Overview of treatments used in transthyretin-related hereditary amyloidosis: a systematic review
| Autor: | Miguel Ángel Martín Vega, Ana Lara Pelayo-Negro, David Gómez Gómez, Héctor Cristóbal Gutiérrez, Marta Valero Domínguez |
|---|---|
| Rok vydání: | 2019 |
| Předmět: |
Tafamidis
medicine.medical_specialty Transthyretin-related hereditary amyloidosis medicine.medical_treatment Cochrane Library Liver transplantation 030226 pharmacology & pharmacy 03 medical and health sciences chemistry.chemical_compound 0302 clinical medicine Internal medicine Post-hoc analysis medicine Humans 030212 general & internal medicine RNA Small Interfering General Pharmacology Toxicology and Pharmaceutics Amyloid Neuropathies Familial Benzoxazoles business.industry Diflunisal medicine.disease Liver Transplantation Clinical trial chemistry Cardiac amyloidosis Observational study Systematic Review business |
| Zdroj: | Eur J Hosp Pharm |
| ISSN: | 2047-9964 2047-9956 |
| DOI: | 10.1136/ejhpharm-2018-001823 |
| Popis: | Objective To carry out a systematic review of the literature to analyse the efficacy and safety of treatments available or under investigation for amyloidosis due to mutations in the transthyretin gene (ATTR). Methods A bibliographic search was carried out in the following electronic databases up to September 2017: PubMed, Cochrane Library and EMBASE. The inclusion criteria were: efficacy and/or safety studies conducted in humans, studies that included treatments, including treatments in the research phase, and studies that included 10 or more patients. Results A total of 21 articles were included; 16 were clinical trials, eight of them (50%) phase III trials, and five were observational studies. Of the total number of studies selected, 11 were on tafamidis, four on diflunisal, two on liver transplantation, two on patisiran and two on other therapeutic alternatives. Of the 11 studies related to the drug, the pivotal trial, the results of its two extension studies and an additional post hoc analysis were selected. In addition, two phase III trials were included in specific populations, two phase II studies, one safety study and two observational studies. Regarding the four included studies related to the drug, one was the pivotal trial that gave the indication to diflunisal, another a safety summary of the pivotal trial, and the other two trials were carried out in specific populations, one in a Japanese population and another phase I trial in cardiac amyloidosis in the USA. As far as other alternatives are concerned, of the six studies included in this section, two were related to liver transplantation, two to patisiran and two to different therapeutic alternatives. Conclusions Sufficient evidence has not been found that demonstrates superiority among the available oral alternatives, diflunisal or tafamidis, in the treatment of ATTR. Direct comparisons between both drugs and pharmacoeconomic studies would be necessary to select the most efficient treatment. |
| Databáze: | OpenAIRE |
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