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1. A 50-year-old man has type 2 diabetes that has been well controlled for 5 years. Over the past 6 months, burning numbness in his feet has worsened quickly. He now has bilateral foot drop, a Charcot joint, bilateral carpal tunnel syndrome, trigger fingers, and lumbar stenosis, and he was recently diagnosed with HFpEF. EMG/NCS shows a length-dependent axonal sensorimotor neuropathy with no signs of demyelination. What is the most appropriate next step?
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Attribute the neuropathy to diabetes, tighten glycemic control, and reassess in 6 months
Start a trial of IVIg for presumed CIDP, since CIDP is a common cause of rapidly progressive neuropathy
Order TTR genetic testing with genetic counseling, along with monoclonal protein testing to rule out AL amyloidosis
Defer further workup unless an echocardiogram shows an apical-sparing strain pattern
2. Which statement about hATTR-PN therapy is best supported by the evidence presented?
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HELIOS-A compared vutrisiran with a concurrent randomized placebo arm
Tafamidis is FDA-approved for hATTR-PN based on its NIS-LL endpoint
TTR gene silencers showed benefit on mNIS+7 compared with placebo across baseline neuropathy severity, including more advanced disease
Gene silencers reliably restore large-fiber and motor function to normal
3. The patient from question 1 has a confirmed pathogenic TTR variant (Ser97Tyr) and AL amyloidosis has been ruled out. He has HFpEF, a borderline echocardiogram, and a non-healing plantar wound. He has two adult children, ages 22 and 26. Which plan best reflects individualized care with shared decision-making?
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Wait on disease-modifying therapy until cardiac amyloidosis is confirmed, since his echocardiogram is not typical of advanced disease
Choose therapy based only on mNIS+7 severity, and test both children right away
Coordinate with cardiology, genetic counseling, and wound/PT care. Review gene silencer and stabilizer options against his goals and preferences for how the drug is given. Offer his children genetic counseling, with cascade testing timed around the predicted age of onset
Refer to palliative care alone, given how advanced his neuropathy is
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