114 Chapter 4 Various PROMIS measures have been recently validated in (young) adults with hemophilia 8-10. The validation of PROMIS item banks by adults with hemophilia shows that the PROMIS Profile-29 questionnaire, the 8-item PROMIS anxiety and depression short forms, as well as the PROMIS CATs physical functioning, pain interference, satisfaction with social roles and activities, and fatigue are feasible, reliable and valid alternatives to legacy instruments 8-10. Studies to validate the use of PROMIS CATs in children with hemophilia, in children and adults with other inherited bleeding disorders such as von Willebrand disease, platelet function disorders and the rare bleeding disorders are currently ongoing (Figure 1). These latter studies are currently being performed in collaboration with the SYMPHONY NWO-NWA research consortium. We expect that the ongoing studies will further highlight the advantages of PROMIS. Subsequently, implementation of PROMIS measures may be more feasible within the field of inherited bleeding disorders. These PROMIS sets will need to be enriched with disease specific questions, as these cannot be completely replaced. The joint commitment to PROMIS by Dutch healthcare professionals involved in inherited bleeding disorders within the hemophilia treatment centers, will not only facilitate the implementation of outcome measurement in overall care for persons with inherited bleeding disorders (inter)nationally, but is also a stepping stone for healthcare innovations in rare diseases at large. To achieve all advantages PROMIS has to offer to the field of inherited bleeding disorders, PROMIS needs to become the national and international standard for generic outcome measurement by persons with inherited bleeding disorders.
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