Neurologia · 2026
Redefining therapeutics in vATTR: Evaluation of response predictors to tafamidis and patisiran treatment in a non-endemic area. A proposal for a novel individualised therapeutic approach.
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Martínez-Vicente L, Gajate-García V, Gutiérrez-Gutiérrez G, Guerrero-Peral Á L, Horga A, Guerrero-Sola A, Álvarez-Troncoso J, Restrepo-Córdoba M A, Goirigolzarri-Artaza J, Martín-Albarrán S, Valverde-Megías A, González-López E, García-Pavía P, Matías-Guiu J, Galán-Dávila L
Аннотация
INTRODUCTION: Hereditary transthyretin amyloidosis (vATTR) is a progressive genetic disorder with several approved treatments. We investigated treatment responses to tafamidis and patisiran in vATTR patients to identify predictive response factors.
METHODS: Retrospective analysis on vATTR patients treated with tafamidis or patisiran from October 2012 to September 2022. Treatment responses were assessed as "good," "partial," or "non-response." We analysed pre-treatment clinical and laboratory data to identify predictors of treatment response.
RESULTS: Of the 53 patients, 44 received tafamidis and 23 received patisiran; 14 were treated with both drugs sequentially. Predictors of good response to tafamidis were shorter diagnostic delay (≤1 year), less severe neurological impairment (Coutinho stage 1, Neuropathy Impairment Score [NIS]≤7), and better sudomotor function in the feet (≥50μS) before treatment. Factors associated with non-response were greater disability (baseline Polyneuropathy Disability score=2), large fibre involvement, and significant weight loss. Predictors of a good response to patisiran included lower pre-treatment disease severity (Coutinho stage 1, NIS≤40). We propose an individualised therapeutic approach using predictive factors to guide initial treatment.
CONCLUSIONS: This study identifies predictive factors for response to tafamidis and patisiran in vATTR patients, highlighting baseline NIS as a critical predictor. We propose a novel therapeutic algorithm for personalised treatment strategies with potential to avoid years of ineffective treatment.
METHODS: Retrospective analysis on vATTR patients treated with tafamidis or patisiran from October 2012 to September 2022. Treatment responses were assessed as "good," "partial," or "non-response." We analysed pre-treatment clinical and laboratory data to identify predictors of treatment response.
RESULTS: Of the 53 patients, 44 received tafamidis and 23 received patisiran; 14 were treated with both drugs sequentially. Predictors of good response to tafamidis were shorter diagnostic delay (≤1 year), less severe neurological impairment (Coutinho stage 1, Neuropathy Impairment Score [NIS]≤7), and better sudomotor function in the feet (≥50μS) before treatment. Factors associated with non-response were greater disability (baseline Polyneuropathy Disability score=2), large fibre involvement, and significant weight loss. Predictors of a good response to patisiran included lower pre-treatment disease severity (Coutinho stage 1, NIS≤40). We propose an individualised therapeutic approach using predictive factors to guide initial treatment.
CONCLUSIONS: This study identifies predictive factors for response to tafamidis and patisiran in vATTR patients, highlighting baseline NIS as a critical predictor. We propose a novel therapeutic algorithm for personalised treatment strategies with potential to avoid years of ineffective treatment.