Lauren Dempsey, MS in Biomedicine and Law, RN, FISM News
In June the FDA approved the first oral blood thinner medication for children. Pradaxa was originally approved in 2010 for adults to help reduce the risk of blood clots and stroke for patients with atrial fibrillation. Blood clots can occur in veins and arteries and, if left untreated, can cause stroke and pulmonary embolism.
This isn’t the first blood thinner that the FDA has approved for use in children. In 2019, the medication Fragmin was approved by the FDA; however, this medication is only available by subcutaneous injection. This was initially approved in 1994 in adult populations and works similarly to heparin. The decision to approve Fragmin for children was based on a single clinical trial that evaluated 38 pediatric patients with deep vein thrombosis or pulmonary embolism.
The June 21st press release details the use of Pradaxa “to treat children 3 months to less than 12 years old with venous thromboembolism (a condition where blood clots form in the veins) directly after they have been treated with a blood thinner given by injection for at least five days,” and importantly, “Pradaxa is the first FDA-approved blood thinning medication that children can take by mouth; the only other approved blood thinning medication for children is given by injection.”
Children that have cancer, congenital heart disease, a central venous catheter, or are admitted to an intensive care unit have a high risk for developing blood clots which can lead to complications, including swelling and pain near the clot, chest pain, lung damage, and death.
Current data indicates that approximately 1 in 10,000 children will get blood clots, this includes as many as 1 in 200 hospitalized children.
The clinical trials began in 2013 and were completed in October of 2019, the main objective of the study being to assess the efficacy of Pradaxa when compared to the current standard of care. Results from the trial showed that 45.8% of the patients taking Pradaxa had not died from a blood clot, their blood clots had completely resolved, and they had no additional blood clots, compared to 42.2% of the patients who received the current standard of care.
In correspondence between the FDA and Boehringer Ingelheim Pharmaceuticals Inc. (Pradaxa’s manufacturer) the clinical trials are clearly outlined, as well as what data from the trials should be submitted prior to approval.
A second study was done to evaluate the long-term safety of Pradaxa and the ability to prevent recurrent blood clots in the same population. The study included 214 patients with a history of blood clots and showed that 1.4% (three patients) had a recurrence of blood clots when compared to the standard of care.
Ann Farrell, M.D., director of the Division of Non-Malignant Hematology in the FDA’s Center for Drug Evaluation and Research, said that “The FDA is committed to helping our youngest patients with serious medical conditions have treatments that are relatively easy to take.” She added, “ With today’s approval of Pradaxa, pediatric patients have another therapeutic option to treat and prevent potentially deadly blood clots.”