The FDA approved dabigatran etexilate (Pradaxa) oral pellets to treat pediatric patients aged 3 months to younger than age 12 years with venous thromboembolism (VTE) directly after they have been treated with a blood thinner given by injection for at least 5 days. The FDA also approved Pradaxa oral pellets to prevent recurrent clots among patients in the same age group who completed their first VTE treatment.
In addition, Pradaxa was approved in capsule form to treat blood clots in patients aged 8 years and older with VTE directly after they have been treated with a blood thinner given by injection for at least 5 days and to prevent recurrent clots in patients aged 8 years and older who completed treatment for their first VTE. Pradaxa is the first FDA-approved anticoagulant that pediatric patients can take orally, as the only other approved anticoagulant for pediatric patients is administered via injection. Pradaxa was originally approved in 2010 to reduce the risk of stroke and systemic embolism in adult patients with nonvalvular atrial fibrillation.
The safety and efficacy of Pradaxa for treating VTE in patients younger than age 18 years was evaluated in a study of 267 pediatric patients. In this open-label trial, patients were randomly assigned to receive either Pradaxa or standard of care. The study compared the two groups to determine the number of patients who met the composite endpoint (a combination of multiple clinical endpoints), which meant that they had not died from a blood clot, their blood clots had completely resolved, and they had no additional blood clots. Results revealed that 81 (45.8%) of the 177 patients taking Pradaxa met the composite endpoint, compared with 38 (42.2%) of the 90 patients who received standard of care.
The safety of Pradaxa to prevent recurrent blood clots in the same pediatric population was evaluated in an open-label, single-arm study in 214 patients with a history of VTE. The primary endpoints of the study were recurrence of blood clots, major and minor bleeding events, and death (both overall and related to blood clots). The safety of Pradaxa with long-term use was comparable to that in the previously discussed study. Recurrence of blood clots occurred in three patients (1.4%), which was analogous to prior standard-of-care treatments.
The most common adverse effects of Pradaxa included digestive-system symptoms and bleeding. This agent can cause serious and fatal bleeding and is not recommended for patients with bioprosthetic heart valves or triple-positive antiphospholipid syndrome. The prescribing information for Pradaxa carries a boxed warning cautioning that early treatment discontinuation may augment the risk of blood clots and that blood accumulation within parts of the spinal cord (spinal or epidural hematomas) in patients undergoing spinal procedures may cause serious adverse effects. Pradaxa received a priority review designation for this indication.
In an FDA press release, Ann Farrell, MD, director of the Division of Non-Malignant Hematology in the FDA's Center for Drug Evaluation and Research, stated, "The FDA is committed to helping our youngest patients with serious medical conditions to have treatments that are relatively easy to take."
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