81 Patient-relevant health outcomes: A delphi study 3 the bleeding disorder on their daily lives. In contrast, healthcare professionals found health outcomes related to treatment side effects, joint health and pain to be of specific importance for patients with autosomal inherited bleeding disorders. These additional health outcomes identified by the different panels highlight the differences between the patient and caregiver, and healthcare professional perspectives. The results of this study are comparable to previous studies that aimed to identify a set of important health outcomes for patients with hemophilia. While those studies did not make a distinction between the patient, caregiver and healthcare professional perspective, several other studies also identified consensus on the importance of the following health outcomes for patients with hemophilia: frequency of bleeding episodes 19, severity of bleeding episodes 12, complications 12, pain 12,13,19,20, joint health 13, and ability to engage in activities of daily life 12,13,20. None of the studies performed in patients with hemophilia identified the importance of health outcomes related to menstruation. This can be explained by the X-linked inheritance of hemophilia; hemophilia predominantly occurs in males 12. The considerable resemblance, however, between the other health outcomes identified in this study as important for patients with autosomal inherited bleeding disorders and the outcome sets determined for patients with hemophilia, indicates limited differences between health outcomes found to be important for patients with hemophilia and patients with autosomal inherited bleeding disorders. Strengths and limitations This study is the first to assess which health outcomes are important for patients with autosomal inherited bleeding disorders as seen from the patient, caregiver and healthcare professional perspective. By choosing to create two Delphi panels, one with patients and caregivers and one with healthcare professionals, we were able to distinguish potential differences in the importance of health outcomes across the two panels. This study identified that healthcare professionals focus more on disease-related health outcomes that are measurable or influenced by the handling of healthcare professionals, while patients and caregivers focus more on the possible influence of the bleeding disorder on the persons’ emotions and various aspects of their daily lives. This additional insight into the importance of health outcomes from both perspectives provides valuable information to guide the implementation of value-based healthcare in this patient population.
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