria section for disease status definitions.

If the recipient’s renal status was not assessed during the reporting period, select “not assessed.” If the recipient never had evidence of renal involvement in their disease, select “not applicable.”

Questions 89-90: Date assessed:

Indicate if the date the best renal response to transplant was assessed is “known,” “unknown,” or “previously reported.” If the renal response is known, report the date in question 90. If the date is unknown, select “unknown” and continue with question 91. If the best response to transplant was already reported in a previous reporting period, select “previously reported” and continue with question 91.

Question 91: Specify the recipient’s best hepatic response to the HCT:

Indicate the recipient’s best hepatic response to HCT to date. See Amyloidosis Response Criteria for disease status definitions.

If the recipient’s hepatic status was not assessed during the reporting period, select “not assessed,” and continue with question 98. If the recipient never had evidence of hepatic involvement in their disease, select “not applicable,” and continue with question 98.

Questions 92-93: Date assessed:

Indicate if the date the best hepatic response to transplant was assessed is “known,” “unknown,” or “previously reported.” If the hepatic response is known, report the date in question 93. If the date is unknown, select “unknown” and continue with question 94. If the best response to transplant was already reported in a previous reporting period, select “previously reported” and continue with question 94.

Question 94: Was hepatomegaly present on radiographic imaging (liver span > 15 cm) or on examination (liver edge palpable > 3 cm below right costal margin)?

At the time of best response, indicate if the liver spanned more than 15 cm (by radiographic imaging) or the edge of the liver was palpable more than 3 cm below the right costal margin (by physical examination). Indicate “yes” if hepatomegaly was present at the time of best response. Indicate “no” if hepatomegaly was not present at the time of best response. Indicate “unknown” if it was not possible to determine the presence or absence of hepatomegaly at the time of best response.

Questions 95-96: Specify the level of serum alkaline phosphatase:

Indicate whether the alkaline phosphatase (ALP) level at the time of best response is “known” or “unknown.” If “known,” report the laboratory count and unit of measure documented on the laboratory report in question 96 and continue with question 97. If “unknown,” continue with question 98.

Question 97: Upper limit of normal for alkaline phosphatase:

Report the upper limit of normal for ALP from the laboratory report.

Question 98: Was there clinical improvement in GI involvement since the date of last report?

Indicate if there was clinical improvement of GI involvement to date. Judgment is required by a clinician to determine if there is evidence of improvement. If “yes” or “no,” continue with question 99. If “unknown” or “not applicable,” continue with question 101. Report “not applicable” if the recipient never had evidence of GI involvement in their disease.

Questions 99-100: Date assessed:

Indicate if the date the GI involvement was assessed is “known,” “unknown,” or “previously reported.” If the date the GI response was assessed is “known,” report the date in question 100. If the date is unknown, select “unknown” and continue with question 101. If the best response to transplant was already reported in a previous reporting period, select “previously reported” and continue with question 101.

Question 101: Specify the recipient’s best peripheral nervous system response:

Indicate the recipient’s best peripheral nervous system response to HCT to date. See Amyloidosis Response Criteria for disease status definitions.

If the recipient’s peripheral neuropathy was not assessed during the reporting period, select “not assessed” and continue with question 104. If the recipient never had evidence of disease-related peripheral neuropathy, select “not applicable” and continue with question 104.

Questions 102-103: Date assessed:

Indicate if the date the best peripheral neuropathy response to transplant was assessed is “known,” “unknown,” or “previously reported.” If the peripheral neuropathy response is “known,” report the date in question 103. If the date is unknown, select “unknown” and continue with question 104. If the best response to transplant was already reported in a previous reporting period, select “previously reported” and continue with question 104.

Question 104: Did the recipient display any other clinical organ involvement?

Indicate if any other system was assessed for response to HCT. If the recipient had other site involvement reported in questions 116-118 of the Pre-HCT Plasma Cell Disorder form (Form 2016) and that site was assessed, the response to HCT must be reported here, even if there was no response. If any other system was assessed, report “yes” and continue with question 105. If no other systems were assessed at the time of best response, report “no” and continue with question 110.

Questions 105-106: Specify the evidence of other organ involvement (check all that apply):

For each option, indicate if there was evidence of other organ involvement. Check all that apply. If there was other organ involvement not listed in this section, select “other organ involvement” in question 105 and specify the other organ in question 106.

Examples may include:

  • Arthropathy is a disease of the joints. An example of a common arthropathy in patients with amyloidosis is carpal tunnel-like symptoms.
  • Amyloid deposits may be found in the lung, impairing their function. Examples of lung involvement may be alveolar-septal disease, nodular disease, intra- and extra-thoracic adenopathy, pleural disease, and diaphragm deposition.
  • Soft tissue involvement, other than those already listed, may include glandular involvement (such as submandibular glands).
  • Any additional organ involvement, other than those already listed, may be reported in this section as “other organ involvement.” The other organ involved will then be specified in question 106.

5 Berk JL, O’Regan A, Skinner M. Pulmonary and tracheobronchial amyloidosis. Semin Respir Crit Care Med. 2002;23(2):155-65.

Question 107: Specify best response to HCT or cellular therapy for this system:

Indicate if the site’s/system’s best response to transplant was “improved response,” “progression,” or “no response/stable disease.”

Questions 108-109: Date assessed:

Indicate if the date the other site’s/system’s best response to transplant was assessed is “known,” “unknown,” or “previously reported.” If the other site’s/system’s response is “known,” report the date in question 109. If the date is unknown, select “unknown” and continue with question 110. If the best response to transplant was already reported in a previous reporting period, select “previously reported” and continue with question 110.

Last modified: Mar 27, 2020

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