To study the efficacy and safety of recombinant human thrombopoietin (rhTPO) combined with dexamethasone as front line regimen in patients with primary immune thrombocytopenia (ITP).

This study was a prospective, randomized, controlled trial. A total of 59 primary ITP patients were enrolled at the First Affiliated Hospital, Xinjiang Medical University from June 2013 to February 2015. All subjects were randomized into study group (30 cases) and control group (29 cases). The study group was scheduled to receive high-dose dexamethasone (40 mg intravenously d1-4) combined with rhTPO (300 U·kg^-1·d^-1 subcutaneously d1-14). Once absolute platelet count reached >50×10 ⁹/L, rhTPO stopped. Patients in control group were just administrated with high-dose dexamethasone (40 mg intravenously d1-4). Efficacy and adverse reactions were evaluated.

The short-term (15 days) and mid-term (3 months) response rates in the study group were 83.3%(25/30) and 76.7%(23/30) respectively, which were both significantly better than those in the control group [51.7%(15/29) and 20.7%(6/29) respectively] (P<0.01). In the study group and control group, the median time platelet count reached 100×10⁹/L was 6.0 and 6.8 days respectively. In the study group, the time of TPO usage was (6.1±1.7) days. The incidence of adverse reactions in both groups was comparable and slight. The most common TPO related adverse events included knee ache and fatigue, which accounted for 6.7% (2/30) in the study group.

Recombinant human TPO combined with dexamethasone as front line treatment for primary ITP shows significant advantages in both short-term and mid-term responses with less and manageable adverse reactions. This may provide a new method to treat patients with primary ITP.

Thrombopoietin; Immune thrombocytopenia; Treatment outcome; Adverse reactions