Childhood stroke is a serious and frequent which affects over 6/100,000 children / year and has a ten percent mortality. Adverse outcomes include neurological deficits or seizures in 70% and recurrent strokes in 20%. In adults, clinical trials provide evidence based specialized and standardized approaches to treatment. The adult trial conclusions may have limited value in children based on differences in pathophysiology. Pediatric stroke is at an early stage of research development with no randomized clinical trials (RCT) except in sickle cell disease and few epidemiological studies. The requisite data and collaboration for pediatric stroke trials is being obtained through a 2 year multi-center study initiated by the applicants and funded in 2002 by the CNS/CNF entitled """"""""Towards the Establishment of Standards of Practice and the Initiation of Multi-Center, Multi-National Clinical Trials for Neonates and Children with Stroke"""""""". This grant arose from an initial NINDS funded meeting in 2000 at which researchers met and developed a 15 center study. This 2 year consecutive cohort study which will be completed in December 2004 is providing data which will enable RCT's including diagnostic definitions, risk factors, current treatments and outcomes. It is now crucial to go the next step, in organizing a meeting, as proposed here, to bring together neurologists, epidemiologists and the FDA with experience in designing adult stroke and pediatric trials with key members of the pediatric neurology and epidemiology researchers to design childhood RCT's.
The specific aims are to apply the experience gleaned from adult stroke trials, and to discuss feasibility, potential design and prioritization of candidate studies, eg 1) RCT of anticoagulants vs. aspirin in secondary prevention of ischemic stroke; 2) intra-arterial tPA in older children with arterial ischemic stroke, and 3) low-molecular weight heparin vs. supportive therapy in neonates with non-hemorrhagic sinus thrombosis. A 3 day meeting is proposed at Glen Cove NY in September 2004. Day 1 will focus on relevant animal and adult stroke research, Day 2 will focus on available data in childhood stroke on: natural history, outcome measures, protection of patients, education strategies to promote early referral for acute therapies, pediatric safety data for tPA and other treatments, and sickle stroke trials. Day 3 will consist of working groups collating information from Days 1 and 2 into specific plans for at least 2 pediatric stroke clinical trials to be developed as grant applications. Participants will include adult stroke and sickle cell disease trialists, translational researchers and co-investigators of the international pediatric stroke study described above.
