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Tafamidis, a Noninvasive Therapy for Delaying Transthyretin Familial Amyloid Polyneuropathy: Systematic Review and Meta-Analysis

Yinan Zhao|Yanguo Xin|Zhuyin Song|Zhiyi He|Wenyu Hu
대한신경과학회 (Korean Neurological Association)|2019.01|Journal of Clinical Neurology|제15권 제1호|pp. 108-115|DOI https://doi.org/10.3988/jcn.2019.15.1.108

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키워드

meta-analysissafetyfamilialamyloid neuropathiestherapeutics.

초록

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.

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.

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관련 논문AI추천

Hereditary Transthyretin Amyloidosis with PolyneuropathyDae-Hyun Jang대한근전도전기진단의학회 · 대한근전도 전기진단의학회지2025 · 제27권 제1호Clinical Cues for the Early Diagnosis of Transthyretin-Related PolyneuropathyChristian Geber, Fabiola Escolano-Lozano, Frank Birklein, Panoraia Baka, Violeta Dimova대한신경과학회 · Journal of Clinical Neurology2024 · 제20권 제6호Hereditary Transthyretin Amyloidosis Misdiagnosed as Demyelinating Neuropathy: A Report of Three Cases오지영, 최교민대한근전도전기진단의학회 · 대한근전도 전기진단의학회지2021 · 제23권 제3호Tafamidis for a Transplant Patient with Transthyretin Amyloid PolyneuropathyClementina Del Canto-Pérez, Jesús Romero-Imbroda, Teresa Sagrario-Fustero대한신경과학회 · Journal of Clinical Neurology2017 · 제13권 제4호Characteristics of South Korean Patients with Hereditary Transthyretin Amyloidosis김남희, 김대성, 김병준, 민주홍, 석진명, 성정준, 신하영, 오지영, 이가현, 전은석, 최교민, 최영철, 선우일남대한신경과학회 · Journal of Clinical Neurology2018 · 제14권 제4호Case Report : Non-Val30Met TTR Type Familial Amyloid Polyneuropathy With Asp38Ala Mutation나동욱, 박은숙, 박천웅, 조유나, 최자영대한근전도전기진단의학회 · 대한근전도 전기진단의학회지2016 · 제18권 제1호

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