Disability due to FAP progression (expressed as the change in R‐ODS) Scale from: 0 (worst disability) to 48 (normal) · people with TTR‐FAP
1 finding from 1 review
Severity of peripheral neuropathy (expressed as the change in mNIS+7ALN) Scale from: 0 (normal) to 304 (total impairment) · people with TTR‐FAP
1 finding from 1 review
QoL · people with TTR‐FAP
1 finding from 1 review
Has died · people with TTR‐FAP
Patisiran showed no clear difference in number of participants who died during the trial compared with placebo (low certainty).
1 finding from 1 review
Withdrawals due to adverse events · people with TTR‐FAP
Patisiran may leads to lower number of dropouts due to adverse events compared with placebo (low certainty).
1 finding from 1 review
Number of participants experiencing at least one severe adverse event · people with TTR‐FAP
Patisiran showed no clear difference in number of participants experiencing at least one severe adverse event compared with placebo (low certainty).
1 finding from 1 review
Severity of peripheral neuropathy (expressed as the change in NIS‐LL) Scale from: 0 (normal) to 88 (total impairment) · people with early‐stage TTR‐FAP
1 finding from 1 review
Disability due to FAP progression Not measured · people with TTR‐FAP
The review found no eligible studies for this question.
1 finding from 1 review
no estimate extracted
Studies not reported
No studies found
Severity of peripheral neuropathy (expressed as the change in mNIS+7Ionis) Scale from: ‐22.3 (normal) to 346.3 (total impairment) · people with TTR‐FAP
1 finding from 1 review
QoL · people with TTR‐FAP
1 finding from 1 review
Has died · people with TTR‐FAP
Inotersen showed no clear difference in number of participants who died during the trial compared with placebo (low certainty).
1 finding from 1 review
Withdrawals due to adverse events · people with TTR‐FAP
Inotersen may leads to higher number of dropouts due to adverse events compared with placebo (low certainty).
1 finding from 1 review
Number of participants experiencing at least one severe adverse event · people with TTR‐FAP
Inotersen showed no clear difference in number of participants experiencing at least one severe adverse event compared with placebo (low certainty).
1 finding from 1 review
QoL · people with early‐stage TTR‐FAP
1 finding from 1 review
Has died · people with early‐stage TTR‐FAP
It is very uncertain whether tafamidis changes number of participants who died during the trial compared with placebo (very low certainty).
1 finding from 1 review
Withdrawals due to adverse events · people with early‐stage TTR‐FAP
It is very uncertain whether tafamidis changes number of dropouts due to adverse events compared with placebo (very low certainty).
1 finding from 1 review
Number of participants experiencing at least one severe adverse event · people with early‐stage TTR‐FAP
It is very uncertain whether tafamidis changes number of participants experiencing at least one severe adverse event compared with placebo (very low certainty).
1 finding from 1 review
Disability due to FAP progression (expressed as the change in Kumamoto Score) Scale from: 0 (normal) to 102 (worst disease severity). · people with TTR‐FAP
1 finding from 1 review
Severity of peripheral neuropathy (expressed as the change in NIS+7) Scale from: 0 (normal) to 270 (total impairment) · people with TTR‐FAP
1 finding from 1 review
Physical quality of life · people with TTR‐FAP
1 finding from 1 review