Findings

What do systematic reviews say?

6 questions · 6 findings · 1 reviews

No studies found 0 · 0%GRADE not reported 0 · 0%Very low 0 · 0%Low 4 · 66.7%Moderate 2 · 33.3%High 0 · 0%

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

Pharmacological treatment for familial amyloid polyneuropathy 2020 · Cochrane Headline outcomes only
no estimate extracted
1 study · 225 participants
Moderate

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

Pharmacological treatment for familial amyloid polyneuropathy 2020 · Cochrane Headline outcomes only
no estimate extracted
1 study · 225 participants
Moderate

QoL · people with TTR‐FAP

1 finding from 1 review

Pharmacological treatment for familial amyloid polyneuropathy 2020 · Cochrane Headline outcomes only
no estimate extracted
1 study · 225 participants
Low

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

Pharmacological treatment for familial amyloid polyneuropathy 2020 · Cochrane Headline outcomes only
RR 0.61 [0.21, 1.74]
1 study · 225 participants
Low

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

Pharmacological treatment for familial amyloid polyneuropathy 2020 · Cochrane Headline outcomes only
RR 0.33 [0.13, 0.82]
1 study · 225 participants
Low

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

Pharmacological treatment for familial amyloid polyneuropathy 2020 · Cochrane Headline outcomes only
RR 0.91 [0.64, 1.28]
1 study · 225 participants
Low