26 Chapter 2 deficiencies. Severe disorders have a low prevalence and usually present in childhood, but milder forms are relatively more common and may not be clinically apparent until later in life when patients have hemostatic challenges (e.g. menstruation, dental procedures, surgery or trauma). 13 The clinical presentation of different types of bleeding disorders tends to overlap and symptoms range from more common features such as easy bruising, mucocutaneous bleedings and heavy menstrual bleeding to more severe and uncommon symptoms such as joint bleeds, gastrointestinal- and intracranial bleedings 14. Patients with similar diagnoses and comparable laboratory results do not always present with the same bleeding tendency 15-17. This inter-individual variability may complicate identifying the individual patient needs. Moreover, the commonly used measures such as bleeding assessment tools do not always reflect the impact of the disease on a patient’s daily life. Incorporating PROs in clinical practice, may support the physician to focus on patient’s (unidentified) needs, to identify the burden of disease for each individual patient and to monitor the treatment effect. Therefore, the implementation of PROs may lead to more personalized treatment in patients with autosomal inherited bleeding disorder. Until now, research on PROs and PROMs in inherited bleeding disorders has mainly focused on hemophilia 18,19. It is unknown which PROs are important for patients with autosomal inherited bleeding disorders and which PROMs are commonly used to measure PROs in this patient population. This systematic literature review aims to summarize the available literature assessing PROs and their measurement methods to identify which patient-reported outcomes could be important for patients with autosomal inherited bleeding disorders. METHODS Article retrieval This systematic review was registered in PROSPERO (registration number CRD42020199444) and followed the PRISMA methodology for systematic reviews and meta-analysis and the COSMIN methodology for systematic reviews of Patient-Reported Outcomes Measures 20. An information specialist experienced in systematic literature reviews co-designed and conducted the search strategy. The initial search was designed in Embase using a combination of Emtree and non-registered index terms and translated into
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