Stakeholder engagement in the development of an upper extremity outcome measure for children with rare musculoskeletal conditions

Caroline Elfassy; Lisa Wagner; Johanne Higgins; Kathleen Montpetit; Laurie Snider; Noémi Dahan-Oliel

doi:10.1186/s40900-023-00479-6

Stakeholder engagement in the development of an upper extremity outcome measure for children with rare musculoskeletal conditions

Res Involv Engagem. 2023 Aug 8;9(1):64. doi: 10.1186/s40900-023-00479-6.

Authors

Caroline Elfassy^{1

2}, Lisa Wagner³, Johanne Higgins⁴, Kathleen Montpetit⁵, Laurie Snider⁶, Noémi Dahan-Oliel^{6

5}

Affiliations

¹ Faculty of Medicine, School of Physical and Occupational Therapy, McGill University, Montreal, Canada. caroline.elfassy@mail.mcgill.ca.
² Clinical Research Department, Shriners Hospital for Children -Canada, 1003 Decarie Boulevard, Montreal, QC, H4A 0A9, Canada. caroline.elfassy@mail.mcgill.ca.
³ Shriners Hospitals for Children, Greenville, USA.
⁴ Université de Montréal, École de Réadaptation, Montreal, Canada.
⁵ Clinical Research Department, Shriners Hospital for Children -Canada, 1003 Decarie Boulevard, Montreal, QC, H4A 0A9, Canada.
⁶ Faculty of Medicine, School of Physical and Occupational Therapy, McGill University, Montreal, Canada.

Abstract

Background: Upper extremity (UE) involvement is prevalent in 73% of individuals with arthrogryposis multiplex congenita (AMC), 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. This study sought to determine the most clinically useful items for an outcome measure 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.

Keywords: Outcome measure development; Rare musculoskeletal condition; Stakeholder engagement; Upper extremity.

Plain language summary

Arthrogryposis multiplex congenita (AMC) is a rare musculoskeletal condition affects the joints and muscles of the body. In about 70% of the cases, it affects the upper extremities (UE). However, there is no specific outcome measure for UE function in children with AMC. An outcome measure refers to a tool or method used to assess and measure the results or effects of a particular treatment, intervention, or condition. It helps healthcare professionals and researchers understand the impact or outcome of a specific situation, such as the level of improvement or changes in a person's health or function. The goal of this study was to develop such a measure while accounting for the perspectives of youth with AMC, their caregivers and clinicians. To achieve this, a study was conducted in two phases. In the first phase, a Nominal Group technique (NGT) was used to gather input from caregivers of children with AMC. Eleven individuals participated and identified 32 items, with the most important being picking up an object, eating, reaching the mouth, and getting out of bed. In the second phase, a three-round survey was sent to 47 experts in the field of AMC, with 20 participants completing the first round, 15 completing the second round, and 13 completing the third round. The survey asked participants about screening UE movement, essential domains to include in the measure, establishing a scoring guide, and identifying tasks associated with joint motion and position. With the help of caregivers’ perspectives and expert opinions, a preliminary version of an UE AMC-specific outcome measure was developed. This measure will be useful in assessing the UE function in children with AMC and will aid clinicians in developing appropriate treatment plans for this rare condition.