14 Chapter 1 AIM AND OUTLINE OF THIS THESIS The overall aim of this thesis is to study the added value of the implementation of VBHC, as defined within the Dutch healthcare context, in routine clinical care for patients with an inherited bleeding disorder. To achieve this aim, this thesis intends to address the following research questions: 1. Which health outcomes, including clinical and patient-reported outcomes, are important for patients with von Willebrand disease, inherited platelet function disorders, and rare bleeding disorders? 2. Which patient-reported outcome measures are suitable to measure patient-reported outcomes in patients with von Willebrand disease, inherited platelet function disorders, and rare bleeding disorders? 3. How do patients experience the implementation of patient-reported outcome measures in routine clinical care? 4. Are there meaningful differences in hemophilia care provided by the hemophilia treatment centers in the Netherlands that can be used to improve quality of care? Part I: Measuring patient-reported outcomes Part I focuses on identifying which health outcomes are important to measure by patients with von Willebrand disease, inherited platelet function disorder and rare bleeding disorders and on how to measure them. Part I therefore answers research questions 1 and 2. Chapter 2 provides a concise overview of which health outcomes might be impacted in patients with von Willebrand disease, inherited platelet function disorders and rare bleeding disorders as found in previous studies. Chapter 3 aims to identify which health outcomes are relevant for this patient population as seen from the patient, caregiver and healthcare professional perspectives. Chapter 4 introduces and describes the possible advantages of using a specific PROM measurement system, called Patient Reported Outcomes Measurement Information System (PROMIS) to measure the identified relevant patient-reported outcomes. In Chapter 5, we aim to identify if the PROMIS instruments are suitable to assess patientreported outcomes in patients with von Willebrand disease, inherited platelet function disorders and rare bleeding disorders. Part II: Patient experiences with patient-reported outcomes Part II investigates how patients experience the collection of outcome information in routine clinical care and answers research questions 3. Chapter 6 describes the experiences of HIV patients with the implementation of three
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