AUTHOR=Lv Wenxiu , Chen Tianping , Wang Shen , Li Chun , Zhang Bo , Wang Liang , Xv Fang , Cao Fang , Wang Jing , Chen Li , Liao Chenglin , Li Na , Liu Hongjun TITLE=Feasibility of high-throughput drug sensitivity screening (HDS)-guided treatment for children with refractory or relapsed acute myeloid leukemia JOURNAL=Frontiers in Pediatrics VOLUME=Volume 11 - 2023 YEAR=2023 URL=https://www.frontiersin.org/journals/pediatrics/articles/10.3389/fped.2023.1117988 DOI=10.3389/fped.2023.1117988 ISSN=2296-2360 ABSTRACT=

Relapsed/refractory (rel/ref) acute myeloid leukemia (AML) has a very high mortality rate. At present, hematopoietic stem cell transplantation (HSCT) is the most effective treatment for rel/ref AML. The remission of the primary disease before HSCT is crucial for the transplantation to be effective. Therefore, it is critical to choose a suitable type of chemotherapy before HSCT. Here, we recorded the outcomes of high-throughput drug sensitivity screening (HDS) in children with rel/ref AML. Thirty-seven pediatric rel/ref AML patients who received HDS from September 2017 until July 2021 were analyzed retrospectively. Most of the patients (24 patients, 64.9%) had adverse cytogenetics. Two patients had rel/ref AML with central nervous system leukemia. The complete remission (CR) rate was 67.6%. Eight patients developed IV grade bone marrow suppression. Twenty-three patients (62.2%) underwent HSCT. The 3-year overall survival (OS) and EFS rates were 45.9% and 43.2%, respectively. Infection in the myelosuppression stage was the main cause of death. The outcome of HDS was superior to the commonly reported rates. These results suggest that HDS may be a novel treatment option for pediatric patients with rel/ref AML, and it is a promising transitional regimen prior to HSCT.