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Evaluating enzyme replacement therapies for Anderson-Fabry disease: commentary on a recent report.

Roberto GiuglianiStephanie WestwoodHartmann WellhoeferJörn SchenkAndrey GurevichChristoph Kampmann
Published in: Genetics and molecular biology (2018)
Anderson-Fabry disease (AFD) is a rare lysosomal storage disorder. Randomized controlled clinical trials (RCTs) are preferred as the highest category of evidence, but limited availability of robust evidence in rare diseases may necessitate the use of less rigorous evidence. An analysis of cohort studies of enzyme replacement therapies for AFD published in 2017 by El Dib and coworkers made treatment recommendations that contradict previously published findings from RCTs and a systematic Cochrane review. Our commentary outlines concerns regarding selection criteria and statistical methods with their analysis.
Keyphrases
  • clinical trial
  • replacement therapy
  • double blind
  • phase iii
  • randomized controlled trial
  • phase ii
  • placebo controlled
  • clinical practice
  • combination therapy
  • meta analyses