Surgical revascularization remains the standard treatment for symptomatic
moyamoya disease (MMD). As with any major surgical treatment, revascularization is associated with risks and limitations, denoting the need for noninvasive treatments to improve ischemic symptoms and prevent
strokes.
Cilostazol is a selective
phosphodiesterase III inhibitor with antiplatelet, antithrombotic, and vasodilatory effects commonly used in
peripheral vascular disease. Clinical studies assessing the efficacy of
cilostazol in the management of
stroke and MMD were recently reported, although a comprehensive assessment of the overall evidence is lacking. A systematic scoping review was conducted to assess the early evidence on
cilostazol administration in patients with MMD. The inclusion criteria encompassed original human studies primarily focused on
cilostazol's safety, efficacy, or utilization in managing MMD patients. A search of the PubMed database was conducted in June 2023, yielding 5 peer-reviewed publications that satisfied the inclusion criteria and were subjected to narrative synthesis. Risk of bias assessment was not applicable due to the scoping nature of this review. East Asian studies demonstrate increasing rates of
cilostazol prescriptions for patients with MMD. In a large population-based study,
cilostazol was compared to other antiplatelet medications and yielded the largest decrease in mortality among patients with newly diagnosed MMD. Other studies reported significant improvements in cerebral blood flow and cognitive function, which were deemed to be independent of one another. There are limited data on the safety profile of
cilostazol in the MMD population, although the evidence derived from various studies performed in the general
stroke population can likely provide insights into its potential utility in MMD patients.
Cilostazol targets several critical pathways involved in the pathophysiology of MMD. The evidence corroborates the potential benefits of
cilostazol for the management of MMD, although these findings should be interpreted with caution due to the small number of studies and lack of randomized trials. Subgroups of patients need to be identified who can safely undergo medical management in lieu of revascularization surgery or to improve surgical outcomes. Additional studies are needed to assess the efficacy and safety of
cilostazol therapy, especially in Western populations.