Practice guideline for the treatment of familial amyloid polyneuropathy

MARCELINA CARRETERO ; MARIA SOLEDAD SAEZ; MARIA LOURDES POSADAS MARTINEZ; 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

MEDICINA (BUENOS AIRES)

MEDICINA (BUENOS AIRES)

Lugar: Buenos Aires; Año: 2022

0025-7680

SaveEmailSend toDisplay optionsfull text linksfull text provider logoactionsCiteFavoritesshare page navigationTitle & authorsAbstractPublication typesMeSH termsSubstancesSupplementary conceptsRelated informationLinkOut - more resourcesPractice Guideline Medicina (B Aires). 2022;82(2):262-274.[Practice guideline for the treatment of familial amyloid polyneuropathy][Article in Spanish]Marcelina Carretero 1, María S Sáez 2, María L Posadas-Martínez 3, María A Aguirre 4, Patricia Sorroche 2, Agustina Negro 5, Cristian R Calandra 6, Valeria Salutto 7, Andrea Lautre 8, Eugenia Conti 9, Luciana León-Cejas 9, Ricardo Reisin 9, Elsa M Nucifora 10, Marcelo Rugiero 11Affiliations expandPMID: 35417391Free articleAbstract in English, SpanishThis 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).Keywords: GRADE approach; amyloidosis; health planning guideline; recommendations; treatment outcome.