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

This study has been completed.
National Cancer Institute (NCI)
Information provided by (Responsible Party):
Mayo Clinic
ClinicalTrials.gov Identifier:
First received: June 13, 2007
Last updated: October 15, 2014
Last verified: October 2014

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 Intervention Phase
Nonneoplastic Condition
Biological: Rituximab
Drug: Prednisone
Phase 0

Study Type: Interventional
Study Design: Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: A Pilot Study of Rituximab in Combination With Corticosteroids for the Initial Treatment of Immune Thrombocytopenic Purpura

Resource links provided by NLM:

Further study details as provided by Mayo Clinic:

Primary Outcome Measures:
  • Failure-free survival at 6 months [ Time Frame: 6 months ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • Time to platelet recovery [ Time Frame: 1 year ] [ Designated as safety issue: No ]
  • Duration of platelet recovery [ Time Frame: 1 year ] [ Designated as safety issue: No ]
  • Effect of treatment on prevention of spontaneous bleeding events [ Time Frame: 1 year ] [ Designated as safety issue: No ]

Estimated Enrollment: 22
Study Start Date: January 2007
Study Completion Date: November 2008
Primary Completion Date: May 2008 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
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

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.


Ages Eligible for Study:   21 Years and older
Genders Eligible for Study:   Both
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
  Contacts and Locations
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Please refer to this study by its ClinicalTrials.gov identifier: 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
  More Information

Responsible Party: Mayo Clinic
ClinicalTrials.gov Identifier: NCT00486421     History of Changes
Other Study ID Numbers: CDR0000529883  P30CA015083  MC0481  2071-04  U2985s 
Study First Received: June 13, 2007
Last Updated: October 15, 2014
Health Authority: United States: Food and Drug Administration

Keywords provided by Mayo Clinic:
idiopathic thrombocytopenic purpura

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

ClinicalTrials.gov processed this record on May 26, 2016