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Introduction: One of the recent advances in assessing outcomes of medical care is the inclusion of the patient perspective. The term patient-reported outcome (PRO) is used to reflect the patient perceptions of disease and its consequences as well as of treatment and health-care provision. The development of PRO measures has advanced rapidly, and implementation in clinical research and practice is now underway. From an evaluation perspective, recommendations for the choice of PRO measures and an appraisal of the potential benefits of PRO data collection within specific health conditions are needed. Methods: Hemophilia is a rare and clinically well-defined health condition with established and cost-intensive treatment strategies, in which PRO assessment is increasingly recognized as important. For this reason, measurement of PROs in hemophilia focusing on health-related quality of life (HRQoL) and patient preferences were reviewed to identify appropriate measures, to make recommendations for their choice, and to critically examine their impact in international hemophilia research and practice. Results: Using literature searches and expert discussion strategies, generic and targeted measures for HRQoL and patient preferences in adults and children with hemophilia were screened, and 20 were reviewed on the basis of their psychometric properties and international availability. Only a few of the 20 measures have been used in clinical settings or research related to persons with hemophilia. Conclusion: Consequently, an increased use of these measures is recommended to understand patient views on disease and treatment and to judge the impact of PROs for improvements in health care.