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Chapter 6160checklists to cover relevant disease-specific aspects, as it is not feasible and desirable to have disorder-specific PROMs for all these thousands of disorders.Conclusions The TSC-PROM is the first TSC-specific outcome measure for adults with TSC, which has been developed using the ICF structure covering all relevant aspects of physical functions, mental functions, activities and participation, and social support and with input from individuals with TSC, caregivers, clinicians, as well as literature review and psychometric testing. It appears to have adequate to good psychometric properties of acceptability, reliability, and validity. This TSC-specific PROM provides a unique tool to systematically gain insight into the individuals’ experiences and monitor trial and therapy outcome, taking into account the complexity and heterogeneity of the clinical picture of TSC, and empowering TSC clinicians and researchers in the optimal care for adults with TSC. AcknowledgementsWe would like to acknowledge the individuals with TSC and their representatives who participated in the study and Jo Anne Nakagawa, Eva Schoeters, and Jan-Paul Wagenaar on behalf of the patient organizations from the US (TSC Alliance), Belgium (BeTSC), and the Netherlands (Stichting Tubereuze Sclerosis Nederland), respectively. We also want to thank Molly Griffith and Lauren Davis from Cincinnati Children’s Hospital Medical Center and Le Bonheur Children’s Hospital Memphis who helped with recruitment. Prof. Jan van der Ende is thanked for his methodological contribution to developing the TSC-PROM. Furthermore, we are grateful to the Dutch TSC Foundation (Stichting TSC Fonds) for financially enabling this study. AJ is supported by a Senior Clinical Investigator Fellowship from the Research Foundation Flanders (FWO FKM 1805321N). AvE, LdG, MdW, and LtH are member of European Reference Network (ERN) ITHACA. LdG is affiliated with ENDO-ERN.Annelieke Muller sHL.indd 160 14-11-2023 09:07