1. Vincent P. GibbonsHSR 530-01Intro to Outcomes Research Fall, 2011

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Objectives1. list the possible uses for a tool to measure pediatric seizure burden2. review the list of available tools to measure pediatric seizure burden3. review the steps used to validate new screening questionnaires 3. purpose to quantitate the burdens (personal and family) that stemfrom epilepsy not just numbers of seizures, but all aspects of the health problem of epilepsy to emphasize those burdens that are susceptible to changewith medical treatment to track changes in burden to identify tipping points that require intervention or change in therapy to measure the effectiveness of therapies and interventions 4. domains of burden uncontrolled seizures stigma, limitations injuries family-initiated medications self-initiated sword of Damocles school initiated epilepsy comorbidities employer initiated society initiated depression anxiety neurologic deficits attention problems cognitive learning problems motor 5. burden: seizures postictal injuries depression embarrassmentmed sideactivity effects suddenrestrictionsdeath 6. burden:comorbiditiessleepanxietydepression learningattention disabilitiesdisorder 7. burden:stigma, limitationssports drivingschool societycareersocial choices activities family 8. burden: neurologic disability Some patients have idiopathic epilepsy no recognized additional, recognizable neurologic diagnosis much of this is genetic still at risk for epilepsy comorbidities (q.v.) Some patients have epilepsy as a consequence of, or inassociation with, additional neurologic diagnoses Some of this is genetic (e.g. inborn errors of metabolism) Some of this is remote symptomatic, i.e the consequence of a past event that is done and gone perinatal injury, CNS infections, etc. Some of this is progressive e.g. juvenile Huntingtons disease Some of this is unknown or uncharacterized e.g. cryptogenic infantile spasms These neurologic disabilities are associated with a spectrum ofburdens over and above the burdens attributable to epilepsyitself. 9. burden: neurologic disabilitymotor financialcognitivediseasetempo independence 10. Isnt this just a form of QOLIE? focus is slightly different some burdens dont affect quality of life, but are worth recognizing, measuring, and tracking not all burdens are quality of life burdens emphasis on decision making, prioritizing Is it time to make a change in anticonvulsants? Is it time to emphasize family/school education? At a time of transition, what is our to do list based on ameliorating burdens? How much has treatment A relieved burdens? Is treatment A better than treatment B? 11. What should be the content of the measure? Possibly batteries of specific measures, since interventions and therapychanges will be made in each of the domains separately Well be interested in measuring points in time as well as changes overtime (point in time) Our neurology clinic sees 3000 patients with epilepsy. We anticipate that the epilepsy clinic can accommodate 300 patients, and we want to use the epilepsy burden score to help decide which of the 3000 patients is appropriate for the epilepsy clinic. Where should we set the bar for inclusion to the epilepsy clinic? For invitation for a one- time epilepsy consult and return to the general neurology pool? (changes over time) Johnnys school has been the target over 6 months of intensive education to combat what was perceived as inappropriate limitations in the classroom, cafeteria, and playground. Have the interventions and education been successful? 12. How about a dashboard to summarize and track the components of current burden? seizures & meds comorbidities stigma & limits neuro statustime 1time 2time 3 13. Whats the process of developing and validating such a patient (proxy) tool? Try to avoid re-inventing the wheel. Will a literature search disclose an already-validated tool? What do you want to measure? Will patients and families agree that your tool measures the purported characteristics? Will your tool measure accurately and succinctly whatyou think its measuring? Are the results stable from subject to subject, and from one time to another? Is the tool sufficiently compact and user-friendly? 14. pick domains of interest There may be only one domain. and there may be subdivisions within that domain A Bepilepsy comorbidities affective sleep socialization Cstigma societal school family D 15. face validity:How does this tool accord with currentunderstanding of the measured entity? literature search Are there similar tools already validated? consultations with experts in the field patient and family interviews Make sure the group composition matches theexpected composition of the intended users. 