Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Interventional
Official Title: A Randomized Trial of Sirolimus (Rapamune) for Relapse Prevention in Patients With Severe Aplastic Anemia Responsive to Immunosuppressive Therapy
Brief Summary:
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