The Efficacy and Safety of Huaiqihuang Granules in the Treatment of Childhood Chronic Primary Immune Thrombocytopenia: A Multicentre, Randomized, Double-Blind, Placebo-Controlled Clinical Study

The efficacy and safety of Huaiqihuang Granules in the treatment of childhood chronic primary immune thrombocytopenia: a multicentre, randomized, double-blind, placebo-controlled clinical study

* Correspondence:

Fen Zhou, daisy_may@163.com

Runming Jin, jinrunm@qq.com

Abstract

Background: Chronic immune thrombocytopenia (CITP) is a hemorrhagic disease that seriously affects the quality of life of children and their families. Huaiqihuang Granules, a traditional Chinese medicine, has certain value in the treatment of ITP patients in the preliminary clinical findings. To investigate the efficacy and safety of Huaiqihuang Granules in children with CITP, we conducted a multicenter, randomized, double-blind, placebo-controlled study.

Methods: Patients with CITP were treated with Huaiqihuang Granules 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 Huaiqihuang Granules, and patients treated with Huaiqihuang Granules in the double-blind period continued Huaiqihuang Granules during the following open-label 24-week treatment.The patients were followed up for 96 weeks. Clinical effective (defined as those achieving a platelet count of≥30×10⁹/L and at least twofold more than the basal platelet count, or the degree of bleeding decreased by one or more grade) was evaluated.

Results: Huaiqihuang Granules was superior to placebo in achieving a platelet response and in reducing the bleeding risk. The clinical effective rate of Huaiqihuang Granules group (71.74% in 24-week) was higher than that of the Placebo group (45.65% in 24-week, p=0.0013). The most common severe adverse events were respiratory tract infection (7.8%) and immune thrombocytopenic purpura (6.9%) .

Conclusions: In CITP children, Huaiqihuang granules can improve the clinical effectiveness especially the degree of bleeding, and shows good safety in long-term treatment.

Keywords: Chronic immune thrombocytopenia, Huaiqihuang Granules, efficacy; safety

No relevant conflicts of interest to declare.