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

{"article_title":"[Practice guideline for the treatment of familial amyloid polyneuropathy].","author":"Carretero M, S\u00e1ez MS, Posadas-Mart\u00ednez ML, Aguirre MA, Sorroche P, Negro A, Calandra CR, Salutto V, Lautre A, Conti E, Le\u00f3n-Cejas L, Reisin R, Nucifora EM, Rugiero M","journal_title":"Medicina","issn":"1669-9106","isbn":"","publication_date":"2020 Feb 07","volume":"82","issue":"2","first_page":"262","page_count":"","accession_number":"35417391","doi":"","publisher":"Fundacio\u0301n Revista Medicina (Buenos Aires)","doctype":"Practice Guideline","subjects":"Amyloid Neuropathies, Familial drug therapy; Benzoxazoles therapeutic use; Humans; Prealbumin therapeutic use; Quality of Life; Treatment Outcome","interest_area":["Urology & Nephrology"," Internal Medicine"," Emergency Medicine"],"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 patisir\u00e1n 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).","url":"https:\/\/search.ebscohost.com\/login.aspx?direct=true&db=mdl&AN=35417391&authtype=shib&custid=ns346513&group=main&profile=eds","isPdfLink":true,"isSAML":false,"additionalInfo":{"Authored_By":"Carretero M, S\u00e1ez MS, Posadas-Mart\u00ednez ML, Aguirre MA, Sorroche P, Negro A, Calandra CR, Salutto V, Lautre A, Conti E, Le\u00f3n-Cejas L, Reisin R, Nucifora EM, Rugiero M","Journal_Info":"Publisher: Fundacio\u0301n Revista Medicina (Buenos Aires) Country of Publication: Argentina NLM ID: 0204271 Publication Model: Print Cited Medium: Internet ISSN: 1669-9106 (Electronic) Linking ISSN: 00257680 NLM ISO Abbreviation: Medicina (B Aires) Subsets: MEDLINE","Publication_Type":"Practice Guideline; Journal Article","Published_Date":"2022-01-01","Source":"Medicina [Medicina (B Aires)] 2022; Vol. 82 (2), pp. 262-274.","Languages":"Spanish; Castilian","Electronic_ISSN":"1669-9106","MeSH_Terms":"Amyloid Neuropathies, Familial*\/drug therapy, Benzoxazoles\/therapeutic use ; Humans ; Prealbumin\/therapeutic use ; Quality of Life ; Treatment Outcome","Subjects":"Benzoxazoles therapeutic use, Humans, Prealbumin therapeutic use, Quality of Life, Treatment Outcome, Amyloid Neuropathies, Familial drug therapy","Title_Abbreviations":"Medicina","Volume":"82"},"header":{"DbId":"mdl","DbLabel":"MEDLINE Ultimate","An":"35417391","RelevancyScore":"907","PubType":"Academic Journal","PubTypeId":"academicJournal","PreciseRelevancyScore":"906.698547363281"},"plink":"https:\/\/search.ebscohost.com\/login.aspx?direct=true&site=eds-live&db=mdl&AN=35417391&authtype=shib&custid=ns346513&group=main&profile=eds","upload_link":"https:\/\/search.ebscohost.com\/login.aspx?direct=true&site=eds-live&db=mdl&AN=35417391&authtype=shib&custid=ns346513&group=main&profile=eds"}