Hemophilia trials in the twenty-first century: Defining patient important outcomes.
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ABSTRACT:
SUBMITTER: Konkle BA
PROVIDER: S-EPMC6462740 | biostudies-literature | 2019 Apr
REPOSITORIES: biostudies-literature
Publications
Hemophilia trials in the twenty-first century: Defining patient important outcomes.
Konkle Barbara A BA Skinner Mark M Iorio Alfonso A
Research and practice in thrombosis and haemostasis 20190312 2
Treatment for hemophilia has advanced dramatically over the past 5 decades. Success of prophylactic therapy in preventing bleeding and decreasing associated complications has established a new standard of care. However, with the advent of gene therapy and treatments that effectively mimic sustained coagulation factor replacement, outcome measures that worked well for assessing factor replacement therapies in past clinical trials need to be reassessed. In addition, while therapies have advanced, ...[more]
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