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Tafamidis, a Noninvasive Therapy for Delaying Transthyretin Familial Amyloid Polyneuropathy: Systematic Review and Meta-Analysis
Journal of Clinical Neurology ; : 108-115, 2019.
Artículo en Inglés | WPRIM | ID: wpr-719387
ABSTRACT
BACKGROUND AND

PURPOSE:

Tafamidis functions to delay the loss of function in transthyretin familial amyloid polyneuropathy (TTR-FAP), which is a rare inherited amyloidosis with progressive sensorimotor and autonomic polyneuropathy. This systematic literature review and meta-analysis evaluated the efficacy and safety of tafamidis in TTR-FAP patients, with the aim of improving the evidence-based medical evidence of this treatment option for TTP-FAP.

METHODS:

A systematic search of the English-language literature in five databases was performed through to May 31, 2018 by two reviewers who independently extracted data and assessed the risk of bias. We extracted efficacy and safety outcomes and performed a meta-analysis. Statistical tests were performed to check for heterogeneity and publication bias.

RESULTS:

The meta-analysis identified six relevant studies. The tafamidis group showed smaller changes from baseline in the Neuropathy Impairment Score–Lower Limbs [mean difference (MD)=−3.01, 95% confidence interval (CI)=−3.26 to −2.75, p < 0.001] and the Norfolk Quality of Life-Diabetic Neuropathy total quality of life score (MD=−6.67, 95% CI=−9.70 to −3.64, p < 0.001), and a higher modified body mass index (MD=72.45, 95% CI=69.41 to 75.49, p < 0.001), with no significant difference in total adverse events [odds ratio (OR)=0.69, 95% CI=0.35 to 1.35, p=0.27]. The incidence of adverse events did not differ between tafamidis and placebo treatment except for fatigue (OR=0.13, 95% CI=0.02 to 0.72, p=0.02) and hypesthesia (OR=0.16, 95% CI=0.03 to 0.92, p=0.04).

CONCLUSIONS:

This systematic review and meta-analysis has demonstrated that tafamidis delays neurologic progression and preserves a better nutritional status and the quality of life. The rates of adverse events did not differ between the patients in the tafamidis and placebo groups. Tafamidis might be a safer noninvasive option for patients with TTR-FAP.
Asunto(s)

Texto completo: Disponible Índice: WPRIM (Pacífico Occidental) Asunto principal: Polineuropatías / Calidad de Vida / Características de la Población / Prealbúmina / Sesgo / Índice de Masa Corporal / Estado Nutricional / Incidencia / Sesgo de Publicación / Neuropatías Amiloides Tipo de estudio: Ensayo Clínico Controlado / Estudio de incidencia / Estudio pronóstico / Revisiones Sistemáticas Evaluadas Límite: Humanos Idioma: Inglés Revista: Journal of Clinical Neurology Año: 2019 Tipo del documento: Artículo

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Texto completo: Disponible Índice: WPRIM (Pacífico Occidental) Asunto principal: Polineuropatías / Calidad de Vida / Características de la Población / Prealbúmina / Sesgo / Índice de Masa Corporal / Estado Nutricional / Incidencia / Sesgo de Publicación / Neuropatías Amiloides Tipo de estudio: Ensayo Clínico Controlado / Estudio de incidencia / Estudio pronóstico / Revisiones Sistemáticas Evaluadas Límite: Humanos Idioma: Inglés Revista: Journal of Clinical Neurology Año: 2019 Tipo del documento: Artículo