Visit Schedule and Content Once medication therapy was initiated, close monitoring was instituted. Research study Visits were conducted at
weeks 1, 3, 5, 8 and then every 4weeks until the medication was discontinued by the physician or the medication administration protocol ended. Vital signs, a symptom- directed physical examination, history of adverse events, concomitant medications, compliance with study medica- tions and blood samples were obtained at each visit. Compliance was assessed by a self-report patient diary (completed by either the patient or the care giver) and by vial and pill counts (completed by the site pharmacist or study coordinator). Depending on treatment and response, total therapy could last from 24 to 72 weeks. The protocol was designed for each center to enroll between 5 and 15 children, for a total recruitment of 114 subjects.