George Rodgers, III, MD, discusses antithrombin III's rare blood-clotting disorder expanded label

On November 18, 2025, the FDA expanded the indication of antithrombin III (human) (THROMBATE III) to include pediatric patients with hereditary antithrombin deficiency (hATd), making it the first and only antithrombin concentrate approved for both adults and children with the disorder. The update was supported by extrapolated adult data showing the product can be “safely and effectively used in pediatric patients with hATd,” according to Grifols. The label change marks a significant point for clinicians managing a condition associated with high lifetime thrombotic risk.

In his discussion with Contemporary Pediatrics, George M. Rodgers, III, MD, PhD, Professor of Medicine in the Division of Hematology and Hematologic Malignancies at the University of Utah School of Medicine, said the expanded indication represents a meaningful shift in pediatric care.

“Well, I think it’s an important advance, because prior to this, if they needed correction of low antithrombin levels, they were given plasma — fresh frozen plasma — which has a lot of potential transfusion hazards, and now there will be a sterile plasma-derived product that will specifically correct the deficiency in a more safe fashion,” he said.

Rodgers noted that this approval directly intersects with primary pediatric practice. “I think both have a potential role for the primary pediatrician, because they may, one, encounter the patient who has thrombosis, or perhaps the pediatric patient is asymptomatic but, on questioning the family, the pediatrician finds out that there is a family history of thrombosis.”

“I think that either a pediatric patient presenting with an unprovoked clot or an asymptomatic patient with a family history of clot should prompt testing for antithrombin deficiency, because it’s the most potent thrombophilia — inherited thrombosis risk factor — in terms of the likelihood of lifelong thrombotic risk,” he said.

The availability of a targeted replacement therapy also changes management decisions for children entering higher-risk situations.

“It will be really important to identify early in life, because unlike the other thrombophilias, we have a replacement product for antithrombin-deficient patients when they are in high-risk situations like surgery or hospitalization, or if they have an active clot, antithrombin therapy can make anticoagulation much easier."

Editor's note: Rodgers discloses he is a consultant for Grifols.

Reference:

Fitch J. FDA expands antithrombin III (human) label to pediatric patients with rare blood-clotting disorder. Contemporary Pediatrics. Published November 18, 2025. Accessed November 21, 2025. https://www.contemporarypediatrics.com/view/fda-expands-antithrombin-human-label-pediatric-patients-rare-blood-clotting-disorder