Neurofilament light chain showed potential as marker of active nerve damage and polyneuropathy in hereditary transthyretin amyloidosis (hATTR), according to a new study conducted by a multi-institutional team of researchers.
“NfL [neurofilament light chain] may serve as a biomarker of active nerve damage and polyneuropathy in hATTR amyloidosis, making it potentially useful as a biomarker to monitor disease progression and treatment response adjunct to clinical,” the authors of the study wrote in an abstract published in Clinical Neurophysiology.
Neurofilament light chain levels in patients from the phase 3 APOLLO study decreased following 36 months of patisiran (Onpattro®) treatment (mean, 44.8 pg/mL, n=72 vs mean, 72.0 pg/mL, n=111, at baseline). Patients in the placebo group also experienced a reduction in neurofilament light chain levels at 36 months.
Similarly, neurofilament light chain levels in patients enrolled in the phase 2 open-label extension also decreased following 60 months of patisiran treatment (mean, 26.1 pg/mL, n=19).
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Previous analysis had already demonstrated a significant reduction in neurofilament light chain plasma levels through 18 months of patisiran treatment. Conversely, neurofilament light chain plasma levels increased in the placebo group.
The post hoc analysis included data from the phase 3 APOLLO and the phase 2 open-label extension parent studies, as well as the global open-label extension study (NCT02510261), which enrolled patients who had completed a patisiran parent study and, in the opinion of the investigator, tolerated the study drug.
Gilling K, Aldinc E, Ticau S, et al. P-48 neurofilament light chain as a biomarker in hereditary transthyretin-mediated amyloidosis: 36-month data from the patisiran global open-label extension. Clin Neurophysiol. 2023;148:e30. doi:https://doi.org/10.1016/j.clinph.2023.02.065