The results of a randomized trial suggest that tafamidis treatment may slow the progression of early-stage V30M transthyretin familial amyloid polyneuropathy.
During 18 months of treatment, 45.3% of the 65 patients given tafamidis (see structure) 20 mg/day worsened by less than 2 points on the Neuropathy Impairment Score-Lower Limbs (NIS-LL). The rate among 63 placebo-treated patients was 29.5%, which was a nonsignificant difference.
The Norfolk Quality of Life-Diabetic Neuropathy total score (TQOL) worsened by 2.0 points in the tafamidis group, compared with 7.2 points in the placebo group - also a nonsignificant difference.
But this analysis was based on the intent-to-treat population. A higher than anticipated dropout rate, caused by patients requiring liver transplantation, meant that just 87 of the original 125 patients actually completed the treatment.
In this group, 60.0% versus 38.1% of those treated with tafamidis and placebo had a less than 2-point deterioration on the NIS-LL, and TQOL scores worsened by a corresponding 0.1 versus 8.9.....
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Hint of tafamidis benefit in rare polyneuropathy
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