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Neurofilament light chain as a biomarker for monitoring response to change in treatment in hereditary ATTR amyloidosis

Neurofilament light chain as a biomarker for monitoring response to change in treatment in... AMYLOID https://doi.org/10.1080/13506129.2023.2187678 LETTER TO THE EDITOR Neurofilament light chain as a biomarker for monitoring response to change in treatment in hereditary ATTR amyloidosis Hereditary ATTR (ATTRv) amyloidosis is a fatal autosomal (p.S70A), and I107V (p.I127V), (n¼ 1 each). NfL and NIS dominant disorder in which variants in the transthyretin measurements at baseline were available for all 11 patients. (TTR) gene cause systemic deposition of amyloid fibrils. Serum NfL levels were available for 11/11 and 8/11 patients ATTRv amyloidosis is characterised by progressive length- at one and two years following switch, respectively. NIS dependent sensorimotor neuropathy, autonomic neuropathy, measurements were conducted in 9/11 and 8/11 patients at and non-neuropathic manifestations [1]. Several effective one and two years after switch, respectively. pharmacological therapies are available, including TTR Serum NfL levels significantly decreased at one and two tetramer stabilisers (diflunisal and tafamidis) and nucleic years following switch from tafamidis to patisiran (Figure acid-based medications such as RNA interference therapeu- 1). The mean (±SD) NfL level at baseline, before switch, tics (patisiran and vutrisiran) and anti-sense oligonucleotide was 106.4 (±50.7) pg/mL and it decreased to 72.6 (±47.3) (inotersen) [1,2]. However, evaluation and monitoring of pg/mL one year after switch (p¼ 0.001). In patients http://www.deepdyve.com/assets/images/DeepDyve-Logo-lg.png Amyloid Taylor & Francis

Neurofilament light chain as a biomarker for monitoring response to change in treatment in hereditary ATTR amyloidosis

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Neurofilament light chain as a biomarker for monitoring response to change in treatment in hereditary ATTR amyloidosis

Abstract

AMYLOID https://doi.org/10.1080/13506129.2023.2187678 LETTER TO THE EDITOR Neurofilament light chain as a biomarker for monitoring response to change in treatment in hereditary ATTR amyloidosis Hereditary ATTR (ATTRv) amyloidosis is a fatal autosomal (p.S70A), and I107V (p.I127V), (n¼ 1 each). NfL and NIS dominant disorder in which variants in the transthyretin measurements at baseline were available for all 11 patients. (TTR) gene cause systemic deposition of amyloid fibrils. Serum...
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Publisher
Taylor & Francis
Copyright
© 2023 Informa UK Limited, trading as Taylor & Francis Group
ISSN
1744-2818
eISSN
1350-6129
DOI
10.1080/13506129.2023.2187678
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Abstract

AMYLOID https://doi.org/10.1080/13506129.2023.2187678 LETTER TO THE EDITOR Neurofilament light chain as a biomarker for monitoring response to change in treatment in hereditary ATTR amyloidosis Hereditary ATTR (ATTRv) amyloidosis is a fatal autosomal (p.S70A), and I107V (p.I127V), (n¼ 1 each). NfL and NIS dominant disorder in which variants in the transthyretin measurements at baseline were available for all 11 patients. (TTR) gene cause systemic deposition of amyloid fibrils. Serum NfL levels were available for 11/11 and 8/11 patients ATTRv amyloidosis is characterised by progressive length- at one and two years following switch, respectively. NIS dependent sensorimotor neuropathy, autonomic neuropathy, measurements were conducted in 9/11 and 8/11 patients at and non-neuropathic manifestations [1]. Several effective one and two years after switch, respectively. pharmacological therapies are available, including TTR Serum NfL levels significantly decreased at one and two tetramer stabilisers (diflunisal and tafamidis) and nucleic years following switch from tafamidis to patisiran (Figure acid-based medications such as RNA interference therapeu- 1). The mean (±SD) NfL level at baseline, before switch, tics (patisiran and vutrisiran) and anti-sense oligonucleotide was 106.4 (±50.7) pg/mL and it decreased to 72.6 (±47.3) (inotersen) [1,2]. However, evaluation and monitoring of pg/mL one year after switch (p¼ 0.001). In patients

Journal

Amyloid – Taylor & Francis

Published: Mar 10, 2023

References

  • Guidelines and new directions in the therapy and monitoring of ATTRv amyloidosis
  • Vutrisiran: first approval
  • Neurofilament light chain as a biomarker of hereditary transthyretin-mediated amyloidosis
  • Plasma neurofilament light chain: an early biomarker for hereditary ATTR amyloid polyneuropathy
  • Tafamidis for transthyretin familial amyloid polyneuropathy: a randomized, controlled trial