PurposeTo evaluate the safety and efficacy of alteplase for restoring function to occluded central venous catheters in a pediatric population.Patients and MethodsA phase III, open-label, single-arm, multicenter trial was performed in 995 adult and pediatric patients with dysfunctional nondialysis catheters and ports. This report is a subset analysis of subjects between 2 and 18 years of age (N = 122) who were enrolled in the study. Alteplase (2 mg/2 mL) was instilled into the dysfunctional catheter lumen and assessed at 30 and 120 minutes. Subjects weighing ≥30 kg received 2 mL of alteplase; subjects <30 kg received 110% of the internal lumen volume (not exceeding 2 mL). Alteplase dosing was repeated once after 120 minutes if the catheter remained dysfunctional. The primary safety endpoint was the rate of intracranial hemorrhage (ICH) within 5 days of treatment.ResultsThe overall efficacy following up to two instilled doses of alteplase was 87%. In 70 patients (57%), restoration of catheter flow occurred by 30 minutes following a single dose of alteplase. Restoration of function was related to the duration of occlusion (P= 0.04). For catheters with occlusions of 0, 1 to 14, and >14 days duration, the efficacy was 91%, 78%, and 60%, respectively. Success was independent of the patient's age, sex, body weight, CVC type, or catheter age. There were no cases of death, ICH, major bleeding episodes, or embolic events attributable to treatment.ConclusionsAn alteplase regimen of up to two 2-mg doses is safe and effective for restoration of function to occluded central venous catheters in a pediatric population.