16. construct validity sampling frame of test subjects representative subjects compare responders to nonresponders to make sure the responders continue to be representative convergent validity (new test II correlates to some extent withalready validated test I) roughly similar domains between tests should correlate discriminant validity (domain A measures a different qualitythan domain B) different domains should show appropriately different correlations check ceiling and floor effects, skew internal consistency reliability (Cronbachs ) 17. ceiling effect floor effectgroup responses group responsesskewgroup responses 18. Brevity, Family Focus 19. IMPACT OF PEDIATRIC EPILEPSY SCALE 20. QOLCEQUALITY OF LIFE IN CHILDHOOD EPILEPSYDomains Populations and Use Child Seizure Profile The test was validated using seizure characteristics guardian responses frequency for children 4-18 years old perceived severity with refractory epilepsy (this is medication characteristics thought to encompass perhaps Child Epilepsy Questionnaire 21% of children with epilepsy).Parent (178 items) Children who had had epilepsy physical functioning surgery, and children with emotional well-being visual or hearing impairments cognitive functioningor neurodegenerative disease social functioning were excluded. behavior 20 minutes to complete 21. How about use to monitor change over time?How about extending testing to children with resective surgery? Is it true that you need to be seizure-free after resection to claim QOL benefit? Is it true that the greatest improvements in QOL occur when the resection is done at a young age? 22. consecutive series of 35 children 6-18 y/o undergoing resective surgery post op seizure control 20 seizure-free 4 seizures reduced 8 unchanged 3 increased seizures QOLCE at baseline and 12-18 months post-op 23. What factors predicted change in QOL? predictivenot predictive post-op seizure freedom age at epilepsy onset post-op AED count (?) age at resection baseline QOL score gender intellectual ability extent of resection laterality histopathology 24. What if we look at measuring burden in childrenwith benign centrotemporal epilepsy?What do you think? The main determinants of quality of life in thesechildren include a) seizure frequency b) AED side effects c) injuries during seizures d) episodes of status epilepticus e) none of the above 25. Pediatr Neurol 2006;35:240-245In benign rolandic epilepsy the prognosis for seizure outcome is excellent. Because ofthe benign nature of the epilepsy, it is generally presumed that quality of life is notsignificantly affected. As such, there is a paucity of research in this area. 26. Study designResults Initial recruitment was through the major EEG Clinical variables had minimal impact .laboratories of metropolitan Sydney. There was a higher incidence of The syndrome was defined using the ILAE competence problems compared withclassification. normative data. Patients underwent a comprehensive The average psychosocial score wascognitive assessment. significantly lower than normative data. WISC III, WRAML, WIATS, CELF-3 Controlling for parental emotional impact, Parents completed general intellectual ability predicted quality the Child Health Questionnaireof life in the areas of self-esteem andlanguage. Parental emotional impact was assessedusing a subscale. Parental emotional impact, however, was a Child Behavior Checklistmajor independent predictor of quality oflife. Quality of Life in Childhood EpilepsyQuestionnaire. Quality of life is related to The cohort included 30 patients (22 males, 81)cognitive variablesfemales), mean age 9.67 years.2)emotional impact of the median duration of epilepsy 21 mos (5-69 mos) epilepsy on the parent. 19 taking AEDs 15 with seizures in previous 6 months 27. As measured by the Child Behavior Checklist, six children (21%) scored in theclinically abnormal range, significantly greater than the expected 2%. Competenceproblems are indicated if a child has impaired academic performance reduced responsibilities around the home limited friendships is restricted in activities compared with a normed population of healthychildren. Children with benign Rolandic epilepsy scored, on average, significantly poorer onpsychosocial functioning, a measure assessing self-esteem mental health impact of the epilepsy on the family. Competency and psychosocial difficulties may influence the attainment of life skillsessential for healthy development, such as attaining mastery effective coping adapting to change, developing social skills. 28. The role of parental anxiety Parental anxiety is recognized as an important predictor of reducedquality of life in children with epilepsy. Maternal anxiety influences family adjustment and the quality of life of children with epilepsy. The major factor determining the emotional impact of the epilepsy on the parent was the use of medication and not the seizure variablesoccurrence, severity, age of onset, or duration.Williams J, Steel C, Sharp GB, et al. Parental anxiety and quality of life in children with epilepsy. Epilepsy Behav 2003;4:483-6. A significant finding of the current study was the importance of the emotional impact of the epilepsy on the parent its association with the childs quality of life. Parental emotional impact explained over a third of the variance inpredicting the reduced quality of life scores in the areas of self-esteem and language. 29. We conclude it is the diagnosis of benign Rolandic epilepsy itself,and the use of medicationthat contribute to parental concern. Careful explanation of the natural seizure history ofthe syndrome and the usual limited requirement formedication may lessen the emotional impact of theepilepsy on the parent. Awareness by the pediatrician of the level ofpotential parental concern in a benign epilepsy is asimportant as discussion of the epilepsy variablesand seizure management. 30. Building on the QOLCE apply to controlled as well as refractory epilepsy allow for patient as well as parent responses show responsiveness of the tool when repeatedat various intervals. refine evaluation of stigma 31. references