Distinct Clinical and Radiographic Phenotypes in Pediatric Patients With Moyamoya

Pediatr Neurol. 2021 Jul:120:18-26. doi: 10.1016/j.pediatrneurol.2021.03.002. Epub 2021 Apr 2.

Abstract

Background: Given the expanding evidence of clinico-radiological differences between moyamoya disease (MMD) and moyamoya syndrome (MMS), we compared the clinical and radiographic features of childhood MMD and MMS to identify predictors of ischemic event recurrence.

Methods: We reviewed a pediatric moyamoya cohort followed between 2003 and 2019. Clinical and radiographic characteristics at diagnosis and follow-up were abstracted. Comparisons between MMD and MMS as well as between MMD and two MMS subgroups (neurofibromatosis [MMS-NF1] and sickle cell disease [MMS-SCD]) were performed.

Results: A total of 111 patients were identified. Patients with MMD presented commonly with transient ischemic attacks (TIAs) (35 % MMD versus 13% MMS-NF1 versus 9.5% MMS-SCD; P = 0.047). Symptomatic stroke presentation (MMD 37% versus MMS-NF1 4% versus 33%; P = 0.0147) and bilateral disease at diagnosis (MMD 73% versus MMS-NF1 22 % versus MMS-SCD 67%; P = 0.0002) were uncommon in MMS-NF1. TIA recurrence was common in MMD (hazard ratio 2.86; P = 0.001). The ivy sign was absent on neuroimaging in a majority of patients with MMS-SCD (MMD 67% versus MMS-NF1 52% versus MMS-SCD 9.5%; P = 0.0002). Predictors of poor motor outcome included early age at diagnosis (odds ratio [OR] 8.45; P = 0.0014), symptomatic stroke presentation (OR 6.6; P = 0.019), and advanced Suzuki stage (OR 3.59; P = 0.019).

Conclusions: Moyamoya exhibits different phenotypes based on underlying etiologies. Frequent TIAs is a common phenotype of MMD and symptomatic stroke presentation a common feature of MMD and MMS-SCD, whereas unilateral disease and low infarct burden are common in MMS-NF1. In addition, absence of ivy sign is a common phenotype in MMS-SCD.

Keywords: Childhood moyamoya; Neurocognitive outcome; Neurofibromatosis type 1; Outcome measure; Pediatric stroke; Sickle cell disease.

Publication types

  • Comparative Study

MeSH terms

  • Adolescent
  • Anemia, Sickle Cell / complications*
  • Child
  • Child, Preschool
  • Cognitive Dysfunction / etiology*
  • Cognitive Dysfunction / physiopathology
  • Disease Progression*
  • Female
  • Follow-Up Studies
  • Humans
  • Ischemic Attack, Transient / diagnostic imaging
  • Ischemic Attack, Transient / etiology*
  • Male
  • Moyamoya Disease / complications*
  • Moyamoya Disease / diagnostic imaging
  • Moyamoya Disease / etiology
  • Moyamoya Disease / physiopathology
  • Neurofibromatosis 1 / complications*
  • Outcome Assessment, Health Care
  • Phenotype
  • Stroke / diagnostic imaging
  • Stroke / etiology*