Conclusion
sensation loss, neuropathic impairment, FAP stage, muscle weakness
MUSCLE WEAKNESS, CARDIOMYOPATHY, ATTRV-PN
In conclusion, this is the first research to examine the efficacy of inotersen on NIS and NIS-LL scores among subgroups of patients with ATTRv-PN based on key clinical characteristics, such as genetic mutation, FAP stage, pretreatment status, presence of cardiomyopathy, and age of onset. Additionally, RD thresholds for NIS and NIS-LL scores in patients with ATTRv-PN were estimated using anchor-based and/or distribution-based methods, allowing for improved evaluation of treatment benefit for slowing neuropathic impairment. The mean of these estimates was given as the recommended RD threshold for the total and subscale scores of the NIS (total: 8.1 points; muscle weakness: 5.3; sensation: 2.4; and reflexes: 2.0) and NIS-LL (total: 4.7 points; muscle weakness: 3.4; sensation: 1.3; and reflexes: 0.9). Patient-level responder analyses using these RD thresholds showed that inotersen provides a clinically meaningful benefit for limiting the progression of neuropathic impairment in patients with ATTRv-PN, particularly on measures of muscle weakness and sensation loss. These results support previous evidence demonstrating the efficacy of inotersen in this patient population.