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.