Background:
People with severe aplastic anemia (SAA) do not make enough red and white blood cells, and/or platelets. Their body's immune system stops the bone marrow from making these cells. The treatment cyclosporine leads to better blood counts. But when this treatment is stopped, the disease may return in 1 in 3 people. The drug sirolimus may help by suppressing the immune system.
Objective:
To evaluate and compare the usefulness of sirolimus in preventing aplastic anemia from returning after cyclosporine is stopped, compared with stopping cyclosporine alone.
Eligibility:
People ages 2 and older with SAA who:
Have responded to immunosuppressive therapy that includes cyclosporine, and continue to take cyclosporine
Are not taking drugs with hematologic effects
Design:
Participants will be screened with:
Medical history
Physical exam
Blood and urine tests
Bone marrow biopsy: The area above the hipbone will be numbed. A thin needle will remove
some bone marrow.
Participants will be randomly assigned to a group. All will stop cyclosporine. Group 1 will take sirolimus by mouth at the same time each day for 3 months with close monitoring. Group 2 will not receive the study drug but will be monitored closely.
Participants will have clinical tests for the first 3 months:
Weekly blood test
Monthly fasting blood test
For group 1, measurements of sirolimus in the blood every 1 2 weeks
Participants will have clinic visits at 3 months, 12 months, and annually for 5 years after the study starts. They may have another visit if their SAA returns. These will include:
Blood and urine tests
Bone marrow biopsy