Portal de investigación
Real life experience of tafamidis for the treatment of Spanish patients with Val30Met transthyretin amyloidosis with polyneuropathy
Autores de IIS La Fe
Participantes ajenos a IIS La Fe
- Sanso, MAR
- Rodriguez, AR
- Vicente, LM
- Garro, CB
- Martín, JF
- Vicente, AA
- de la Prida, MM
- Dávila, LG
- Vázquez, LG
- Valle, FM
- Pons, CC
- Bau, AF
- Barroso, EC
- López, IL
- Gonzalez-Moreno, J
Grupos
Abstract
Introduction: Tafamidis 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. Methods: This 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. Results: 100 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 nonresponder with a 60% probability. Conclusions: Our 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.
Datos de la publicación
- ISSN/ISSNe:
- 0025-7753, 1578-8989
- Tipo:
- Article
- Páginas:
- 27-32
- PubMed:
- 38556397
- Factor de Impacto:
- 0,325 SCImago ℠
- Cuartil:
- Q3 SCImago ℠
MEDICINA CLINICA ELSEVIER ESPANA SLU
Citas Recibidas en Web of Science: 1
Documentos
- No hay documentos
Filiaciones
Keywords
- Transthyretin amyloidosis; ATTR; Polyneuropathy; Tafamidis; Real-life
Campos de Estudio
Cita
Sanso M,Rodriguez AR,Vicente LM,SEVILLA T,Garro CB,Martín JF,Vicente AA,de la Prida MM,Dávila LG,Vázquez LG,Valle FM,Pons CC,Bau AF,Barroso EC,López IL,Gonzalez J. Real life experience of tafamidis for the treatment of Spanish patients with Val30Met transthyretin amyloidosis with polyneuropathy. Med. Clin. 2024. 162. (9):p. 27-32. IF:2,100. (2).