Abstract
IntroductionThe effectiveness of tafamidis for the treatment of transthyretin familial amyloid polyneuropathy (TTR-FAP) was evaluated using data from the Transthyretin Amyloidosis Outcomes Survey (THAOS) registry.MethodsSubjects receiving tafamidis (n = 252) were compared with untreated subjects in a non-randomized, matched cohort analysis. Subjects were matched with up to four untreated controls by genetic mutation, region of birth, and mean treatment propensity score.ResultsThe matched, treated sample consisted predominantly of subjects with the Val30Met genotype (92.5%), from Portugal, and with a mean age of 40.4 years. Over the course of the 2-year follow-up period, subjects treated with tafamidis showed significantly less deterioration on the Neuropathy Impairment Score for Lower Limbs (p < 0.001) and its subscales (p < 0.023) compared with untreated subjects. There was significantly less deterioration among tafamidis-treated subjects compared with untreated subjects on the Norfolk Quality of Life scale (p < 0.001). There were no significant differences observed in functional (assessed by Karnofsky Performance Status Scale score) or nutritional (assessed by modified body mass index) status between the treated and untreated groups. The primary model which examined survival from baseline using the matched cohort was not able to yield estimates of the hazard ratio, as there were no deaths in the tafamidis-treated subjects.ConclusionThese findings support the results from clinical trials and strengthen evidence of the effectiveness of tafamidis beyond conventional clinical trials.Trial RegistrationClinicalTrials.gov: NCT00628745FundingPfizer.
Highlights
The effectiveness of tafamidis for the treatment of transthyretin familial amyloid polyneuropathy (TTR-FAP) was evaluated using data from the Transthyretin Amyloidosis Outcomes Survey (THAOS) registry
Transthyretin familial amyloid polyneuropathy (TTR-FAP) is a rare, life-threatening, autosomaldominant illness caused by TTR gene mutations that result in the deposition of amyloid fibrils in Neurol Ther (2018) 7:87–101 peripheral nerves and organs [1,2,3]
THAOS is an observational registry for which any individual with a confirmed TTR mutation or wild-type amyloid TTR (ATTR) amyloidosis is eligible, and includes some patients participating in ongoing clinical trials [20]
Summary
The effectiveness of tafamidis for the treatment of transthyretin familial amyloid polyneuropathy (TTR-FAP) was evaluated using data from the Transthyretin Amyloidosis Outcomes Survey (THAOS) registry. Methods: Subjects receiving tafamidis (n = 252) were compared with untreated subjects in a non-randomized, matched cohort analysis. Subjects were matched with up to four untreated controls by genetic mutation, region of birth, and mean treatment propensity score. Results: The matched, treated sample consisted predominantly of subjects with the Val30Met genotype (92.5%), from Portugal, and with a mean age of 40.4 years. Over the course of the 2-year follow-up period, subjects treated with tafamidis showed significantly less deterioration on the Neuropathy Impairment Score for Lower. There was significantly less deterioration among tafamidistreated subjects compared with untreated subjects on the Norfolk Quality of Life scale (p \ 0.001). The primary model which examined survival from baseline using the matched cohort was not able to yield estimates of the hazard ratio, as there were no deaths in the tafamidis-treated subjects. Transplantation is associated with the risk of mortality and the need for lifelong immunosuppressant therapy, and recent evidence suggests that survival rates are lower in patients with mutations other than Val30Met [11, 12]
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