Influence of baseline neurologic severity on disease progression and the associated disease-modifying effects of tafamidis in patients with transthyretin amyloid polyneuropathy

Table 3 Estimated NIS-LL muscle weakness at Months 6, 12, and 18, according to severity of baseline muscle weakness and treatment group

Baseline NIS-LL muscle weakness	Treatment group	Month 6	Month 12	Month 18
5	T-T	5.5 (0.4)	6.0 (0.4)	6.5 (0.6)
	P-T	6.3 (0.4)	7.5 (0.4)	8.8 (0.6)
15	T-T	16.9 (0.5)	18.1 (0.6)	19.3 (0.7)
	P-T	17.7 (0.5)	19.6 (0.6)	21.6 (0.7)
25	T-T	28.3 (0.8)	30.2 (0.8)	32.2 (1.0)
	P-T	29.1 (0.7)	31.7 (0.8)	34.4 (1.0)

Data are shown as mean (standard error). The reason for the slightly higher mean scores in muscle weakness at certain time points (compared with the overall NIS-LL score) can be explained by the fact that separate models were conducted on NIS-LL and NIS-LL muscle weakness, which makes cross-comparisons problematic. In addition, comparable baseline scores on the two endpoints can be very different in terms of reflecting disease severity and progression; a patient with a muscle weakness score of 15 would likely be more severely ill and progress more rapidly on this scale than a patient with a comparable baseline of 15 on the total NIS-LL (this was confirmed by the present analysis where the baseline coefficient for NIS-LL muscle weakness was greater than the coefficient for NIS-LL, suggesting faster clinical decline in muscle weakness than NIS-LL within the same period, as baseline increases)
NIS-LL Neuropathy Impairment Score − Lower Limbs; P-T placebo-to-tafamidis; T-T tafamidis-to-tafamidis

ISSN: 1750-1172