Neurofilament Light Chain Levels Decrease With Treatment in hATTR Amyloidosis

Among patients with hereditary transthyretin-mediated (hATTR) amyloidosis with polyneuropathy, neurofilament light chain (NfL) levels decreased in response to treatment with patisiran or vutrisiran, according to study results presented at the 2023 American Association of Neuromuscular & Electrodiagnostic Medicine (AANEM) Annual Meeting, held from November 1 to 4 in Phoenix, Arizona.

Researchers have suggested the role of NfL as a potential biomarker of disease progression and treatment response in hATTR amyloidosis with polyneuropathy.

In the study, the researchers analyzed NfL levels among patients with hATTR amyloidosis receiving treatment with patisiran or vutrisiran.

For the analysis, the researchers included data from patients in the APOLLO and HELIOS-A trials. They measured NfL plasma levels at various timepoints in the control and treatment groups.

The researchers found that NfL levels were higher among patients in the APOLLO than the HELIOS-A study (69.4 vs 58.2 pg/mL, respectively).

Among patients in the APOLLO study, those who received placebo had increasing levels of NfL vs those who received patisiran who had decreasing levels of NfL at 4 and 18 months. Similar observations were made among patients in the HELIOS-A study who had decreases in NfL levels from baseline with patisiran and vutrisiran treatment at 4 and 18 months (Table).

Table: NfL levels with placebo vs patisiran/vutrisiran at 4 and 18 months among patients in the APOLLO and HELIOS-A trials

Based on these findings, the researchers noted, “NfL may serve as a biomarker of

treatment response as early as 4 months following initiation of patisiran or vutrisiran.” They concluded, [NfL may be] “potentially useful for monitoring disease progression and treatment response in hATTR amyloidosis with polyneuropathy.”