Current as of December 09, 2023
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...
- 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
- 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
- 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.
The National Institute of Allergy and Infectious Diseases (NIAID, /ˈnaɪ.æd/) is one of the 27 institutes and centers that make up the National Institutes of Health (NIH), an agency of the United States Department of Health and Human Services (HHS). NIAID's mission is to conduct basic and applied research to better understand, treat, and prevent infectious, immunologic, and allergic diseases.
Immunology at National Institute of Allergy and Infectious Diseases (NIAID)
Bethesda, Maryland, United States
All reviews come from applied patients