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In this prospective and retrospective chart review, investigators will evaluate the response rates and duration of response for patients with relapsed and refractory idiopathic thrombocytopenic purpura (ITP) who have been treated with rituximab and repeated courses of dexamethasone. Investigators will also evaluate observed toxicities of the combination, and characteristics associated with response.
Patients with idiopathic thrombocytopenic purpura (ITP) usually respond with an increase in platelet count after treatment with steroids, but most patients will experience recurrence.Rituximab has been a useful treatment for patients with recurrent ITP; many hundreds of patients have been treated. 30-40% of patients will achieve a complete remission (CR: platelet count >150 x 109/l) with initial treatment. However, most patients will relapse between 1 and 3 years from initial treatment suggesting that long-term "cures" only occur in 20% of the initial patients. An increased rate of durable CRs is therefore a desirable goal. One approach would be to use rituximab maintenance; however, this strategy results in long term B cell suppression. Dexamethasone has also been used to achieve "cure" in ITP, especially in adults at or near diagnosis. An initial study suggested that approximately 50% of patients would achieve a long-term response with only one 4-day cycle of high dose (40 mg/day) dexamethasone. A follow up study suggested that 3-4 cycles of dexamethasone would be better than 1 cycle. Finally, a recent publication suggested that rituximab plus one cycle of dexamethasone was superior to dexamethasone alone, with a > 50% CR rate at 6 months. Based on the published activity of rituximab and dexamethasone in this disorder, some patients have received treatment with combined dexamethasone and rituximab. We will review the data of those patients who have received more than one course of dexamethasone together with rituximab for relapsed or refractory ITP in order to determine the response rate, duration of response, toxicity of the combination, and clinical predictors of response in this group.
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| Measure | Description | Time Frame |
|---|---|---|
| An increase in Platelet count | An increase in Platelet count after 8 weeks on treatment | 8 weeks |
| Measure | Description | Time Frame |
|---|---|---|
| Achieve a complete or partial response to treatment | Investigators hope patients will achieve a complete response (a platelet count greater than or equal to 100,000/uL) or a partial response (a platelet count between 50,000/uL and 99,000/uL) not due to rescue medication within an average of 12 weeks | Within an average of 12 weeks |
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Inclusion Criteria:
Exclusion Criteria:
1. Subjects who have not been diagnosed with ITP. 2 Subjects who are younger than 3 years old 3. Subjects who have a medical condition that would be adversely affected by high dose steroids
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Patients who have received more than one course of dexamethasone together with rituximab for relapsed or refractory ITP.
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| Name | Affiliation | Role |
|---|---|---|
| James B Bussel, M.D. | Weill Medical College of Cornell University | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| New York Presbyterian Hospital Weill Cornell Medical College | New York | New York | 10065 | United States |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 24747949 | Derived | Bussel JB, Lee CS, Seery C, Imahiyerobo AA, Thompson MV, Catellier D, Turenne IG, Patel VL, Basciano PA, Elstrom RL, Ghanima W. Rituximab and three dexamethasone cycles provide responses similar to splenectomy in women and those with immune thrombocytopenia of less than two years duration. Haematologica. 2014 Jul;99(7):1264-71. doi: 10.3324/haematol.2013.103291. Epub 2014 Apr 18. |
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| ID | Term |
|---|---|
| D016553 | Purpura, Thrombocytopenic, Idiopathic |
| ID | Term |
|---|---|
| D011696 | Purpura, Thrombocytopenic |
| D011693 | Purpura |
| D001778 | Blood Coagulation Disorders |
| D006402 | Hematologic Diseases |
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| D006425 |
| Hemic and Lymphatic Diseases |
| D057049 | Thrombotic Microangiopathies |
| D013921 | Thrombocytopenia |
| D001791 | Blood Platelet Disorders |
| D000095542 | Cytopenia |
| D006474 | Hemorrhagic Disorders |
| D001327 | Autoimmune Diseases |
| D007154 | Immune System Diseases |
| D006470 | Hemorrhage |
| D010335 | Pathologic Processes |
| D013568 | Pathological Conditions, Signs and Symptoms |
| D012877 | Skin Manifestations |
| D012816 | Signs and Symptoms |