Abstract：Objective To explore the effcacy and safety of recombinant human thrombopoietin (rhTPO) combined with high-dose dexamethasone (DXM) in the treatment of children with refractory immune thrombocytopenic purpura (ITP).Methods Fifty-eight ITP children who had failed frst-line therapy were randomly divided into two groups:DXM treatment (n=27) and rhTPO + DXM treatment (n=31). The DXM treatment group received two continuous cycles of DXM treatment; in each cycle, patients received high-dose DXM (0.6 mg/kg daily) by intravenous drip for 4 days every 28 days. The rhTPO group received subcutaneous injection of rhTPO (300 U/kg daily) for 14 days additional to DXM treatment. The overall response rate (marked response rate+slight response rate) and adverse reactions were evaluated after 3, 7, and 14 days and 1, 2, and 3 months of treatment.Results After 7 and 14 days and 1 month of treatment, the rhTPO+DXM treatment group had a signifcantly higher marked response rate and a signifcantly higher overall response rate than the DXM treatment group (P < 0.05). After 2 months of treatment, the rhTPO + DXM treatment group had a signifcantly higher overall response rate than the DXM group (P < 0.05). One patient in the DXM treatment group had liver damage during the frst week of treatment. There was no hypertension, fever, rash, allergy, or weakness in the two groups.Conclusions rhTPO combined with high-dose DXM is an effective and safe approach for treating refractory ITP.
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