The efficacy and safety of Huaiqihuang Granule in the treatment of childhood chronic primary immune thrombocytopenia: a multi-centre, randomized, double-blind, placebo-controlled clinical study (Han et al., 2024)

Ethnopharmacological relevance: Chronic immune thrombocytopenia (CITP) seriously affects the patients' quality of life. In the real world, a traditional Chinese medicine, Huaiqihuang Granule (HQH) shows certain value in ITP treatment.It primarily comprises Huai'er (Trametes robiniophia Murr), Gouqizi (Lycium chinense Mill), Huangjing (Polygonatum sibiricum F. Delaroche). Aim of the study: This study aims to investigate the clinical efficacy and safety of HQH in children with CITP. Materials and methods: A multi-centre, randomized, double-blind, placebo-controlled clinical trial was conducted among 216 children with CITP from June 27, 2017 to October 14, 2023, at 13 hospitals in China. Patients were followed up for 96 weeks. Patients with CITP were treated with HQH or with matching placebo in a randomized, double-blind, 24-week treatment period. Patients who received placebo and completed 24 weeks of treatment switched to receive HQH, and patients treated with HQH in the double-blind period continued HQH during the following open-label 24-week treatment. Results: HQH was superior to placebo in decreasing the bleeding grade and achieving a platelet response. The total clinical effectiveness rate of HQH group (71.74 % in 24-week) was higher than that of the placebo group (45.65 % in 24-week, p = 0.0013). The result was similarly seen when the data of patients with rescue treatment was analyzed as ineffectiveness or excluded. AEs and serious AE was comparable between the two groups. The severe AEs included thrombocytopenia (6.9 %), respiratory tract infection (5.1 %) and hemorrhagic episodes (2.8 %). Conclusions: In CITP children, HQH can improve the clinical effectiveness especially the degree of bleeding, and shows good safety even with long-term treatment.