Patient-Reported Outcomes in Childhood Moyamoya Arteriopathy

Background: Little is known about quality of life, sleep, and mental health in children with moyamoya arteriopathy (MMA). A better understanding of patient-reported outcomes may lead to improved treatment.

Methods: Patients with MMA <26 years old and their caretakers completed validated, age-appropriate questionnaires including the Pediatric Quality of Life (PedsQL) Inventory and Patient-Reported Outcomes Measurement Information System (PROMIS) measuring health-related quality of life, sleep, anxiety, depression, and overall health.

Results: All 21 caretaker-proxies and eight of 21 patients with MMA completed questionnaires. Median age of participants with MMA was 7.8 years. The MMA cohort included 10 children with moyamoya disease and 11 with moyamoya syndrome, and 11 participants (52.4%) had a prior clinical stroke. Nine siblings unaffected by moyamoya and 20 participants with perinatal stroke were enrolled as comparator groups. Participants with MMA had greater impairment in school/work quality of life compared with normative values on caretaker and child-reported PedsQL surveys (85.5 vs 62.3 [P < 0.001] and 78.6 vs 51.2 [P = 0.017], respectively). Participants with MMA did not have significantly different physical impairment compared with normative values and siblings on caretaker and child-reported surveys. Caretaker-proxy PROMIS scores revealed high rates of moderate-severe anxiety (57.1% vs 25%, P < 0.001), sleep impairment (47.6% vs 25%, P = 0.017), and fair-poor global health (76.2% vs 25%, P < 0.001) in participants with MMA compared with the general population.

Conclusions: Patients with MMA have high rates of school/work impairment, anxiety, sleep impairment, and fair-poor global health. Screening for patient-reported outcomes, even in the absence of stroke or physical impairment, may improve treatment.