Efficacy and safety of migalastat, an oral pharmacologic chaperone for Fabry disease: results from two randomized phase 3 studies, FACETS and ATTRACT

Molecular Genetics and Metabolism(2017)

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摘要
Infection by cytomegalovirus (CMV) is a major cause of morbidity among immunosuppressed patients, especially after solid organ transplantation. The risk of CMV after organ transplantation is strongly related to the serology of the donor and the recipient. The objective of this study was to analyze the outcomes and costs of pre-emptive therapy in patients after liver transplantation with donor-positive/recipient-negative (D+/R−) serostatus.This retrospective study analyzed all patients who underwent liver transplantation with CMV serostatus D+/R− between January 2012 and December 2015. The service protocol adopts pre-emptive therapy. The outcomes and costs of this therapy are described.Of the 119 patients undergoing liver transplantation, 19 were D+/R− and entered the main analysis. Of these, 7 had positive polymerase chain reaction (PCR) results, and 1 developed CMV disease. Of the 6 patients who received no treatment, none developed CMV disease. Analyzing costs, pre-emptive therapy for these patients generated service savings of R$32,346.00.Although outcomes of universal prophylaxis and pre-emptive therapy are similar, pre-emptive therapy save on costs and have to be considered in patients with high-risk CMV disease after liver transplantation.
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fabry disease,oral pharmacologic chaperone,migalastat
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