Efficacy and safety of short course high-dose Dexamethasone as first-line treatment regimen for childhood newly diagnosed immune thrombocytopenia / 中华实用儿科临床杂志

ABSTRACT

Objective To compare the efficacy and safety of short course and high-dose Dexamethasone (HDD) and conventional Prednisone as first-line strategy for children newly diagnosed as primary immune thrombocytopenia (ITP).Methods This study analyzed pre-experimental data of a single center prospective randomized controlled clinical trial.Newly diagnosed but untreated ITP patients enrolled at the Department of Blood and Cancer Center,Beijing Children's Hospital,Capital Medical University from November 2016 to May 2017 were randomized into HDD group[Dexamethasone 0.6 mg/(kg · d),intravenous injection for 4 days] and Prednisone group [Prednisone 2 mg/(kg · d) for 14-28 days and then tapered within 1-2 months,the course of treatment less than 3 months].Initial response,sustained response and adverse effects after therapy were observed in 2 groups.Results Sixty-six children with ITP were included in the study32 patients were in the HDD group and 34 patients were in the Prednisone group.Two groups were matched in the baseline characteristics including gender,age,platelet counts and disease course before therapy and bleeding assessment (all P ＞ 0.05).The initial response (the response of HDD group within 10 days of treatment and Prednisone group within 28 days of treatment)overall initial response had no statistical difference between the HDD group and the Prednisone group[90.6％ (29/32 cases) vs.100.0％ (34/34 cases),x2 =1.528,P ＞ 0.05].HDD group had a lower incidence of complete response compared with that in the Prednisone group [54.4％ (19/32 cases) vs.94.1％ (32/34 cases),x2 =11.330,P =0.001];median time of response in two groups showed no statistically difference (2 d vs.1 d,Z =-0.149,P ＞ 0.05).There was no significant difference in the recovery of skin and mucosal bleeding after treatment between the Dexamethasone group and the Prednisone group (Z =-1.413,-1.031,all P ＞ 0.05).The sustained response (the response lasted for up to 6 months and above)overall and complete sustained response had no statistically difference between the HDD group and the Prednisone group [92.9％ (26/ 28 cases) vs.85.3％ (29/34 cases),P =0.594;78.9％ (15/19 cases) vs.81.3％ (26/32 cases),P=1.000].Log-rank test showed no significant difference in the duration of response between 2 groups (P =0.341).The side effects in the Prednisone group included weight gain or Cushing sign (94.1％) and mental and emotional changes (23.5％);in the HDD group 15.6％ of children had infection,without other glucocorticoid-related side effects.There was no significant difference in the incidence of infection between two groups[15.6％ (5/32 cases) vs.26.5 ％ (9/34 cases),P =0.281].All of the above infections were of respiratory tract infections and mild gastrointestinal infections.Conclusions Efficacy of the HDD group in the initial and sustained responses is similar,but side effects were apparently lower compared with that in the Prednisone group.However,a large multicenter randomized controlled clinical study is necessary to confirm this result.