[Practice guideline for the treatment of familial amyloid polyneuropathy].

Abstract

This clinical practice guideline for the treatment of familial amyloid polyneuropathy is based on the best available evidence of clinical effectiveness. A list of questions was generated with a PICO format focused on the effectiveness and safety of the treatment of familial amyloid polyneuropathy. The search was carried out in PubMed, Cochrane and Epistemonikos. The levels of evidence and grades of recommendation were based on the GRADE system. Recommendations were graded according to their direction and their strength and were evaluated with the GLIA tool for their implementation. In patients with familial amyloid polyneuropathy and stage I and II neuropathy, it is suggested: inotersen 300 mg subcutaneous weekly or patisirán 0.3 mg/kg intravenously once every 3 weeks, since they probably stabilize or slow the progression of neuropathy and worsening quality of life (moderate quality of evidence; strength of recommendation weak). In patients with familial amyloid polyneuropathy and stage I neuropathy, treatment with tafamidis 20 mg orally, once a day, is suggested, as it could slow the progression of neuropathy and worsen quality of life (low quality of evidence; strength of recommendation weak). In patients with familial amyloid polyneuropathy and symptomatic neuropathy and in the absence of other treatments with approved efficacy, treatment with oral diflunisal 250 mg twice daily is suggested, as it could prevent the progression of neuropathy (quality evidence low; strength of recommendation weak).

• Authors: Marcelina Carretero, María S Sáez, María L Posadas-Martínez, María A Aguirre, Patricia Sorroche, Agustina Negro, Cristian R Calandra, Valeria Salutto, Andrea Lautre, Eugenia Conti, Luciana León-Cejas, Ricardo Reisin, Elsa M Nucifora, Marcelo Rugiero
• Journal: Medicina (Medicina (B Aires)) Vol. 82 Issue 2 Pg. 262-274 ( 2022) ISSN: 1669-9106 [Electronic] Argentina
• Vernacular Title: Guía de práctica clínica de tratamiento de la polineuropatía amiloidótica familiar.
• PMID: 35417391 (Publication Type: Practice Guideline, Journal Article)

Chemical References

• Benzoxazoles
• Prealbumin

Topics

• Amyloid Neuropathies, Familial (drug therapy)
• Benzoxazoles (therapeutic use)
• Humans
• Prealbumin (therapeutic use)
• Quality of Life
• Treatment Outcome