A Single-Arm Phase 2 Study With Optimized Standard Protocol for Severe Aplastic Anemia
Status:
Unknown status
Trial end date:
2017-09-01
Target enrollment:
Participant gender:
Summary
Severe acquired aplastic anaemia (SAA) is a bone marrow failure disease characterized by
pancytopenia and a hypocellular bone marrow. The corn pathophysiological mechanism is the
destruction of hematopoietic stem/progenitor cells mediated by auto-reactive effector T
cells. Immunosuppressive therapy with horse antithymocyte globulin (ATG) plus cyclosporine
(CSA) is currently the standard of treatment in patients with aplastic anaemia who are not
eligible for bone marrow transplantation and with response rates from 40% to 70%. Previous
studies showed that horse ATG (hATG) is apparently more effective than rabbit ATG (rATG) as
the latter has higher treatment related mortality (TRM). Unfortunately hATG is unavailable in
China, so we conduct a optimized standard treatment (9 days protocol) of rATG plus CSA and
Levamisole (LMS) Sequential maintaining (termed Optimized Standard Protocol, OSP) for severe
aplastic anemia. This prospective study is designed to evaluate the efficacy and safety of
Optimized Standard Protocol as first line therapy in newly diagnosed severe aplastic anemia
patients.