Association of Gene Mutations with Response to Arsenic-Containing Compound Qinghuang Powder (复方青黄散) in Patients with Myelodysplastic Syndromes

To investigate the relationship between gene mutations and response to Compound Qinghuang Powder (复方青黄散, CQHP) in patients with myelodysplastic syndrome (MDS). Forty-three MDS patients were genotyped by ultra-deep targeted sequencing and the clinical data of patients were collected and the relationship between them was analyzed. Up to 41.86% of patients harbored genet mutations, in most cases with more than one mutation. The most common mutations were in SF3B1, U2AF1, ASXL1, and DNMT3A. After treatment with CQHP, about 88.00% of patients no longer required blood transfusion, or needed half of prior transfusions. CQHP is an effective treatment for patients with MDS, especially those with gene mutations in SF3B1, DNMT3A, U2AF1, and/or ASXL1.