摘要
Chronic myeloid leukemia(CML)is relatively rare in children,with an average annual incidence of 0.6 to 1.0 case per million in children<15 years and 2.2 cases per million in adolescents aged 15 to 19 years,accounting for 2%to 3%and 9%of all newly diagnosed leukemia cases in these two age groups,respectively.[1]Imatinib mesylate(IM)was approved by the US Food and Drug Administration(FDA)in 2003 and has gradually replaced hematopoietic stem cell transplantation(HSCT)as the first-line treatment for pediatric patients with chronic-phase CML(CML-CP).[2]However,IM treatment is discontinued in 25%to 29%of pediatric patients with CML-CP because of drug resistance or intolerance.[3]For such patients,second-generation tyrosine kinase inhibitors(2G-TKIs),including dasatinib and nilotinib,were approved by the FDA as first-and second-line therapies in 2017 and 2018,respectively.[2]However,given the rarity of this neoplasm and the lack of clinical trial data,treatments for pediatric CML follow the recommended adult regimen,and little is known about the long-term efficacy and safety of these treatments in children and adolescents.[2]Furthermore,there are few reports detailing the sequential use of IM as first-line treatment followed by 2G-TKIs as second-line therapy in Chinese pediatric patients with CML.Therefore,there is a strong need to investigate the long-term effects of IM treatment in a large cohort of Chinese pediatric patients.In this report,we retrospectively analyzed the long-term follow-up results of 58 pediatric patients with CML-CP treated with IM as first-line therapy and 2G-TKIs as second-line therapy in a single South China center.
基金
supported by the Construction Project of Fujian Medical Center of Hematology(No.Min201704)。