Syed Ameen Ahmad 1 John R Gatti 1 Rachel K Peterson 12 Noah Burton 1 Laura A Malone 234 Lisa R Sun MD 4 1 Johns Hopkins School of Medicine Baltimore MD USA 2 Kennedy Krieger Institute Baltimore MD ID: 1002328
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1. Patient-Centered Outcomes in Pediatric Moyamoya Arteriopathy: A Prospective Cohort StudySyed Ameen Ahmad1; John R. Gatti1; Rachel K. Peterson1,2; Noah Burton1, Laura A. Malone2,3,4; Lisa R. Sun, MD41Johns Hopkins School of Medicine, Baltimore, MD, USA; 2 Kennedy Krieger Institute, Baltimore, MD; ; 3Department of Physical Medicine and Rehabilitation, Johns Hopkins School of Medicine, Baltimore, MD, USA; 4Department of Neurology, Johns Hopkins School of Medicine, Baltimore, MD, USA
2. Moyamoya Arteriopathy (MMA)Progressive cerebral arteriopathy often occurring in childhoodSignificant risk factor for ischemic strokeIn absence of stroke, chronic cerebral hypoperfusion may affect neural circuitryMertens, R., Graupera, M., Gerhardt, H., Bersano, A., Tournier-Lasserve, E., Mensah, M. A., Mundlos, S., & Vajkoczy, P. (2022). The Genetic Basis of Moyamoya Disease. Translational stroke research, 13(1), 25–45. https://doi.org/10.1007/s12975-021-00940-2
3. Current Literature in MMAWhat is AvailableObjective Outcomes:Incidence of MMAOccurrence of strokeMortalityFunctionalityWhat is not well understoodPatient-Centered Outcomes:Quality of lifeMental healthSleep qualityGlobal health
4. Patient-Centered OutcomesWhy are they important to understand?Focused and improved counseling/treatmentMismatch between objective and patient-centered outcomesMoyamoya disease. Child Neurology Foundation. (2022, November 10). Retrieved January 29, 2023, from https://www.childneurologyfoundation.org/disorder/moyamoya-disease/
5. ObjectivesCharacterize a host of patient-centered outcomes, such as global health, mental health, quality of life, and quality of sleep, in a prospective cohort of pediatric patients with MMACompare patient-centered outcomes in patients with MMA to pediatric perinatal stroke and typically developing siblings
6. MethodsPatients with MMA treated at the Johns Hopkins Children’s Center were recruited for survey participationPatients (when developmentally appropriate) and their caretakers completed a battery of questionnaires via a secure RedCAP linkT-test and Wilcoxon Rank Sum test utilized to compare data between patients with MMA and normative values
7. Comparator GroupsUnaffected Siblings of patients with MMA:Raised in same environment but not affected by disease process Patients with Perinatal Stroke:History of stroke in first month of life (28 days)Does not have ongoing stroke risk
8. Questionnaires CompletedPatient-Reported Outcomes Information System (PROMIS)Anxiety/DepressionSleep Disturbance/Sleep ImpairmentPediatric Quality of Life (PedsQL) InventoryPhysicalEmotionalSocialSchool/Work
9. Demographic InformationMMA = Moyamoya Arteriopathy, SCD = Sickle Cell Disease, NF1 = Neurofibromatosis Type-1
10. Demographic InformationCharacteristicsMMA (N=21)Perinatal Stroke (N=18)Unaffected Siblings (N=8)Age, median (IQR) (years)7.8 (6.7,10.1)7.8 (4.8, 14.1)7.5 (4.7, 12.4)Sex, n (%) Male12 (57.1)11 (61.1)3 (37.5)Female9 (42.9)7 (38.9)5 (62.5)Stroke Occurrence, n (%)14 (66.7)18 (100)---Age at Moyamoya Presentation, median (IQR) (years)5.68 (3.2, 6.8)------Presentation of Moyamoya, n (%) ------Ischemic Stroke5 (23.8)Transient Ischemic Attack4 (19.1)Seizure2 (9.5)Headache1 (4.8)Radiographic8 (38.1)Other1 (4.8)
11. *** represents P < .05 when compared to MMA.Normative Values: Mean = 50, SD = 10n = 21
12. ********* represents P < .05 when compared to MMA.100 = Best Quality of Lifen = 21
13. **** represents P < .05 when compared to MMA.100 = Best Quality of Life
14. ConclusionsPatients with MMA had greater impairment in sleep disturbance, global health, and school/work quality of life compared to normative valuesPatients with perinatal stroke had higher rates of impairment in physical and school/work quality of life compared to patients with MMANeed for screening in patients with MMA without significant physical impairment
15. LimitationsLong battery of questionnairesSmall sample size in unaffected sibling group (n=8)Have yet to account for stroke impairment and functionality as mediating variables for patient-centered outcomes
16. Next StepsUtilizing the Pediatric Stroke Outcome Measure (PSOM) and the Recurrence and Recovery Questionnaire (RRQ) to account for stroke impairment as mediating variablesPerforming multiple linear regression to adjust for imaging, surgical data, and headache-related disability
17. AcknowledgementsLisa Sun (MD)Jack GattiLaura Malone (MD)Rachel Peterson (PhD)Noah BurtonThis work is supported by the Laney Jaymes Foundation for Pediatric Stroke, the American Heart Association [Career Development Award (850044)], the D.C. Women’s Board, and the Johns Hopkins Scholarly Concentration Program.
18. Questions?
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