"Diagnosed with cardiac amyloidosis" -To your good health - Dr Keith Roach

DEAR DR. ROACH: My husband has been diagnosed with cardiac amyloidosis. The doctor has started him on Vyndamax, a very expensive drug. Without this drug, he is lucky to live, at the most, two years. Is this drug a miracle, and does it guarantee that he will live many more years? — T.W.
ANSWER: There are several types of cardiac amyloidosis — a disease in which abnormal proteins are depos-ited in many tissues, including the heart, where the protein interferes with heart function and leads to heart failure.  Tafadamis (also known by its brand name, Vyndamax) is used in transthyretin amyloid cardiomyopa-thy (ATTR, of which there are two types that both get treated with tafadamis).
Subjects in the trial on tafadamis were followed for two-and-a-half years. Half of the group were given tafadamis, while the other half were given a placebo, which looked like the real drug but had no activity. Of those who got the inactive placebo, 43% died in the two-and-a-half-year time span of the study, but of those who received tafadamis, 30% died. Thirteen percent, or about one in eight people, who received tafadamis lived, when they were expected to die. In addition, there were fewer hospitalizations and less loss of ability to exercise in those taking tafadamis, com-pared with the placebo. There were no serious side effects that were more frequent in the tafadamis group.
Tafadamis definitely affords a big improvement in the outcome of people with ATTR-CA. However, I would not call the drug a “miracle,” nor is there a guarantee of living many more years. To me, a miracle is when someone recovers when no doctor expected them to. I have seen a few in my career, and they are very powerful. I have seen many treatments that have incremental improvements in outcomes, and over time, that has led to dramatic improve-ments in many diseases.
Dr. Roach regrets that he is unable to answer individual questions, but will incorporate them in the column whenever possible. Readers may email questions to ToYourGoodHealth@ med.cornell.edu.
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