Severe acquired aplastic anaemia (SAA) is a life-threatening disease characterized by pancytopenia and hypoplastic bone marrow. Immunosuppressive treatment with antithymocyte globulin (ATG)and cyclosporine remain the standard regimen with response rates of 70% or more and excellent overall survival. However ,there are no clinical trials to illustrate the response and complete remission rate with different doses of ATG.And there are no data reported on children with SAA so far.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
100
Drug ATG:2.5mg/kg/d or 3.75mg/kg/d for 5 days; Drug Cyclosporine A (CSA):3-10mg/kg/d ,Adjust the dose to maintain drug levels between 150 and 300ng/ml; Drug prednisone:1mg/kg/d,d1-21 from the first dosage of ATG; Drug Granulocyte Colony-Stimulating Factor(G-CSF):5ug/kg/d until absolute neutrophil count (ANC) \>1×109/L.
Department of Pediatrics, Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences
Tianjin, Tianjin Municipality, China
RECRUITINGthe response and complete remission rate with different doses of ATG to treat child severe aplastic anemia
Complete response (CR) was defined as achieving normal levels of hemoglobin adjusted for age, platelet count \>100×109/L, and ANC\>1.5×109/L. Partial response (PR) was defined as transfusion independence, reticulocyte count \>30×109/L, platelet count \>20×109/L, and ANC \>0.5×109/L above the baseline. Persistence of transfusion requirement or death was evidence of no response (NR).
Time frame: 1 years
the relapse rate with different doses of ATG to treat child severe aplastic anemia
relapse was defined as transfusion dependence again; or progressed to paroxysmal nocturnal hemoglobinuria (PNH) /acute myeloid leukemia/myelodysplasia syndrome (MDS); or CSA dependence
Time frame: 4-10 years
This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.