[ATTRv amyloidosis with early improvement demonstrated by the 6-minute walk test following Patisiran therapy: a case report]

Rinsho Shinkeigaku. 2022 May 31;62(5):375-379. doi: 10.5692/clinicalneurol.cn-001693. Epub 2022 Apr 26.
[Article in Japanese]

Abstract

We report the case of a 65-year-old man who gradually developed numbness in both hands, lower limb muscle weakness and atrophy, and orthostatic hypotension over two and a half years. These symptoms indicated hereditary ATTR amyloidosis (ATTRv amyloidosis), and the final diagnosis was established through proof of TTR gene mutation (V30M). We initiated patisiran therapy, and a continuous 6-minute walking test performed 3 weeks from the start of therapy demonstrated improvement in the walking distance. This is a single case report showing the improvement in the motor and sensory function on administration of patisiran monotherapy from an early stage.

Keywords: 6-minute walking test; ATTRv amyloidosis; V30M mutation; neuropathy; patisiran.

Publication types

  • Case Reports

MeSH terms

  • Aged
  • Amyloid Neuropathies, Familial* / diagnosis
  • Amyloid Neuropathies, Familial* / drug therapy
  • Amyloid Neuropathies, Familial* / genetics
  • Humans
  • Male
  • Prealbumin / genetics
  • RNA, Small Interfering
  • Walk Test

Substances

  • Prealbumin
  • RNA, Small Interfering
  • patisiran