Stakeholder Engagement in the Development of an Upper Extremity Outcome Measure for Children with Rare Musculoskeletal Conditions

Abstract

Abstract Background Individuals with arthrogryposis multiplex congenita (AMC) have upper extremity (UE) involvement in 73% of cases, yet no AMC specific outcome measure exists. When developing a measure specific to a population with a rare musculoskeletal condition, clinicians’ and patients’ perspectives and involvement is a crucial and necessary step. Therefore, this study determines the most clinically useful items for an outcome measures of UE function for children with AMC as defined by caregivers and clinicians.Methods To ensure the perspectives and needs of caregivers of children with AMC and clinicians were considered in the development of the UE measure for AMC, a Nominal Group technique (NGT) with caregivers of children with AMC (phase 1) followed by a three-round survey with clinicians (phase 2) were carried out.Results Phase 1: Eleven individuals participated in the nominal group technique and identified 32 items. The most important items were Picking up an object (n = 11), Eating (n = 10), Reaching mouth (n = 10), Getting out of bed (n = 10). Phase 2: Invitations to participate to an online survey was sent to 47 experts in the field of AMC, 20 participants completed round 1, 15 completed round 2 and 13 completed round 3. Throughout the survey, participants were asked about movement required to screen the UE, essential domains to be included in the measure, establishing a scoring guide and identifying tasks associated with joint motion and position.Conclusion A preliminary version of an UE AMC-specific outcome measure was developed with the help of caregivers’ perspectives and expert opinions.