Treatment of Autoimmune Thrombocytopenia (AITP)

Launched by NATIONAL HEART, LUNG, AND BLOOD INSTITUTE (NHLBI) · Nov 3, 1999

Keywords

ClinConnect Summary

Autoimmune Thrombocytopenia (AITP) is a disorder of low blood platelet counts in which platelet destruction is caused by antiplatelet autoantibodies. A large proportion of patients with chronic AITP are refractory to standard therapies including corticosteroids, immune globulin and splenectomy. Cyclophosphamide is a cytotoxic immunosuppressive agent which may induce durable remissions of refractory autoimmune diseases. High-dose cyclophosphamide with peripheral blood stem cell (PBPC) rescue has been proposed as a potential definitive therapy for AITP; however, the infusion of autoreactive l...

Gender

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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:
• • 1. Platelet count frequently below 20,000/mm(3) despite active
• • treatment for a period of greater than 6 months.
• • 2. Normal or increased megakaryocytes on bone marrow
• • aspirate/bx.
• • 3. No plausible alternative etiology such as drug-mediated
• • thrombocytopenia, marrow failure syndrome or thrombocytopenia
• • related to viral or bacterial infection.
• 4. 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:
• • 1. History of coronary artery disease, angina pectoris or congestive heart failure.
• • 2. 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.