Author Of 2 Presentations
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.
P0424 - A Longitudinal Analysis of Functional Outcomes among Young People with MS Using Group-Based Trajectory Modelling (ID 750)
Abstract
Background
Studies on the natural history of MS can provide important and valid information on the long-term outcomes. Understanding cumulative disability caused by the neurological deficits will allow patients and clinicians to make objective assessments of prognosis. In light of the evidence of a MS prodome, our a-priori hypothesis was that the natural history of MS disability progression would be affected by an earlier age of onset of MS.
Objectives
To identify longitudinal patterns of disability progression among young people who are eligible for MS drug therapies and to estimate the extent to which two-year disability progression among those aged 25 and under differs from those aged between 26 and 35 years, where disability progression is defined by changes in the functional tests included in the Multiple Sclerosis Functional Composite (MSFC).
Methods
This study used pooled data from the placebo arms of clinical drug trials compiled by the Critical Path Institute. The longitudinal patterns on the four performance measures were identified using group-based trajectory models (GBTM). People with pediatric-onset MS were identified in each trajectory model. Linear mixed models were used to estimate the average change over time, assuming random intercepts and slopes and fixed effects of age (≤25 years and 26 to 35 years) and sex. To identify the difference between the expected and observed proportions of people with pediatric-onset MS in each trajectory, the chi-square statistic (χ2) was used.
Results
The analysis included 676 people, 174 (25.7%) below 25 years of age. GBTM showed a wide range of performance ability across outcomes but little variability over time. Results of mixed models showed that younger people (18 to 25 years) performed better over time for gait speed (β =0.04, t=2.6), dexterity (β =-1.05, t=-4.5; β =-0.96, t=-4.0 for dominant and non-dominant hands respectively), and the PASAT (β =1.39, t=3.9). Men performed poorer than women on tests of dexterity (dominant: β =2.42, t=11.0; non-dominant: β =1.21, t=-5.3), and the SDMT (β =-3.09, t=-3.0) but not on gait speed (β =0.20, t=-13.3). The effect of relapses was observed on dexterity (dominant: β =1.08, t=5.3; non-dominant: β =1.2, t=5.7), and the SDMT (β =-3.20, t=-3.4). Distribution of the 31 people with pediatric onset differed from expected on the test of dexterity (non-dominant hand) (χ2 = 20.3, p<0.001), cognition (χ2 = 10.5, p<0.025), and the EDSS (χ2 = 21.5, p<0.001) with more people represented in the more disabled group.
Conclusions
People who have an earlier onset of MS were observed to have a different disability profile from a slightly older group. The combined use of trajectory models and linear mixed models provided rich information about the variability in function over time and the effects of age and sex.
Presenter Of 2 Presentations
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.
P0424 - A Longitudinal Analysis of Functional Outcomes among Young People with MS Using Group-Based Trajectory Modelling (ID 750)
Abstract
Background
Studies on the natural history of MS can provide important and valid information on the long-term outcomes. Understanding cumulative disability caused by the neurological deficits will allow patients and clinicians to make objective assessments of prognosis. In light of the evidence of a MS prodome, our a-priori hypothesis was that the natural history of MS disability progression would be affected by an earlier age of onset of MS.
Objectives
To identify longitudinal patterns of disability progression among young people who are eligible for MS drug therapies and to estimate the extent to which two-year disability progression among those aged 25 and under differs from those aged between 26 and 35 years, where disability progression is defined by changes in the functional tests included in the Multiple Sclerosis Functional Composite (MSFC).
Methods
This study used pooled data from the placebo arms of clinical drug trials compiled by the Critical Path Institute. The longitudinal patterns on the four performance measures were identified using group-based trajectory models (GBTM). People with pediatric-onset MS were identified in each trajectory model. Linear mixed models were used to estimate the average change over time, assuming random intercepts and slopes and fixed effects of age (≤25 years and 26 to 35 years) and sex. To identify the difference between the expected and observed proportions of people with pediatric-onset MS in each trajectory, the chi-square statistic (χ2) was used.
Results
The analysis included 676 people, 174 (25.7%) below 25 years of age. GBTM showed a wide range of performance ability across outcomes but little variability over time. Results of mixed models showed that younger people (18 to 25 years) performed better over time for gait speed (β =0.04, t=2.6), dexterity (β =-1.05, t=-4.5; β =-0.96, t=-4.0 for dominant and non-dominant hands respectively), and the PASAT (β =1.39, t=3.9). Men performed poorer than women on tests of dexterity (dominant: β =2.42, t=11.0; non-dominant: β =1.21, t=-5.3), and the SDMT (β =-3.09, t=-3.0) but not on gait speed (β =0.20, t=-13.3). The effect of relapses was observed on dexterity (dominant: β =1.08, t=5.3; non-dominant: β =1.2, t=5.7), and the SDMT (β =-3.20, t=-3.4). Distribution of the 31 people with pediatric onset differed from expected on the test of dexterity (non-dominant hand) (χ2 = 20.3, p<0.001), cognition (χ2 = 10.5, p<0.025), and the EDSS (χ2 = 21.5, p<0.001) with more people represented in the more disabled group.
Conclusions
People who have an earlier onset of MS were observed to have a different disability profile from a slightly older group. The combined use of trajectory models and linear mixed models provided rich information about the variability in function over time and the effects of age and sex.