This research involves the use of immune base therapy as an adjunct to plasma exchange, the present standard of care for thrombotic thrombocytopenic purpura (TTP). Funding source -FDA OOPD
TTP is a rare blood disorder that causes blood clots to form in the small blood vessels throughout the body, including the kidneys, brain, abdomen, and the heart. Plasma exchange is the standard treatment for TTP. Plasma exchange is a treatment that removes the plasma (the liquid portion of the blood without any cells) from a patient and replaces it with plasma from a donor. With plasma exchange, 90% of patients achieve a remission of the disease. Unfortunately, up to one half of patient will relapse after the plasma exchange has stopped, leading to significant complications and added risks to the patient. This study randomizes patients to receive either prednisone or cyclosporine as an adjunct to plasma exchange, with the cyclosporine arm being the experimental arm of the study. All patients will undergo plasma exchange but will be randomized to receive either prednisone or cyclosporine as an adjunct to plasma exchange. Previous studies suggested that cyclosporine was superior to prednisone as an adjunct to plasma exchange, and therefore this randomized study attempts to confirm the findings of two previous single institution studies.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
26
2-3 mg/kg orally in a twice day divided dose for 6 months
1 mg/kg orally, daily for at least 30 days, then tapered over 30 days after achieving remission.
Ohio State University
Columbus, Ohio, United States
Number of Participants With Exacerbations in the CSA/PEX Arm Compared to the Steroids/PEX Arm
Number of Participants with Exacerbations in the CSA/PEX Arm Compared to the Steroids/PEX Arm
Time frame: From the start of treatment until 30 days after discharge from the last PEX procedure
Time in Days to Achieve a Clinical Response, Comparing the CSA/PEX Arm to the Steroids/PEX Arm.
Days to achieve a clinical response, defined as a normal platelet count (\>150 x 109/L), normal LDH, and no new end organ injury.
Time frame: Time to starting treatment until 6 months after the last PEX procedure
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