Study of Cyclosporine or Corticosteroids as an Adjunct to Plasma Exchange in Thrombotic Thrombocytopenic Purpura (TTP)
Study of Cyclosporine or Corticosteroids as an Adjunct to Plasma Exchange in Thrombotic Thrombocytopenic Purpura (TTP)
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.
Inclusion Criteria:

 -  Patients with a clinical diagnosis of idiopathic TTP as defined by a microangiopathic
 hemolytic anemia and thrombocytopenia (<100 x 103)

 -  Additional components of the pentad (fever, renal and neurologic abnormalities) need
 not be present.

 -  Additional explanations for the microangiopathic changes including DIC and malignancy
 should be excluded.

 -  Patients with pregnancy associated TTP will be permitted on this therapeutic trial if
 the child is delivered prior to the initiation of therapy for TTP. However, female
 patients that are breastfeeding and are unwilling to discontinue breastfeeding at the
 time of enrollment will be excluded from this study

 -  Patients with a previous diagnosis of TTP are eligible to be enrolled provided they
 meet eligibility criteria and have not been treated for an TTP in the past 30 days

 -  Given the potential for nephrotoxicity with CSA, all patients must have a serum
 creatinine of < 2.5 mg/dl prior to enrollment

 Exclusion Criteria:

 -  In light of concern for the prompt initiation of PE, all patients with suspected TTP
 may be enrolled on this trial. If it is subsequently found that the patient does not
 meet enrollment criteria, they will be removed and their spot replaced for study
 purposes. Patients removed from the study after enrollment will continue to be
 followed longitudinally for 6 months to be monitored for safety and will be included
 in the safety database.

 -  Patients with TTP clinically categorized as secondary to stem cell transplant and
 solid organ, bloody diarrhea associated, malignancy associated, and drug associated
 will not be enrolled on this therapeutic study.

 -  Incarcerated patients will be excluded from the study due to the inherent difficulties
 in maintaining close follow-up for study purposes in patients who are incarcerated.

 -  Any patients already being treated chronically with corticosteroids or cyclosporine
 and taking these at the time of their presentation will be excluded from this study.

 -  Female patients that are breastfeeding and are unwilling to discontinue breastfeeding
 at the time of enrollment will be excluded from this study

 -  Patients taking any medications contraindicated in combination with CSA that cannot be
 safely discontinued will be excluded from this study.