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Developing outcome measures of disease activity in pediatric myasthenia
Devin E Prior1, Baillee A Cooper2, Bo Zhang3
1Department of Neurology, Lahey Hospital and Medical Center, Burlington, Massachusetts, USA.
Insights
The Myasthenia Gravis Composite (MGC) and Pediatric Myasthenia-Quality of Life 15 (PM-QOL15) scales reliably assess pediatric myasthenia disease activity in clinics. These tools aid in evaluating treatment response for juvenile myasthenia gravis and congenital myasthenic syndrome.
Area of Science:
- Neurology
- Pediatrics
- Clinical Assessment
Background:
- Pediatric myasthenia, including juvenile myasthenia gravis (JMG) and congenital myasthenic syndrome (CMS), presents challenges in assessing disease activity and treatment response.
- Unlike adult populations, validated outcome measures for pediatric myasthenia have been lacking, hindering clinical evaluation.
Purpose of the Study:
- To evaluate the validity and reliability of the Myasthenia Gravis Composite (MGC) and Pediatric Myasthenia-Quality of Life 15 (PM-QOL15) scales as outcome measures in pediatric myasthenia.
- To establish these scales as practical tools for assessing disease activity in a clinical setting for children with myasthenia.
Main Methods:
- Prospective study of 33 pediatric patients (0-18 years) with JMG or CMS over a 3-year period.
- Data collection included demographic information, diagnosis, Myasthenia Gravis Foundation of America (MGFA) class, MGC scores, and PM-QOL15 scores at routine clinical visits.
- Statistical analyses were performed to establish known-groups validity, concurrent validity (using receiver-operating characteristic curves), and concordance (Pearson and Spearman correlations).
Main Results:
- The MGC and PM-QOL15 scales demonstrated known-groups validity when compared to the MGFA class.
- Concurrent validity was established, with optimal thresholds identified for MGC and PM-QOL15 to detect more severe disease (MGFA class III or higher).
- A statistically significant positive correlation was found between MGC and PM-QOL15 scores, indicating concordance between the two measures.
Conclusions:
- The MGC and PM-QOL15 are valuable, easily administered tools for reliably assessing pediatric myasthenia disease activity in clinical practice.
- These scales can aid clinicians in monitoring disease progression and treatment effectiveness in children with myasthenia.
- Further research is recommended to validate these measures for use in pediatric clinical trials.
Introduction:
Pediatric myasthenia encompasses juvenile myasthenia gravis (JMG) and congenital myasthenic syndrome (CMS), which are chronic disorders with fluctuating symptoms amenable to medical therapy. Disease activity and treatment response may be difficult to assess, but, unlike adults, outcome measures have not been developed in children.
Methods:
The study was performed in children (0-18 years of age) at the neuromuscular center of a pediatric hospital over a 3-year period. Patients were recruited prospectively as part of their routine clinical care. Demographic data, diagnosis (JMG/CMS), and the following scales were recorded at each visit: Myasthenia Gravis Foundation of America (MGFA) class, Myasthenia Gravis Composite (MGC), and Pediatric Myasthenia-Quality of Life 15 (PM-QOL15).
Results:
Thirty-three patients (24 JMG, 9 CMS) were included in the study, 22 had two or more visits. We established known-groups validity of the MGC and PM-QOL15 scores as compared with the MGFA class. To establish concurrent validity, we constructed a receiver-operating characteristic curve and calculated threshold values of MGC and PM-QOL15 with optimal sensitivity and specificity for identifying a patient with more severe (MGFA III or higher) disease. Finally, we demonstrated the concordance between the MGC and PM-QOL15 by their statistically significant positive Pearson and Spearman correlations.
Discussion:
Our study suggests that MGC and PM-QOL15 are important disease outcome measures in pediatric myasthenia that are easy to administer and provide reliable assessment of disease activity in the clinic setting. Further studies are needed to validate their use for pediatric clinical research trials.
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