Cyclophosphamide Plus Cyclosporine in Treatment-Naive Severe Aplastic Anemia
Status:
Completed
Trial end date:
2014-09-01
Target enrollment:
Participant gender:
Summary
Background:
- Severe aplastic anemia (SAA) can lead to problems with bone marrow health and result in
low blood cell counts, which require frequent transfusions. Standard treatment for SAA
involves injections of antithymocyte globulin (ATG) plus cyclosporine (CsA). This
regimen has been shown to improve the blood counts in about two-thirds of patients.
However, the ATG/CsA regimen has the following limitations: (a) the disease can come
back (relapse) in about one-third of patients who improve initially; and (b) in about
10% to 15% of cases, certain types of bone marrow cancer (such as myelodysplasia and
leukemia) can develop (called evolution). Experience with other drugs in SAA such as
cyclophosphamide suggests that similar response rates to ATG/CsA can be achieved with a
lower risk of relapse and clonal evolution. However, cyclophosphamide was found to have
significant side effects in SAA when investigated over 10 years ago due to increase risk
of fungal infections.
- Better antibiotic drugs against fungus have been developed and are widely used to treat
patients who have low white blood cell counts and are at risk of developing infections.
In SAA patients in particular, these newer antibiotics have had a large impact in
preventing and treating fungus infections. Researchers are revisiting the use of
cyclophosphamide in SAA treatment, and plan to give a lower dose of CsA in combination
with the immune-suppressing drug cyclophosphamide, as well as antibiotics to protect
against infections, as a possible treatment for the disease.
Objectives:
- To determine the safety and effectiveness of the combination of cyclophosphamide and
cyclosporine in treating severe aplastic anemia that has not been treated with
immunosuppressive therapy.