Treatment response and neurofilament light chain levels with long-term patisiran in hereditary transthyretin-mediated amyloidosis with polyneuropathy: 24-month results of an open-label extension study.
ATTRv amyloidosis
hATTR amyloidosis
neurofilament light chain
patisiran
polyneuropathy
Journal
Amyloid : the international journal of experimental and clinical investigation : the official journal of the International Society of Amyloidosis
ISSN: 1744-2818
Titre abrégé: Amyloid
Pays: England
ID NLM: 9433802
Informations de publication
Date de publication:
20 Jul 2023
20 Jul 2023
Historique:
pubmed:
20
7
2023
medline:
20
7
2023
entrez:
20
7
2023
Statut:
aheadofprint
Résumé
Longitudinal changes in neurofilament light chain (NfL) levels were evaluated alongside prespecified clinical assessments 24 months into the patisiran Global open-label extension (OLE) study in patients with ATTRv amyloidosis with polyneuropathy. All patients enrolled in the Global OLE, from phase III APOLLO and phase II OLE parent studies, received patisiran. Assessments included measures of polyneuropathy (modified Neuropathy Impairment Score+7 (mNIS+7)), quality of life (QOL; Norfolk QOL-Diabetic Neuropathy questionnaire (Norfolk QOL-DN)), and plasma NfL. Patients receiving patisiran in the parent study (APOLLO-patisiran, Long-term patisiran treatment led to sustained improvements in neuropathy and QOL, with NfL demonstrating potential as a biomarker for disease progression and treatment response in ATTRv amyloidosis with polyneuropathy.
Sections du résumé
BACKGROUND
UNASSIGNED
Longitudinal changes in neurofilament light chain (NfL) levels were evaluated alongside prespecified clinical assessments 24 months into the patisiran Global open-label extension (OLE) study in patients with ATTRv amyloidosis with polyneuropathy.
METHODS
UNASSIGNED
All patients enrolled in the Global OLE, from phase III APOLLO and phase II OLE parent studies, received patisiran. Assessments included measures of polyneuropathy (modified Neuropathy Impairment Score+7 (mNIS+7)), quality of life (QOL; Norfolk QOL-Diabetic Neuropathy questionnaire (Norfolk QOL-DN)), and plasma NfL.
RESULTS
UNASSIGNED
Patients receiving patisiran in the parent study (APOLLO-patisiran,
CONCLUSIONS
UNASSIGNED
Long-term patisiran treatment led to sustained improvements in neuropathy and QOL, with NfL demonstrating potential as a biomarker for disease progression and treatment response in ATTRv amyloidosis with polyneuropathy.
Identifiants
pubmed: 37469249
doi: 10.1080/13506129.2023.2232520
doi:
Types de publication
Journal Article
Langues
eng
Sous-ensembles de citation
IM
Pagination
1-11Commentaires et corrections
Type : ErratumIn