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. 2020 Apr 20;2020(4):CD012395. doi: 10.1002/14651858.CD012395.pub2

Comparison 1. Tamafidis versus placebo in TTR‐FAP.

Outcome or subgroup title No. of studies No. of participants Statistical method Effect size
1 Severity of peripheral neuropathy (expressed as the change from baseline in NIS‐LL) 1   Mean Difference (IV, Fixed, 95% CI) Subtotals only
1.1 12 months 1 125 Mean Difference (IV, Fixed, 95% CI) ‐3.83 [‐5.99, ‐1.67]
1.2 18 months 1 125 Mean Difference (IV, Fixed, 95% CI) ‐3.21 [‐5.63, ‐0.79]
2 Change in mBMI 1   Mean Difference (IV, Fixed, 95% CI) Subtotals only
2.1 12 months 1 125 Mean Difference (IV, Fixed, 95% CI) 50.2 [24.46, 75.94]
2.2 18 months 1 125 Mean Difference (IV, Fixed, 95% CI) 70.6 [41.96, 99.24]
3 Quality of life (expressed as the change from baseline in Norfolk QOL‐DN total score) 1   Mean Difference (IV, Fixed, 95% CI) Subtotals only
3.1 12 months 1 125 Mean Difference (IV, Fixed, 95% CI) ‐3.50 [‐9.48, 2.48]
3.2 18 months 1 125 Mean Difference (IV, Fixed, 95% CI) ‐4.5 [‐11.27, 2.27]
4 Number of participants who died during the trial (published paper ‐ see text) 1 128 Risk Ratio (M‐H, Fixed, 95% CI) 0.65 [0.11, 3.74]
5 Number of participants experiencing at least one adverse event 1 128 Risk Ratio (M‐H, Fixed, 95% CI) 0.95 [0.88, 1.04]
6 Number of participants experiencing mild adverse events 1 128 Risk Ratio (M‐H, Fixed, 95% CI) 0.99 [0.87, 1.12]
7 Number of dropouts due to adverse events 1 128 Risk Ratio (M‐H, Fixed, 95% CI) 1.29 [0.30, 5.54]
8 Number of participants experiencing at least one severe adverse event 1 128 Risk Ratio (M‐H, Fixed, 95% CI) 1.16 [0.37, 3.62]