Using dapagliflozin to reduce symptoms in transthyretin cardiac amyloidosis: An n-of-1 trial series pilot study.

What was studied

This pilot study tested dapagliflozin in transthyretin cardiac amyloidosis (ATTR-CM). The aim was to plan a larger randomized trial. People were followed for 30 weeks. They had 8 weeks without the drug. Then came 12 weeks on dapagliflozin 10 mg a day. Then came 10 weeks off it. It was open-label and not randomized. The team tracked blood pressure, NT-proBNP, symptom scores and a 6-minute walk.

What they found

One person left because of genitourinary side effects, and others left for unrelated reasons. NT-proBNP fell by 142 pg/mL, but the range included no change. Walk distance rose by 24.3 m, but the range included no change. The symptom score changed by 0.4 points, with a wide range. Sitting systolic blood pressure was 8.8 mmHg lower during treatment. No rise in low blood pressure symptoms or heart side effects was seen.

What it means for patients

The authors say dapagliflozin was generally well tolerated. They say the exploratory findings support further testing in an adequately powered randomized trial.

Why it is not certain yet

The differences in NT-proBNP, walk distance and symptom score were imprecise. They gave no firm proof of benefit. The study was open-label and not randomized. The team calls for a larger trial with enough power.

Source

Using dapagliflozin to reduce symptoms in transthyretin cardiac amyloidosis: An n-of-1 trial series pilot study.

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