Cochrane · systematic review · CD012395
Pharmacological treatment for familial amyloid polyneuropathy
Magrinelli, F, Fabrizi, GM, Santoro, L, et al. · Cochrane Database of Systematic Reviews · 2020 · Cochrane Library
NeuromuscularAMSTAR 2: not assessed
SoF tableOnly the outcomes this review put in its Summary of Findings table are listed (the Cochrane Handbook caps them at about seven per comparison). Its other analyses are not shown here.
Questions
25
current version
GRADE rated
23
No studies
2
Included studies
NR
No studies 2 · 8%Ungraded 0 · 0%Very low 9 · 36%Low 11 · 44%Moderate 3 · 12%High 0 · 0%
Ungraded means an effect was reported without a GRADE rating; it is not a gap. No studies means the review found nothing to answer the question. The six categories are a display order, not a score.
Questions and results
Tafamidis compared to placebo for people with transthyretin‐familial amyloid polyneuropathy (TTR‐FAP)
Population: people with early‐stage TTR‐FAP
| Outcome | Effect | Evidence | Status / GRADE |
|---|---|---|---|
Disability due to FAP progression Not measured Tafamidis vs placebo | no estimate extracted | NR | No studies |
Severity of peripheral neuropathy (expressed as the change in NIS‐LL) Scale from: 0 (normal) to 88 (total impairment) Tafamidis vs placebo | no estimate extracted | 1 study · 125 participants | Low Downgraded: risk of bias, imprecision |
Quality of life (expressed as the change in 35‐Item Norfolk QOL‐DN total score) Scale from: ‐2 (best possible quality of life) to 138 (worst possible quality of life) Tafamidis vs placebo | no estimate extracted | 1 study · 125 participants | Very low Downgraded: risk of bias, imprecision, indirectness |
Number of participants who died during the trial Tafamidis vs placebo | RR 0.65 [0.11, 3.74] | 1 study · 128 participants | Very low Downgraded: risk of bias, imprecision, indirectness |
Number of dropouts due to adverse events Tafamidis vs placebo | RR 1.29 [0.3, 5.54] | 1 study · 128 participants | Very low Downgraded: risk of bias, imprecision, indirectness |
Number of participants experiencing at least one severe adverse event Tafamidis vs placebo | RR 1.16 [0.37, 3.62] | 1 study · 128 participants | Very low Downgraded: risk of bias, imprecision, indirectness |
Diflunisal compared to placebo for people with transthyretin‐familial amyloid polyneuropathy (TTR‐FAP)
Population: people with TTR‐FAP
| Outcome | Effect | Evidence | Status / GRADE |
|---|---|---|---|
Disability due to FAP progression (expressed as the change in Kumamoto Score) Scale from: 0 (normal) to 102 (worst disease severity). Diflunisal vs placebo | no estimate extracted | 1 study · 130 participants | Low Downgraded: risk of bias, imprecision |
Severity of peripheral neuropathy (expressed as the change in NIS+7) Scale from: 0 (normal) to 270 (total impairment) Diflunisal vs placebo | no estimate extracted | 1 study · 130 participants | Low Downgraded: risk of bias, imprecision |
Quality of life (expressed as the change in SF‐36 physical component score) Scale from: 0 (worst possible quality of life) to 100 (best possible quality of life) Diflunisal vs placebo | no estimate extracted | 1 study · 130 participants | Very low Downgraded: risk of bias, imprecision, indirectness |
Quality of life (expressed as the change in SF‐36 mental component score) Scale from: 0 (worst possible quality of life) to 100 (best possible quality of life). Diflunisal vs placebo | no estimate extracted | 1 study · 130 participants | Very low Downgraded: risk of bias, imprecision, indirectness |
Number of participants who died during the trial Diflunisal vs placebo | RR 0.46 [0.15, 1.41] | 1 study · 130 participants | Very low Downgraded: risk of bias, imprecision |
Number of dropouts due to adverse events Diflunisal vs placebo | RR 2.06 [0.39, 10.87] | 1 study · 130 participants | Very low Downgraded: risk of bias, imprecision |
Number of participants experiencing at least one severe adverse event Diflunisal vs placebo | RR 0.77 [0.18, 3.32] | 1 study · 130 participants | Very low Downgraded: risk of bias, imprecision |
Patisiran compared to placebo for people with transthyretin‐familial amyloid polyneuropathy (TTR‐FAP)
Population: people with TTR‐FAP
| Outcome | Effect | Evidence | Status / GRADE |
|---|---|---|---|
Disability due to FAP progression (expressed as the change in R‐ODS) Scale from: 0 (worst disability) to 48 (normal) Patisiran vs placebo | no estimate extracted | 1 study · 225 participants | Moderate Downgraded: imprecision |
Severity of peripheral neuropathy (expressed as the change in mNIS+7ALN) Scale from: 0 (normal) to 304 (total impairment) Patisiran vs placebo | no estimate extracted | 1 study · 225 participants | Moderate Downgraded: imprecision |
Quality of life (expressed as the change in 35‐Item Norfolk QOL‐DN total score) Scale from: ‐4 (best possible quality of life) to 136 (worst possible quality of life) Patisiran vs placebo | no estimate extracted | 1 study · 225 participants | Low Downgraded: imprecision, indirectness |
Number of participants who died during the trial Patisiran vs placebo | RR 0.61 [0.21, 1.74] | 1 study · 225 participants | Low Downgraded: imprecision |
Number of dropouts due to adverse events Patisiran vs placebo | RR 0.33 [0.13, 0.82] | 1 study · 225 participants | Low Downgraded: imprecision |
Number of participants experiencing at least one severe adverse event Patisiran vs placebo | RR 0.91 [0.64, 1.28] | 1 study · 225 participants | Low Downgraded: imprecision |
Inotersen compared to placebo for people with transthyretin‐familial amyloid polyneuropathy (TTR‐FAP)
Population: people with TTR‐FAP
| Outcome | Effect | Evidence | Status / GRADE |
|---|---|---|---|
Disability due to FAP progression Not measured Inotersen vs placebo | no estimate extracted | NR | No studies |
Severity of peripheral neuropathy (expressed as the change in mNIS+7Ionis) Scale from: ‐22.3 (normal) to 346.3 (total impairment) Inotersen vs placebo | no estimate extracted | 1 study · 172 participants | Moderate Downgraded: imprecision |
Quality of life (expressed as the change in 35‐Item Norfolk QOL‐DN total score) Scale from: ‐4 (best possible quality of life) to 136 (worst possible quality of life) Inotersen vs placebo | no estimate extracted | 1 study · 172 participants | Low Downgraded: imprecision, indirectness |
Number of participants who died during the trial Inotersen vs placebo | RR 5.94 [0.33, 105.6] | 1 study · 172 participants | Low Downgraded: imprecision |
Number of dropouts due to adverse events Inotersen vs placebo | RR 8.57 [1.16, 63.07] | 1 study · 172 participants | Low Downgraded: imprecision |
Number of participants experiencing at least one severe adverse event Inotersen vs placebo | RR 1.48 [0.85, 2.57] | 1 study · 172 participants | Low Downgraded: imprecision |