• Medicina clinica · May 2024

    Multicenter Study

    Real life experience of tafamidis for the treatment of Spanish patients with Val30Met transthyretin amyloidosis with polyneuropathy.

    • Maria Antonia Ribot Sanso, Adrián Rodriguez Rodriguez, Laura Martínez Vicente, Teresa Sevilla, Cristina Borrachero Garro, Julian Fernández Martín, Adrián Antón Vicente, Moises Morales de la Prida, Lucía Galán Dávila, Laura González Vázquez, Ferran Martínez Valle, Carlos Casasnovas Pons, Arturo Fraga Bau, Eugenia Cisneros Barroso, Inés Losada López, and Juan González-Moreno.
    • Servicio de Medicina Interna, Unidad Amiloidosis por Trastirretina, Hospital Universitario Son Llàtzer, Instituto de Investigación Sanitaria Illes Balears (idISBA), Palma de Mallorca, Spain.
    • Med Clin (Barc). 2024 May 17; 162 (9): e27e32e27-e32.

    IntroductionTafamidis is the only approved transthyretin stabiliser approved for the treatment of variant transthyretin amyloidosis (A-ATTRv) related polyneuropathy (PNP). The aim of this study is to analyse the effectiveness of tafamidis in a real-world setting in Spain.MethodsThis is a national multicenter study in which patients with V30M A-ATTR related PN treated with tafamidis for at least 1 year were included. Clinical, demographic, analytical and neurophysiological variables were analysed.Results100 patients were recruited. Overall, 47 patients (47%) were classified as complete responders, 32 (32%) as partial responders and 21 (21%) as non-responders. The median duration of treatment with tafamidis was 35 months. Better treatment response was shown in patients with in polyneuropathy disability score (PND) I, lower neuropathy impairment score (NIS), compound muscle action potential (CMAP) and Norfolk QoL questionnaire. Higher albumin levels and lower NTproBNP levels were also associated with better treatment response. A basal NIS≥15 predicts that the patient could be a non-responder with a 60% probability.ConclusionsOur results reinforce the tafamidis efficacy to treat A-ATTRv-PNP if started early in the disease course. Patients with the V30M variant, NIS<15 and PND I are the most appropriate subjects for this treatment.Copyright © 2024 Elsevier España, S.L.U. All rights reserved.

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