Shannon van Hoorn

80 Chapter 3 Outcomes at the end of round 3 At the end of the third Delphi round, both panels individually achieved consensus on the importance of 20 health outcomes. Of these 20 health outcomes, six health outcomes are overlapping. In other words, both panels achieved consensus on the importance of six health outcomes: 1) number of bleeding episodes per year that require treatment, 2) total number of lifethreatening bleeding episodes, 3) severity of the bleeding episode, 4) lifethreatening complications, 5) intensity of menstrual bleeding, and 6) impact of menstrual bleeding on daily life (Table 3). Each panel individually achieved consensus on 14 additional health outcomes. The patients and caregivers panel achieved consensus on the importance of health outcomes regarding response to treatment, use of on-demand medication, the impact of menstruation on multiple aspects of daily life, impact of the bleeding disorder on the patients’ emotions, knowledge about the bleeding disorder, and concerns related to the bleeding disorder in general and concerns regarding pregnancy and inheritance of the disease. The healthcare professionals panel achieved consensus on the importance of several health outcomes related to bleeding episodes, treatment side effects including inhibitor development and allergic/ hypersensitivity reactions, the presence and degree of joint damage and the interference of pain on the patients’ daily life. DISCUSSION In this three-round Delphi study, we aimed to assess which health outcomes are important for patients with autosomal inherited bleeding disorders as seen from the patient, caregiver and healthcare professional perspectives. Our study shows both the patient and caregivers, and healthcare professionals panel reached consensus on the importance of six health outcomes for patients with autosomal inherited bleeding disorder, namely: 1) number of bleeding episodes per year that require treatment, 2) total number of life-threatening bleeding episodes, 3) severity of the bleeding episode, 4) life-threatening complications, 5) intensity of menstrual bleeding, and 6) impact of menstrual bleeding on daily life. In addition to these health outcomes, the patients and caregivers panel and the healthcare professionals panel independently identified several other health outcomes that they deem to be important for patients with autosomal inherited bleeding disorders. Patients and caregivers reached consensus on the importance of health outcomes related to menstruation and the impact of

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