Changes in the clinical spectrum of pediatric moyamoya disease over 40 years

Area of Science:

• Neurology
• Pediatric Neurology
• Vascular Neurology

Background:

• Moyamoya disease is a rare progressive cerebrovascular disorder.
• Pediatric-onset moyamoya disease presents unique challenges in diagnosis and management.
• Understanding chronological changes in presentation and prognosis is crucial for pediatric moyamoya disease care.

Purpose of the Study:

• To analyze temporal trends in the clinical characteristics and long-term outcomes of pediatric moyamoya disease.
• To compare patient cohorts from two distinct 20-year periods (1981-2000 vs. 2001-2020).
• To identify risk factors associated with poor long-term outcomes in pediatric moyamoya disease.

Main Methods:

• Retrospective evaluation of 282 pediatric moyamoya disease patients (onset ≤15 years) from 1981-2020.
• Division of patients into two cohorts: 1981-2000 (former period) and 2001-2020 (latter period).
• Comparison of clinical presentation, demographic data, and long-term outcomes between the two periods, including multivariate analysis for risk factors.

Main Results:

• The latter period showed an increased number of patients, higher onset age, and more cases with family history, mild symptoms, or headaches.
• A significant decrease in poor long-term outcomes was observed in the latter period (24.9% vs. 6.7%).
• Stroke onset, subsequent cerebrovascular events, and postoperative complications were identified as independent risk factors for adverse long-term outcomes.

Conclusions:

• Pediatric moyamoya disease incidence has risen over 40 years, with a trend toward milder presentations and improved outcomes in recent cohorts.
• Chronological shifts in patient demographics and presentation must be considered in clinical research.
• Further research is needed to determine if increased diagnostic rates contribute to reduced long-term cerebrovascular events and improved lifelong outcomes.