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Phase II Study of High-Dose Cyclophosphamide in Patients With Severe Autoimmune Hematologic Disease

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: NCT00010387
Recruitment Status : Completed
First Posted : February 2, 2001
Last Update Posted : September 10, 2008
Information provided by:
Office of Rare Diseases (ORD)

Brief Summary:


I. Determine the response rate and 1-year event-free survival in patients with severe autoimmune hematologic disease treated with high-dose cyclophosphamide.

Condition or disease Intervention/treatment Phase
Anemia, Hemolytic, Autoimmune Felty Syndrome Purpura, Thrombocytopenic Autoimmune Diseases Drug: cyclophosphamide Drug: filgrastim Phase 2

Detailed Description:

PROTOCOL OUTLINE: Patients receive high-dose cyclophosphamide IV on days 1-4 and filgrastim (G-CSF) starting on day 10 and continuing until blood counts recover.

Patients are followed at 1, 3, 6, and 12 months and then annually thereafter.

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Study Type : Interventional  (Clinical Trial)
Enrollment : 32 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Primary Purpose: Treatment
Study Start Date : March 1999
Actual Primary Completion Date : August 2007

Information from the National Library of Medicine

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Ages Eligible for Study:   Child, Adult, Older Adult
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No


--Disease Characteristics--

  • Diagnosis of severe autoimmune hematologic disease Autoimmune hemolytic anemia OR Immune thrombocytopenia
  • Failure of at least 2 standard treatment approaches (e.g., prednisone therapy, splenectomy, intravenous immunoglobulin, or other immunosuppressants)
  • Inability to taper prednisone dose to less than 10 mg/day OR Autoimmune neutropenia including the following: Felty's syndrome OR Disorders of large granular lymphocytes with recurrent infections or absolute neutrophil count less than 200/mm3

--Prior/Concurrent Therapy--

  • See Disease Characteristics

--Patient Characteristics--

  • Age: Not specified
  • Performance status: Not specified
  • Hematopoietic: See Disease Characteristics
  • Hepatic: Not specified
  • Renal: Creatinine no greater than 2.5 mg/dL
  • Cardiovascular: Ejection fraction at least 40%
  • Pulmonary: FVC, FEV1, or DLCO at least 50% predicted


  • Not pregnant or nursing
  • Negative pregnancy test
  • Fertile patients must use effective contraception
  • Not preterminal or moribund

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): NCT00010387

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United States, Maryland
Johns Hopkins Oncology Center
Baltimore, Maryland, United States, 21231
Sponsors and Collaborators
Johns Hopkins University
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Study Chair: Robert A. Brodsky Johns Hopkins University
Layout table for additonal information Identifier: NCT00010387    
Other Study ID Numbers: 199/15672
First Posted: February 2, 2001    Key Record Dates
Last Update Posted: September 10, 2008
Last Verified: September 2008
Keywords provided by Office of Rare Diseases (ORD):
Felty syndrome
autoimmune hemolytic anemia
autoimmune neutropenia
hematologic disorders
immune thrombocytopenic purpura
rare disease
Additional relevant MeSH terms:
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Felty Syndrome
Purpura, Thrombocytopenic
Anemia, Hemolytic
Anemia, Hemolytic, Autoimmune
Autoimmune Diseases
Pathologic Processes
Hematologic Diseases
Blood Coagulation Disorders
Skin Manifestations
Immune System Diseases
Thrombotic Microangiopathies
Blood Platelet Disorders
Arthritis, Rheumatoid
Joint Diseases
Musculoskeletal Diseases
Rheumatic Diseases
Connective Tissue Diseases
Immunosuppressive Agents
Immunologic Factors
Physiological Effects of Drugs
Antirheumatic Agents
Antineoplastic Agents, Alkylating
Alkylating Agents