69 Patient-relevant health outcomes: A delphi study 3 INTRODUCTION The group of patients with an autosomal inherited bleeding disorder predominantly consists of patients with von Willebrand disease (VWD), followed by patients with hereditary rare bleeding disorders (RBD) or inherited platelet function disorders (IPFD). These bleeding disorders are caused by different defects in the hemostatic process. These defects lead to heterogeneous bleeding phenotypes which range from mild bleeding, such as easy bruising and epistaxis, to severe or even life-threatening bleeding manifestations as gastrointestinal or intracranial bleeding 1-3. Additionally, women may experience menorrhagia and post-partum hemorrhage 4,5. This heterogeneity in clinical presentation often complicates the diagnosis and treatment of autosomal inherited bleeding disorders and calls for personalized treatment strategies. In recent years, the trend to move healthcare services towards value-based organizations has emphasized the importance of creating value for patients 6-8. Value-based healthcare was introduced by Michael Porter and Elizabeth Teisberg in 2006 with the aim to improve patient value, which is defined as health outcomes that matter to patients divided by the cost of achieving those outcomes 6,9. By focusing on improving outcomes that matter to patients, healthcare services are in theory able to 1) align their care to drive improvement in the health outcomes that matter most to both patients and healthcare professionals, 2) improve how patients experience their health, and 3) reduce the complexity and progression of disease that drive the need for more care 10. The systematic measurement of outcomes that matter to patients in clinical practice is therefore indispensable to improving patient value 6,10. Previous research has focused on determining which health outcomes are important for many different patient populations, including patients with hemophilia 11-13. It is uncertain whether these identified health outcomes that are important for patients for hemophilia are transferable to patients with autosomal inherited bleeding disorders. Whereas the hemophilia population largely consists of men, a large proportion of patients with autosomal inherited bleeding disorders are female, who experience gender-specific health outcomes such as menorrhagia and pregnancy complications 14,15. Therefore, this study aims to assess which health outcomes are important specifically for patients with autosomal inherited bleeding disorders, consisting of von Willebrand disease, platelet function disorders and rare bleeding disorders, from the patient, caregiver and healthcare professional perspectives.
RkJQdWJsaXNoZXIy MTk4NDMw