Rituximab and Prednisone as First-Line Therapy in Treating Patients With Immune Thrombocytopenic Purpura

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT00486421
Recruitment Status : Completed
First Posted : June 14, 2007
Last Update Posted : October 17, 2014
National Cancer Institute (NCI)
Information provided by (Responsible Party):
Mayo Clinic

Brief Summary:

RATIONALE: Rituximab and prednisone may increase the number of platelets in patients with immune thrombocytopenic purpura.

PURPOSE: This phase II trial is studying the side effects and how well giving rituximab together with prednisone works as first-line therapy in treating patients with immune thrombocytopenic purpura.

Condition or disease Intervention/treatment Phase
Nonneoplastic Condition Biological: Rituximab Drug: Prednisone Early Phase 1

Detailed Description:



  • Determine the efficacy of rituximab, when administered with standard prednisone treatment, in maintaining a platelet count ≥ 50,000/mm³ at 6 months without further therapies (e.g., splenectomy or other salvage therapies) in patients with immune thrombocytopenic purpura.
  • Determine the safety of this regimen in these patients.


  • Determine the time to platelet recovery in patients treated with this regimen.
  • Determine the duration of platelet recovery in patients treated with this regimen.
  • Assess efficacy of this regimen in preventing spontaneous bleeding events in these patients.
  • Determine the response in patients treated with this regimen.

OUTLINE: This is a pilot study.

Patients receive rituximab IV on days 1, 8, 15, and 22 and oral prednisone once daily on days 1-14 followed by a taper to day 56. Treatment is administered in the absence of disease relapse or unacceptable toxicity.

After completion of study therapy, patients are followed periodically for up to 3 years.

Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 22 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Pilot Study of Rituximab in Combination With Corticosteroids for the Initial Treatment of Immune Thrombocytopenic Purpura
Study Start Date : January 2007
Actual Primary Completion Date : May 2008
Actual Study Completion Date : November 2008

Arm Intervention/treatment
Experimental: PRED & RITUX Biological: Rituximab
375mg/m2 IV weekly times 4 (days 1, 8, 15, 22)
Other Name: Rituxan

Drug: Prednisone
1mg/kg/d PO, taper to off by 8 weeks

Primary Outcome Measures :
  1. Failure-free survival at 6 months [ Time Frame: 6 months ]

Secondary Outcome Measures :
  1. Time to platelet recovery [ Time Frame: 1 year ]
  2. Duration of platelet recovery [ Time Frame: 1 year ]
  3. Effect of treatment on prevention of spontaneous bleeding events [ Time Frame: 1 year ]

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Ages Eligible for Study:   21 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No


  • Diagnosis of immune thrombocytopenic purpura (ITP)

    • Diagnosis must be made according to American Society of Hematology diagnostic guidelines by a member of Mayo Rochester's Division of Hematology/Oncology within the past year
    • ITP must be confirmed by bone marrow aspiration and biopsy in all patients ≥ 60 years of age*

      • Bone marrow studies performed outside Mayo must be reviewed by a Mayo hematopathologist to confirm diagnosis and exclude evidence of other hematologic disorders NOTE: *Bone marrow evaluation is discretionary for all other patients
  • Requires treatment, as defined by 1 of the following parameters:

    • Platelet count ≤ 30,000/mm³
    • Platelet count ≤ 50,000/mm³ with episodic bleeding (i.e., spontaneous or with minimal trauma) requiring treatment
  • No concurrent diagnosis of a condition known to cause secondary immune (or nonimmune) thrombocytopenia, including, but not limited to, any of the following:

    • Rheumatological conditions, such as lupus, rheumatoid arthritis, scleroderma, or mixed connective tissue disorder

      • Patients with positive serologies and no concurrent, clinically evident condition are eligible
    • HIV positive or AIDS
    • Non-Hodgkin's lymphoma, Hodgkin's lymphoma, chronic lymphocytic lymphoma, multiple myeloma, or other malignant hematological conditions
    • Clinically evident antiphospholipid antibody syndrome* or heparin-induced thrombocytopenia
    • Clinically overt liver disease, hepatitis B surface antigen positive, hepatitis C serology positive, or evidence of a microangiopathic hemolytic anemia, such as disseminated intravascular coagulation, hemolytic-uremic syndrome, thrombotic thrombocytopenic purpura, or preeclampsia NOTE: *Positive laboratory tests without the defined clinical criteria for a diagnosis of antiphospholipid antibody syndrome is allowed


  • ECOG performance status 0-2
  • Creatinine ≤ 2 times upper limit of normal (ULN)
  • Direct bilirubin ≤ 1.5 times ULN
  • Total bilirubin ≤ 1.5 times ULN
  • AST ≤ 2.5 times ULN
  • Hemoglobin ≥ 10 g/dL
  • WBC ≥ 3,000/mm³
  • Not pregnant or nursing
  • Negative pregnancy test
  • Fertile patients must use effective contraception
  • No hypersensitivity to murine or chimeric proteins
  • No other disease, metabolic dysfunction, physical examination finding, or clinical laboratory finding giving reasonable suspicion of a disease or condition that contraindicates the use of an investigational drug or that may affect the interpretation of the results or render the patient at high risk for treatment complications
  • Able to take a proton-pump inhibitor while on corticosteroids
  • No unresolved or incompletely treated infection within the past 14 days


  • No prior corticosteroid therapy since the diagnosis of ITP

    • Corticosteroid therapy is allowed for up to 14 days prior to study entry, once the baseline CBC has been established
  • No prior rituximab
  • No other concurrent therapy for ITP, including androgens, IV immunoglobulins, RH_o (D) immune globulin, cyclosporine, or azathioprine sodium

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT00486421

United States, Minnesota
Mayo Clinic
Rochester, Minnesota, United States, 55905
Sponsors and Collaborators
Mayo Clinic
National Cancer Institute (NCI)
Study Chair: Ruben A. Mesa, M.D. Mayo Clinic

Responsible Party: Mayo Clinic Identifier: NCT00486421     History of Changes
Other Study ID Numbers: CDR0000529883
P30CA015083 ( U.S. NIH Grant/Contract )
MC0481 ( Other Identifier: Mayo Clinic Cancer Center )
2071-04 ( Other Identifier: Mayo Clinic IRB )
U2985s ( Other Identifier: Genentech protocol )
First Posted: June 14, 2007    Key Record Dates
Last Update Posted: October 17, 2014
Last Verified: October 2014

Keywords provided by Mayo Clinic:
idiopathic thrombocytopenic purpura

Additional relevant MeSH terms:
Purpura, Thrombocytopenic
Purpura, Thrombocytopenic, Idiopathic
Blood Coagulation Disorders
Hematologic Diseases
Pathologic Processes
Skin Manifestations
Signs and Symptoms
Thrombotic Microangiopathies
Blood Platelet Disorders
Immune System Diseases
Hemorrhagic Disorders
Autoimmune Diseases
Antineoplastic Agents
Immunologic Factors
Physiological Effects of Drugs
Antirheumatic Agents
Anti-Inflammatory Agents
Hormones, Hormone Substitutes, and Hormone Antagonists
Antineoplastic Agents, Hormonal