Prospective multicenter clinical research studies in pediatric hydrocephalus are relatively rare. They cover a broad spectrum of hydrocephalus topics, including management of intraventricular hemorrhage in premature infants, shunt techniques and equipment, shunt outcomes, endoscopic treatment of hydrocephalus, and prevention and treatment of infection. The research methodologies include randomized trials, cohort studies, and registry-based studies. This review describes prospective multicenter studies in pediatric hydrocephalus since 1990. Many studies have included all forms of hydrocephalus and used device or procedure failure as the primary outcome. Although such studies have yielded useful findings, they might miss important treatment effects in specific subgroups. As multicenter study networks grow, larger patient numbers will allow studies with more focused entry criteria based on known and evolving prognostic factors. In addition, increased use of patient-centered outcomes such as neurodevelopmental assessment and quality of life should be measured and emphasized in study results. Well-planned multicenter clinical studies can significantly affect the care of children with hydrocephalus and will continue to have an important role in improving care for these children and their families.
Keywords: AIC = antibiotic-impregnated catheter; BASICS = British Antibiotic- and Silver-Impregnated Catheters Study; CDP = Core Data Project; CI = confidence interval; CPC = choroid plexus cauterization; DRIFT = drainage, irrigation, and fibrinolytic therapy; ESIT = Endoscopic Shunt Insertion Trial; ETV = endoscopic third ventriculostomy; HCRN = Hydrocephalus Clinical Research Network; HR = hazard ratio; IVH = intraventricular hemorrhage; OSV = Orbis Sigma Valve; PHVD = posthemorrhagic ventricular dilation; RCT = randomized controlled trial; SDT = Shunt Design Trial; cohort study; multicenter; pediatric hydrocephalus; prospective; randomized controlled trial; sIRB = single IRB.