Treatment of Autoimmune Thrombocytopenia (AITP)
Autoimmune Disease, Autoimmune Hemolytic Anemia, Thrombocytopenia
About this trial
This is an interventional treatment trial for Autoimmune Disease focused on measuring Immunosuppression, Thrombocytopenia, Autoimmune Disease, Evan's Syndrome, Autoimmune Hemolytic Anemia, Episodic Bleeding
Eligibility Criteria
INCLUSION CRITERIA: Male or female, ages 18-65 years old. Refractory severe chronic autoimmune thrombocytopenia, with or without autoimmune hemolytic anemia (Evan's syndrome), with all the following: Platelet count frequently below 20,000/mm(3) despite active treatment for a period of greater than 6 months. Normal or increased megakaryocytes on bone marrow aspirate/bx. No plausible alternative etiology such as drug-mediated thrombocytopenia, marrow failure syndrome or thrombocytopenia related to viral or bacterial infection. Failure of treatment with: i. conventional-dose steroids (e.g., prednisone or dosage of 40 mg/day or equivalent, followed by dosage taper) for at least 3 months. ii. intravenous immunoglobulin. iii. splenectomy. e. Episodic bleeding requiring transfusions or ecchymoses interfering with ordinary daily activities. EXCLUSION CRITERIA: ECOG performance status greater than 1. Cardiopulmonary disease including: History of coronary artery disease, angina pectoris or congestive heart failure. LV ejection fraction less than 40 percent by 2D echocardiogram. Renal disease, serum creatinine greater than 2.5 mg/dL or creatinine clearance less than 30 mL/min. Significant hepatic dysfunction, bilirubin greater than 2 mg/dL or transaminases greater than 2 times UNL. Uncorrected coagulopathy. Bone marrow aplasia (cellularity less than 10 percent), single or multilineage hematopoietic failure, myelodysplastic syndrome, or extensive marrow fibrosis. History or active diagnosis of malignancy (except treated non-melanoma skin cancer or cevical carcinoma in situ). HIV positive. Pregnancy or lactation, unwillingness to practice adequate birth control in the peritransplant period. Psychiatric illness or mental incapacity to understand and give informed consent. Other medical illness or condition which, in the opinion of the Investigators, may contraindicate participation in this study due to patients' risk or compromise of study integrity.
Sites / Locations
- National Institutes of Health Clinical Center, 9000 Rockville Pike