Moyamoya Program | Research & Innovation

Our current research projects with promise for treating moyamoya and pediatric stroke include:

• Neurosurgeon’s pioneering technique helps thousands of children: Using 20 years of data, Boston Children’s R. Michael Scott, MD and Edward Smith, MD, track the long-term outcomes of pial synangiosis, the surgical treatment for moyamoya pioneered by Scott in the 1980s, in 59 of their patients.
• Higher volume hospitals have better outcomes: Dr. Smith and his colleagues published an analysis of 2,454 admissions of pediatric moyamoya patients across the U.S. in the largest study of its kind. The results show that higher volume centers provide improved care and reduced mortality.
• Evaluating possible growth factors in blood, cerebrospinal fluid, and urine: Dr. Smith and his colleagues are studying molecular compounds and growth factors in blood and in cerebrospinal fluid. They believe that these growth factors may yield crucial insights into the causes and progression of moyamoya, and may lead to therapies not only for that disorder, but also for cancer and other conditions involving the proliferation of blood vessels.
• Considering possible nonsurgical treatments for moyamoya: Dr. Smith is also working to determine whether new drug therapies may provide a reasonable alternative to surgery for children with moyamoya.
• Identifying biological warning signs for moyamoya: Our researchers are working to identify telltale proteins in blood and urine that could serve as early warning signals for the presence of disease by developing a urinary biomarker panel.
• Understanding the role of certain proteins in moyamoya and other disorders: Dr. Smith is investigating whether specific molecules may be culpable in the onset of several diseases of the central nervous system, including moyamoya, brain tumors, and vascular malformations of the brain.

Selected papers

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• Gaillard J, Klein J, Duran D, Storey A, Scott RM, Kahle K, Smith ER. Incidence, clinical features, and treatment of familial moyamoya in pediatric patients: a single-institution series. J Neurosurg Pediatr. 2017 May; 19(5):553-559.
• Titsworth WL, Scott RM, Smith ER. National Analysis of 2454 Pediatric Moyamoya Admissions and the Effect of Hospital Volume on Outcomes. Stroke. 2016 May; 47(5):1303-11.
• Smith ER. Structural causes of ischemic and hemorrhagic stroke in children: moyamoya and arteriovenous malformations. Curr Opin Pediatr. 2015 Dec; 27(6):706-11.
• See P; et al. Down syndrome and moyamoya: Clinical presentation and surgical management. J Neurosurg Pediatr. 2015 Jul;16(1):58-63.
• Smith ER. Moyamoya Biomarkers. J Korean Neurosurg Soc. 2015 Jun; 57(6):415-21.
• Ganesan V; et al. Moyamoya: defining current knowledge gaps. Dev Med Child Neurol. 2015 Feb 13 (DOI: 10.1111/dmcn.12708).
• Baird LC; et al. Moyamoya syndrome associated with Alagille syndrome: Outcome after surgical revascularization. J Pediatr. 2015 Feb; 166:470-3.
• A case report of five children.
• Gross BA; et al. Occipital pial synangiosis. Acta Neurochir (Wien) 2014 Jul; 156:1297-300.
• An illustrated, instructional video demonstration in a 5-year-old child shows that pial synangiosis can be successfully applied to moyamoya affecting the posterior circulation.
• Jackson EM; et al. Pial synangiosis in patients with moyamoya less than 2 years of age. J Neurosurg Pediatr. 2014 Apr; 13:420-5.
• A review of 34 procedures over a period of 12 years finds that the majority of children who have pial synangiosis for moyamoya before age 2 have a good long-term prognosis.
• Lin N; et al. Treatment of moyamoya disease in the adult population with pial synangiosis. J Neurosurg. 2014 Mar; 120:612-7.
• A review of 66 procedures from 1985 to 2010 finds pial synangiosis to be a safe and effective way to provide more blood to the brain in adults with moyamoya.
• Smith ER; Scott RM. Spontaneous occlusion of the circle of Willis in children: Pediatric moyamoya summary with proposed evidence-based practice guidelines. J Neurosurg Pediatrics. 2012; 9:353-60.
• This review summarizes current studies of pediatric moyamoya disease, proposing a framework for evidence-based treatment guidelines.
• Lin N; et al. Discovery of asymptomatic moyamoya arteriopathy in pediatric syndromic populations: radiographic and clinical progression. Neurosurg Focus.2011; 31(6):E6.
• Data on 418 patients who underwent surgical revascularization demonstrate that moyamoya is a progressive disorder, even when it does not cause symptoms, and supports the use of early surgical intervention to minimize morbidity from stroke.
• Scott RM; Smith ER. Moyamoya disease and moyamoya syndrome [review]. N Engl J Med. 2009; 360:1226-37.
• This comprehensive review of moyamoya covers who is at risk, presenting features, diagnosis, treatment and outcomes.
• Smith ER; et al. Pial synangiosis in patients with moyamoya syndrome and sickle cell anemia: perioperative management and surgical outcome. Neurosurg Focus. 2009; 26:E10.
• In this series of 12 patients with sickle-cell disease and moyamoya, 11 of whom had a prior stroke, pial synangiosis appears to be safe and confers long-lasting protection against further stroke.
• Scott RM; et. al. Long-term outcome in children with moyamoya syndrome after cranial revascularization by pial synangiosis. Journal of Neurosurgery: Pediatrics, 2004; 100(2), 142-149