Fig. 4.

Tafamidis treatment significantly reduced circulating NNTTR levels while increasing tetrameric or tTTR levels in V30M TTR FAP patients (n = 81). (A) NNTTR levels were measured using the NNTTR-Dx assay (one-way ANOVA, Dunnett test, P = 0.0001 vs. pretreatment for both 12 and 24 mo). (B) tTTR levels measured employing a sandwich ELISA using antibody 6H3 as the capture and detection pair (one-way ANOVA, Dunnet test, P = 0.003 and 0.001 for 12 and 24 mo, respectively). All assays were run with blinded plasma samples. (C) % Change in total native TTR levels after tafamidis treatment for the R and NR groups at 12 and 24 mo (range with 95% confidence interval). No statistically significant difference in total native TTR changes was seen among the groups. (D) There is a weak negative correlation (Pearson’s r coefficient is −0.3773) between NNTTR and native TTR levels in plasma samples (n = 219).