The Efficacy And Safety Study Of RhTPO For ITP With Different Dosing Frequency

[Objectives]To evaluate the efficacy and safety of recombinant human thrombopoietin (rhTPO) in dealing with primary (ITP) with different administration frequency.[Methods]This is a multicenter, randomized, open, parallel-group trial.20 cases(>18 years old) who is suffering from the immune thrombocytopenia was chosen as sample according strictly to the inclusion and exclusion criteria, and were randomly divided into two groups. Group A achieved continuous dosing with rhTPO dosage regimen:300 U/Kg, 1 time a day for 14 times; Follow-up of 12 weeks. Group B was treated with rhTPO dosage regimen:300 U/Kg,1 time every other day for seven times; Follow-up of 12 weeks. PLT levels were observed during the treatment and follow-up. At day 14 and week 14 after the drug was primarily used, the change of PLT level relative to the baseline value was recorded as the primary endpoint. And to assess the secondary endpoint:1) the ratio of remission(R) and complete remission(CR) patients of all subjects after 14 days’and 14 weeks’ treatment; 2) the average or median of PLT counts during the follow-up period; 3) the number of days when PLT<30xl09/L during the treatment and follow-up period. Routine physical examination, vital signs., blood routine, urinalysis, serum biochemical examination, bleeding score, electrocardiogram, blood coagulation function and other projects was recorded during the trial to assist in evaluating the efficacy and safety of the study, evaluating the incidence and severity of adverse events, outcomes, etc.[Results]11 and 9 patients were divided into A and B group respectively. In group A,1 patient lost too much observations and other 2 patients changed therapeutic schedule. In group B,1 patients were lost to follow-up due to diagnosis change. No statistical significance (P>0.05) was found between the two groups in sex, age, BMI, course of disease, prior treatment bleeding score, prior treatment PLT level, and other general condition. During treatment phase, the median value of PLT increases,74.5×109/L (0～306×109/L) and 0×109/L (-5～166×109/L) respectively, had statistical significance. The complete response rate(CRR) after initial treatment of A and B group were 50% and 12.5% respectively. The overall response rate(ORR) after initial treatment of A and B group were 87.5% and 37.5% respectively. However, no statistic significance was found between the two groups (P=0.119). There showed no significant difference of bleeding score and the days of PLT<30×109/L between the two groups. The long term rates(after 12 weeks follow-up) showed no statistical significance in either PLT change, overall response rate, or the number of days of PLT <30×109/L. No serious adverse events occurred in the final included patients both in group A and in group B.[Conclusions]rhTPO can rapidly enhance the level of platelet and has good curative effect for adult ITP; Compared with alternate-day dosing, continuous dosing has better effect enhancing the level of platelet, but there is no statistical difference in complete response rate and overall response rate. Similar 3-month remission rate and long-term curative effects are found in these two kinds of dosage regimen. What’s more, there is no statistical difference in treatment safety and bleeding risk between the two groups.