A Randomized Trial of Antithymocyte Globulin Versus Cyclosporine to Treat the Cytopenia of Myelodysplastic Syndrome
Status:
Completed
Trial end date:
2000-05-01
Target enrollment:
Participant gender:
Summary
Approximately 40% of the patients with myelodysplastic syndrome (MDS) die as a consequence of
their cytopenia. As in aplastic anemia, the cytopenia of MDS may be partly due to cytotoxic T
cell activity. Immunosuppressive therapy may therefore reverse the cytopenia. In a phase II
pilot study of anti-thymocyte globin (ATG) to treat myelodysplastic syndrome (MDS); 41% of
patients (61% of patients with refractory anemia) have responded in terms of transfusion
independence. Recently, Jonasova et al [32] reported a 82% substantial hematological response
rate in 18 patients with MDS of the refractory anemia (RA) subtype treated with cyclosporine
alone. Just over 50% of the patients in this series had MDS of the hypocellular type.
Cyclosporine alone if indeed efficacious would be a powerful therapeutic option that could be
readily used by hematologists in the community to treat patients with MDS. This efficacy
needs to be proven in a larger study which includes patients with the other subtypes of MDS
and more patients with the non-hypocellular forms of MDS (which constitute approximately 70%
of the cases in the community). As MDS is a heterogeneous group of disorders, a randomized
comparison with the other immunomodulating intervention of proven benefit, ATG, is
appropriate. In this randomized study patients with MDS will receive either ATG alone or
cyclosporine alone.
Phase:
Phase 2
Details
Lead Sponsor:
National Institutes of Health Clinical Center (CC)