Mycophenolate Mofetil and Cyclosporine to Treat Relapsing Aplastic Anemia

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Brief Title

Mycophenolate Mofetil and Cyclosporine to Treat Relapsing Aplastic Anemia

Official Title

A Randomized Trial for the Treatment of Relapsing Aplastic Anemia With Mycophenolate Mofetil (MMF) and Cyclosporine (CSA)

Brief Summary

      This study will examine the safety and effectiveness of a new drug combination for treating
      patients with severe aplastic anemia. Patients with aplastic anemia produce too few blood
      cells, causing fatigue, easy bruising and bleeding, and susceptibility to infections. In many
      cases, the very low blood counts result from an autoimmune process-that is, the patient's own
      immune system suppresses production of blood cells by the bone marrow. Although
      immune-suppressing drugs, such as cyclosporine, can restore normal cell counts, many patients
      have disease relapses. These patients require long-term therapy with cyclosporine, which can
      cause harmful side effects. This study will examine whether a lower dose of cyclosporine
      given together with mycophenolate mofetil (MMF) can maintain blood counts as effectively as
      full-dose cyclosporine treatment, and whether MMF alone can reduce the chances of future
      relapses.

      Patients 4 years of age and older with severe aplastic anemia who have relapsed after immune
      suppressing therapy may be eligible for this study. Participants will be randomly assigned to
      receive either standard cyclosporine therapy or experimental therapy with cyclosporine and
      MMF.

      Patients receiving standard cyclosporine therapy will receive a full dose of the drug for at
      least 3 months. Those taking both cyclosporine and MMF will take MMF plus half-dose
      cyclosporine for 3 months and continue MMF for an additional 6 months. Both drugs are taken
      twice a day by mouth. All patients will have about 120 milliliters (4 ounces) of blood drawn
      at the beginning of the study to evaluate immune system activity and bone marrow function,
      and to look for genetic material of certain viruses. Bone marrow aspirations and biopsies
      will be done at the beginning of the study, and at 6 and 12 months. For these tests, the area
      of the hip is anesthetized and a special needle is used to draw bone marrow from the hipbone.

      The patient's local doctor will be asked to do blood tests for chemistries, liver function
      and cyclosporine levels weekly for the first month and then every other week. Patients will
      return to NIH for evaluations 3, 6 and 12 months after treatment and then once a year. About
      100 ml (7 tablespoons) of blood will be drawn at each visit.
    

Detailed Description

      Aplastic anemia is characterized by trilineage hematopoietic failure with an apparently empty
      bone marrow. While the precise mechanism of disease has yet to be elucidated, much evidence
      indicates an immunologically mediated pathophysiology. Clinical trials have shown that
      approximately 75-80% of patients who are treated with immunosuppressive drugs, especially the
      combination of antithymocyte globulin (ATG) and cyclosporine (CSA), demonstrate a return of
      hematopoieses and improved blood counts. This therapy now is considered standard care for the
      treatment of aplastic anemia in all patients who lack a histocompatibility antigen-matched
      sibling donor and also in older patients regardless of donor status. However, with longer
      length of evaluation of patients after initial treatment, it is becoming increasingly clear
      that a substantial proportion will suffer relapse of pancytopenia. ATG and CSA do not appear
      to cure the disease in these patients but only disrupt a chronic autoimmune process. Recent
      data from our own series of patients treated with ATG and CSA, and studies of European
      patients who are treated with antilymphocyte globulin (ALG) and CSA, indicate that
      approximately 1/3 of responding patients will relapse and require treatment within 1-2 years
      of discontinuation of CSA. About 15% of patients become dependent on continued CSA
      administration in order to maintain blood counts. Chronic CSA toxicities include increased
      susceptibility to infections, hypertension, and irreversible renal damage, as well as
      hypertrichosis, hyperaesthesias, gingival hyperplasia, headaches, tremors, and other
      troubling complaints; there is also a possible increased risk of late malignant diseases.
      Therefore, a major priority in clinical research in aplastic anemia is the development of
      strategies to produce more durable responses, as well as to identify immunosuppressive agents
      that can be used as effectively and with fewer side affects than cyclosporine. Mycophenolate
      mofetil (MMF) is a novel immunosuppressive drug with proven efficacy in the treatment of
      graft rejection in renal transplantation. MMF has a different toxicity profile from
      cyclosporine, and specifically does not damage the kidneys. In this study, we will randomize
      patients who are judged to have relapsed to receive either standard treatment with full dose
      cyclosporine or half dose cyclosporine combined with MMF. We anticipate that the combination
      of cyclosporine with MMF will be as effective as conventional high dose cyclosporine for the
      purpose of treating relapse of aplastic anemia and would provide a less toxic regimen for
      long-term treatment of this disease.
    

Study Phase

Phase 2

Study Type

Interventional




Condition

Aplastic Anemia

Intervention

Mycophenolate mofetil


Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information


Recruitment Status

Drug

Estimated Enrollment

130

Start Date

June 2000

Completion Date

March 2002


Eligibility Criteria

        Subjects with a history of severe aplastic anemia successfully treated by immunosuppression
        will be included.

        Subjects with relapse, as defined above by either return of blood counts to satisfy
        criteria for severity or consistently declining blood counts will be included.

        Subjects age 4 and above will be included.

        Subjects with the presence of a medical or surgical condition making survival for at least
        3 months unlikely will be excluded.

        Subjects with inability to confer informed consent or assent, in the case of a child,
        either written or verbal, will be excluded.
      

Gender

All

Ages

N/A - N/A

Accepts Healthy Volunteers

No

Contacts

, , 

Location Countries

United States

Location Countries

United States

Administrative Informations


NCT ID

NCT00005935

Organization ID

000157

Secondary IDs

00-H-0157


Study Sponsor

National Heart, Lung, and Blood Institute (NHLBI)


Study Sponsor

, , 


Verification Date

March 2002