A multicentre randomized open‐label phase 2 trial of dexamethasone plus glycyrrhizinate versus dexamethasone in newly diagnosed immune thrombocytopenia

Summary Standard first‐line therapy with dexamethasone requires further optimization due to unsatisfying long‐term outcomes in patients with primary immune thrombocytopenia (ITP). Previous research suggested that glycyrrhizinate induces platelet responses in ITP murine models. This study aimed to compare the efficacy and safety of dexamethasone plus glycyrrhizinate with dexamethasone in patients with newly diagnosed ITP. Eligible patients aged ≥18 years with newly diagnosed, treatment‐naïve ITP who had baseline platelet count of <30 × 10 9 /L were enrolled during routine outpatient visits. Recruited patients were randomly assigned 1:1 to receive either dexamethasone plus glycyrrhizinate ( n = 55) or dexamethasone ( n = 56). The modified intention‐to‐treat analysis included 96 patients (48 vs. 48) who received at least one dose of allocated treatments. The primary end‐point overall response, defined as a platelet count of ≥30 × 10 9 /L, at least doubling of baseline platelet count and no bleeding, was reached in more patients in combination group [56.25% (27/48)] than that in the monotherapy group [31.25% (15/48)] at month 6 (* p = 0.0231), with a longer duration of response, but not at day 14. The most common treatment‐emergent adverse events were gastrointestinal reactions and anxiety. No grade 3–5 adverse events or deaths occurred. Dexamethasone plus glycyrrhizinate was an optimized initial therapy in ITP (NCT 05023915).