Comparison 3. Patisiran versus placebo in TTR‐FAP.

Outcome or subgroup title	No. of studies	No. of participants	Statistical method	Effect size
1 Disability due to FAP progression (expressed as the change from baseline in the R‐ODS), at 18 months	1	225	Mean Difference (IV, Fixed, 95% CI)	8.9 [7.00, 10.80]
2 Severity of peripheral neuropathy (expressed as the change from baseline in the mNIS+7Alnylam), at 18 months	1	225	Mean Difference (IV, Fixed, 95% CI)	‐33.99 [‐39.86, ‐28.12]
3 Change in mBMI, at 18 months	1	225	Mean Difference (IV, Fixed, 95% CI)	115.7 [82.40, 149.00]
4 Quality of life (change in Norfolk QOL‐DN total score), at 18 months	1	225	Mean Difference (IV, Fixed, 95% CI)	‐21.1 [‐27.20, ‐15.00]
5 Number of participants who died during the trial	1	225	Risk Ratio (M‐H, Fixed, 95% CI)	0.61 [0.21, 1.74]
6 Number of participants experiencing at least one adverse event	1	225	Risk Ratio (M‐H, Fixed, 95% CI)	0.99 [0.95, 1.04]
7 Number of participant dropouts due to adverse events (data from published paper ‐ see text)	1	225	Risk Ratio (M‐H, Fixed, 95% CI)	0.33 [0.13, 0.82]
8 Number of participants experiencing at least one severe adverse event	1	225	Risk Ratio (M‐H, Fixed, 95% CI)	0.91 [0.64, 1.28]