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

Objectives: 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). Methods: 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. Results: 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. Conclusions: CQHP is an effective treatment for patients with MDS, especially those with gene mutations in SF3B1, DNMT3A, U2AF1, and/or ASXL1. [ABSTRACT FROM AUTHOR]