含砷中药复方治疗骨髓增生异常综合征多中心随机对照研究OA

Objective To evaluate the clinical efficacy and safety of arsenic-containing Chinese medicine(CM)compound(Qinghuang Powder combined with Bushen Jianpi Formula)combined with Western medicine in the treatment of myelodysplastic syndromes(MDS).Methods A multicenter,double-blind,randomized controlled trial was performed.A total of 138 MDS patients were assigned to experimental group and control group,69 in each group.Both groups received conventional treatment.The experimental group received Bushen Jianpi Formula and Qinghuang Powder additionally,while the control group received a CM placebo.Each treatment course lasted for 3 months,with continuous observation over 3 courses.The primary outcome was hematological improvement[including red blood cell count(RBC),hemoglobin(HGB),mean corpuscular volume(MCV),red cell distribution width-coefficient of variation(RDW-CV),platelet count(PLT),platelet distribution width(PDW),white blood cell count(WBC),and absolute neutrophil count(ANC)].The secondary indicator was efficacy of CM syndrome.Safety indicators and adverse events were also recorded in both groups.Results A total of 126 patients completed the trial,including 64 in the experimental group and 62 in the control group.The total effective rate in the experimental group was superior to that in the control group(92.8％vs.71.0％,P＜0.001).Erythroid response and platelet response were also better in the experimental group(P＜0.05).In the experimental group,HGB levels increased after each treatment course compared to baseline(P＜0.05)and were superior to those in the control group at the corresponding time points(P＜0.001).PLT levels in the experimental group increased after two treatment courses compared to baseline(P＜0.05).In the control group,ANC levels decreased after treatment(P＜0.05).The efficacy of CM syndrome improvement in the experimental group was superior to that in the control group(P＜0.001).No serious adverse events related to the treatment were observed during the trial.Conclusions The combination of arsenic-containing CM compounds with Western medicine can improve erythroid and platelet responses,alleviate TCM syndromes,and enhance the quality of life in MDS patients.This approach demonstrates superior efficacy compared to Western medicine alone and exhibits a favorable safety profile.(Chinese Clinical Trial Registry No.ChiCTR2100051306)