PRPP-ASSESSMENT TO MEASURE CHANGE 197 8 Introduction Pediatric health care is shifting to more attention for a child-centered tailor-made approach, also referred to as person-centered care1. In addition, e-health, or telemedicine, has gained momentum with health professionals in pediatric care throughout the past decades2. The COVID-19 pandemic has further accelerated these developments and has served as an impetus for care-at-distance3-5. These developments challenge pediatric care to provide child-centered care-at-distance, also referred to as home-based care. Child-centered home-based care can be beneficial for children with multiple disabilities for many reasons. Firstly, with home-based care the child can practice in his/her own environment. It is known that practicing in the home environment is beneficial when it comes to the long-term effect of therapy: the more similar the context, the better the transfer of learning6, 7. Secondly, it avoids (long) travel times, thereby enhancing accessibility to treatment and lowering the burden on the child and his/her family. Thirdly, home programs can enable a higher frequency of practicing, thus contributing to its effectiveness8, 9. Lastly, home programs with the involvement of parents can increase parents’ knowledge on skill acquisition as well as their self-confidence10. To be able to design tailored care that focuses on activities that matter for the child, and show that this is effective, we should be able to measure, on a personalized level, using instruments that guide and evaluate goal-setting and are responsive to change11, 12. In addition, for home-based care the measurements need to be ecologically valid, which means that the outcome should represent behavior in the ‘real world’13. To be applied to all children with multiple disabilities, an assessment needs to be flexible enough to measure personalized goals with various levels of functioning. In this case series, we included children with a mitochondrial disorder, who can serve as representatives of children with various levels of functioning due to fatigue, speech and language problems, muscle weakness and/or developmental delay14-16. Mitochondrial disorders are disorders in which many organs can be involved. The symptoms can vary with the underlying genetic defects. Some patients have severe cognitive impairment, others have normal cognitive development, but suffer from (cardio) myopathy. Symptoms like epilepsy, dystonia, ataxia, exercise intolerance, deafness and retinopathy can among other symptoms occur14-16. Furthermore, mitochondrial disorder are rare diseases, which makes it difficult to develop evidence-based treatments as research is challenging due to the heterogeneity of the disease and the limited number of individuals who are eligible to participate in any given study17. Also, there is a need for patient-oriented outcome measures within this field of rare diseases18-20.
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