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Déjerine-Roussy – Treatment — SCE Neurology MCQ

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HardCerebrovascular DiseaseDéjerine-Roussy – TreatmentSCE Neurology

A 56-year-old with genetically confirmed hereditary transthyretin amyloidosis has progressive sensory, motor and autonomic neuropathy but can still walk with one stick, corresponding to stage 2 polyneuropathy. Which statement about NHS disease-modifying therapy is correct?

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Correct answer: A — NICE recommends vutrisiran for ambulant hereditary ATTR polyneuropathy within its marketing authorisation

Explanation lettering: E = shown as C · C = shown as E

A is correct: NICE recommends vutrisiran within its marketing authorisation for adults with hereditary ATTR amyloidosis and stage 1 or stage 2 polyneuropathy. B proposes presymptomatic treatment outside the recommendation. C delays treatment beyond the appraised ambulatory stages. D confuses hereditary polyneuropathy with wild-type cardiac amyloidosis. E is wrong because ATTR is multisystem disease; treatment choice and follow-up still require specialist assessment of neuropathy, autonomic function and cardiac involvement.

Reference: NICE TA868: vutrisiran for hereditary transthyretin-related amyloidosis: https://www.nice.org.uk/guidance/ta868/chapter/1-Recommendations