Koert Gooijer

132 This chapter presents the conclusions of this thesis and discusses the key findings in a broader perspective. This thesis focuses on quality of life in Osteogenesis Imperfecta (OI) and bleeding tendency. Both topics are relatively understudied extra-skeletal features of OI. Implications for clinical care and directions for future research are outlined. The two topics are addressed and discussed in two parts, based on the findings of this thesis and the current literature, in line with the research questions. Part 1. Quality of life in Osteogenesis Imperfecta What is the quality of life in people with OI? People with OI rate their perceived general health significantly lower than the control population 1,2, as we showed in Chapter 2. This is in line with other studies investigating general health in OI 3–7. However, perceived general health is not the same as quality of life. Quality of life is a multidimensional concept that captures broad topics such as functional status, well-being and general health assessments 8–10. There are no agreed definitions of what dimensions should be measured when assessing quality of life, but it is well established that quality of life should be assessed by the patients themselves and that it is a multidimensional construct 11. These various dimensions of quality of life, combined with the diversity of the different OI types, present a challenge in finding the right answer to the question “What is quality of life in OI?”. Despite these challenges, we were able to evaluate several aspects of quality of life in comparison to reference populations. Functional status Regardless of the type of OI, adults with OI scored significantly worse than controls on all physical domains of quality of life (Chapter 2). There was a significant difference in physical functioning between the types of OI, with physical limitations and problems being more pronounced in the severe type of OI (type 3). The significantly worse physical domains in OI compared to reference populations are confirmed by other studies on quality of life in adult OI patients 3,5,7,12,13. Functional quality of life includes not only physical functioning, but also role-limitations due to emotional problems and social functioning. Measurements of physical function relate to limitations in daily activities such as climbing stairs, washing and dressing, or lifting groceries, while measuring role-limitations relates to limitations in work and other daily functions, and measures of social function relate to the impact on social activities.

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