by Massachusetts General Hospital researchers, led by Dr. Pavan K. Bendapudi, have reported the first successful use of a novel drug to treat a patient with immune thrombotic thrombocytopenic purpura (iTTP), a rare and potentially life-threatening blood clotting disorder. The team’s findings were published in the New England Journal of Medicine.
iTTP is characterized by uncontrolled clotting in small blood vessels due to an autoimmune attack against the enzyme ADAMTS13, which is responsible for cleaving a large protein involved in blood clotting. The standard treatment for this disorder has been plasma exchange, which removes the harmful autoantibodies and provides additional ADAMTS13.
In this case, the team employed a genetically engineered version of the missing enzyme to treat a patient with an extremely severe form of iTTP. According to Dr. Bendapudi, the lead author and an investigator in the Division of Hematology Analyzer and Blood Transfusion Service at Massachusetts General Hospital, as well as an assistant professor of Medicine at Harvard Medical School, the drug effectively reversed the disease process in the patient.
Dr. Bendapudi stated, “We have demonstrated the first clinical use of this drug for iTTP. Our findings suggest that this innovative approach could potentially offer a more targeted and effective treatment option for patients with this life-threatening disorder.”
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