Mycophenolate Mofetil and Cyclosporine to Treat Relapsing Aplastic Anemia
Status:
Completed
Trial end date:
2002-03-01
Target enrollment:
Participant gender:
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.