Author Of 1 Presentation
P0130 - Pediatric MS: different disease course, different impact, different measurement approach needed (ID 972)
Abstract
Background
To understand the impact of pediatric mulitple sclerosis (MS), generic health related quality of life (HRQL) measures have been used. However, generic measures do not always reflect the relevant domains of health that are affected by MS. Specific areas of concern of youths with MS are not known and are assumed to be the same as parents, clinicians, or children with other disabilities or children in general.
Objectives
The objectives of this study were to identify the domains of life that are important to youth with pediatric MS and to contribute evidence towards the development of a condition-specific measure for children and adolescents with MS.
Methods
In conjunction with patient partners, an online survey for youths with MS and parents was developed based on the Patient Generated Index (PGI). Text threads generated by the PGI were mapped onto the International Classification of Functioning, Disability and Health (ICF) and the Comprehensive ICF Core Set for MS.
Results
A total of 19 people completed the PGI survey of which 10 were youths with MS aged 14 to 22 years with a median onset age of 13.5 years. Over 80% of the areas nominated by youths with MS related to activities and participation and only 20% related to impairments. In contrast, 62% of the areas nominated by parents related to impairments. This input indicated that a measure of HRQL would need to include both the impairments associated with MS that need to be targeted with therapies and the activities and roles that are important that need to be encouraged and celebrated. The new approach has a disability component covers MS related impairments and is completed using the PGI system where the youth (or parent for young children) selects 5 disability areas affected by MS, rate severity, and prioritize each area for improvement. The “quality of life” component, to be completed only by the youth, queries those areas that are going well.
Conclusions
In a rare disease like pediatric MS, progress is slow in this area as condition-specific measures are not available. The rarity of the conditions makes it difficult to adhere to best practice guidelines developing measures for these unique populations. The use of this new measurement approach could prove useful in overcoming challenges of measurement development for children with rare diseases.