Treatment regimen
IST products for AA in our center included rATG and CSA from March 2013 to July 2020. Before rATG administration, the patients received a subcutaneous test dose to assess potential hypersensitivity. rATG dose was 3.3 – 3.5 mg/kg/day, administered by intravenous injection for 5 consecutive days. Methylprednisone at 2 mg/kg/day was injected intravenously for 5 days (before each rATG), followed by oral prednisone, which was gradually decreased within 14 days. From day 1, G-CSF was injected subcutaneously to maintain granulocytes at 1.0×109/L; from day 1, CSA was administered orally at a dose of 5 mg/kg/day to maintain the serum trough concentration at about 100-200 mg/dl. Serum CSA levels were measured every 2-4 weeks during drug administration. CSA was administered at full dose for at least 1.5 years, with a decrease at 1.5 years, unless dose reduction or frequent interruption was needed due to toxicity. The selected patients were infused blood products according to institutional policies.
We added eltrombopag to the standard IST since June 2017. The specific usage was as follows: eltrombopag was administered orally on an empty stomach at a dose of 75 mg daily in patients ≥6-years old and 2.5 mg/kg body weight/day in 2-5-year-old individuals. At platelet count > 300×109/L or toxicity considered to be related to eltrombopag, the drug was discontinued until platelet count dropped to < 100 × 109/L. For responders not meeting the above criteria, eltrombopag was continued indefinitely or stopped at the discretion of the attending physician.