The U.S. Food and Drug Administration (FDA) has approved Cablivi (caplacizumab) for the treatment of pediatric patients ages 12 years and older with acquired thrombotic thrombocytopenic purpura (aTTP) in combination with plasma exchange and immunosuppressive therapy.
aTTP is a blood disorder characterized by low platelets, small areas of bleeding under the skin, low red blood cell count, and hemolytic anemia. aTTP causes blood clots to form in small blood vessels throughout the body. Complications may include neurological problems, fever, abnormal kidney function, abdominal pain, and heart problems. Hemolytic anemia can lead to paleness, jaundice, fatigue, shortness of breath, and a rapid heart rate. aTTP is caused when a person’s body mistakenly makes antibodies that block the activity of the ADAMTS13 enzyme.
Caplacizumab is a von Willebrand factor (vWF)-directed antibody fragment that targets the A1 domain of vWF and inhibits the interaction between vWF and platelets. The therapy was originally approved to treat adults with aTTP in 2019.
The efficacy of caplacizumab to treat pediatric patients was evaluated in a retrospective chart review study of 30 patients ages 2 to 18 years old. Results illustrated that 80% of patients achieved clinical remission, defined as achieving a normal platelet count and having LDH levels less than 1.5 times the upper limit of normal for 30 days or more.
Due to caplacizumab targeting abnormal blood clotting, the treatment can also cause serious and fatal bleeding. However, the most common side effects are nose bleeds, headache, and gingival bleeding.
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To learn more about aTTP and other rare hematologic disorders, visit https://checkrare.com/diseases/hematologic-disorders/
