This presentation brings together 3 currents in my approach to treating pediatric epilepsy: a recognition of the importance of epilepsy comorbidities, partnering with families in patient assessment and decision making, and creative use of informatics/EHR's to gather information about clinical aspects of epilepsy. A presentation to the Missouri Valley Child Neurology Colloquium, March 2012.

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

2. Objectives
1. list the possible uses for a tool to
measure pediatric seizure burden
2. review the list of available tools to
measure pediatric seizure burden
3. review the steps used to validate new
screening questionnaires

3. purpose
 to quantitate the burdens (personal and family) that stem
from epilepsy
 not just numbers of seizures, but all aspects of the health
problem of epilepsy
 to emphasize those burdens that are susceptible to change
with 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
embarrassment
med side
activity
effects sudden
restrictions
death

6. burden: comorbidities
sleep
anxiety
depression
learning
attention disabilities
disorder

7. burden: stigma, limitations
sports driving
school
society
career
social 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 in
association 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 Huntington’s disease
 Some of this is unknown or uncharacterized
 e.g. cryptogenic infantile spasms
 These neurologic disabilities are associated with a spectrum of
burdens over and above the burdens attributable to epilepsy
itself.

9. burden: neurologic disability
motor financial
cognitive
disease
tempo
independence

10. Isn’t this just a form of QOLIE?
 focus is slightly different
 some burdens don’t 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 therapy
changes will be made in each of the domains separately
 We’ll be interested in measuring points in time as well as changes over
time
 (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) Johnny’s 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 status
time 1
time 2
time 3

13. What’s 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 what
you think it’s 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
B epilepsy comorbidities
 affective
 sleep
 socialization
C stigma
 societal
 school
 family
D

15. face validity:
How does this tool accord with current
understanding 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 the
expected 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 with
already validated test I)
 roughly similar domains between tests should correlate
 discriminant validity (domain A measures a different quality
than domain B)
 different domains should show appropriately different
correlations
 check ceiling and floor effects, skew
 internal consistency reliability (Cronbach’s α)

17. ceiling effect floor effect
group responses group responses
skew
group responses

19. Brevity, Family Focus

20. IMPACT OF PEDIATRIC EPILEPSY SCALE

21. QOLCE
QUALITY OF LIFE IN CHILDHOOD EPILEPSY
Domains 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 functioning or neurodegenerative disease
 social functioning were excluded.
 behavior  20 minutes to complete

22. 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?

24.  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

25. What factors predicted change in QOL?
predictive not 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

26. What if we look at measuring burden in children
with benign centrotemporal epilepsy?
What do you think?
 The main determinants of quality of life in these
children include
a) seizure frequency
b) AED side effects
c) injuries during seizures
d) episodes of status epilepticus
e) none of the above

27. Pediatr Neurol 2006;35:240-245
In benign rolandic epilepsy the prognosis for seizure outcome is excellent. Because of
the benign nature of the epilepsy, it is generally presumed that quality of life is not
significantly affected. As such, there is a paucity of research in this area.

28. Study design Results
 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 with
classification. normative data.
 Patients underwent a comprehensive  The average psychosocial score was
cognitive 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 Questionnaire
of life in the areas of self-esteem and
language.
 Parental emotional impact was assessed
using a subscale.  Parental emotional impact, however, was a
 Child Behavior Checklist major independent predictor of quality of
life.
 Quality of Life in Childhood Epilepsy
Questionnaire.  Quality of life… is related to
 The cohort included 30 patients (22 males, 8 1) cognitive variables
females), 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 AED’s
 15 with seizures in previous 6 months

30.  As measured by the Child Behavior Checklist, six children (21%) scored in the
clinically abnormal range, significantly greater than the expected 2%. Competence
problems 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 healthy
children.
 Children with benign Rolandic epilepsy scored, on average, significantly poorer on
psychosocial 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 skills
essential for healthy development, such as
 attaining mastery
 effective coping
 adapting to change,
 developing social skills.

31. The role of parental anxiety
 Parental anxiety is recognized as an important predictor of reduced
quality 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 variables—occurrence, 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 child’s quality of life.
 Parental emotional impact explained over a third of the variance in
predicting the reduced quality of life scores in the areas of self-
esteem and language.

32.  We conclude it is
 the diagnosis of benign Rolandic epilepsy itself,
and
 the use of medication
that contribute to parental concern.
 Careful explanation of the natural seizure history of
the syndrome and the usual limited requirement for
medication may lessen the emotional impact of the
epilepsy on the parent.
 Awareness by the pediatrician of the level of
potential parental concern in a benign epilepsy is as
important as discussion of the epilepsy variables
and seizure management.

33. 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 repeated
at various intervals.
 refine evaluation of stigma

34. references