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Res Pract Thromb Haemost. 2019 Mar 12;3(2):184-192. doi: 10.1002/rth2.12195. eCollection 2019 Apr.

Hemophilia trials in the twenty-first century: Defining patient important outcomes.

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Bloodworks Northwest Seattle Washington.
Department of Medicine University of Washington Seattle Washington.
Institute for Policy Advancement, Ltd. Washington District of Columbia.
Department of Health Resource Methods, Evidence, and Impact McMaster University Hamilton Canada.
Department of Medicine McMaster University Hamilton Canada.


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, so has the science of outcome assessment, including recognition of the importance of patient important and patient reported outcomes. This manuscript reviews strengths and limitations of outcome measures used in hemophilia from both a provider and patient perspective.


bleeding; coagulation factor; gene therapy; hemophilia; outcomes research

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