Objective To explore the clinical efficacy and safety of dabrafenib as first-line therapy in multisystem infants LCH with BRAFV600E-mutation.Methods The clinical data of 11 infants with multisystem LCH with BRAFV600E-mutation in Beijing Children's Hospital from January 2020 to December 2022 was retrospectively analyzed,including the effective rate,adverse reactions and recurrence rate.Results Six males and 5 females were included in this study.The median age of diagnosis was 10.5 months(range,2.0-20.8months).The median time on dabrafenib was 23 months(range,14-36 months).The response rate was 100％.After treatment,the involvements of risk organs were completely recovered,3/4 children with pituitary involvement recovered.The median follow-up time was 38.2 months(range,14-51.8 months).All the other 10 patients were alive except for one child who was lost to follow-up.Among the 6 children who finished treatment completely,four cases relapsed.The 3-year event-free survival rate was 71.4％(95％confidence interval:35.9-91.8).During the treatment of dabrafenib,skin rash occurred in 4 cases and myocardial damage occurred in one case,all symptoms were improved after symptomatic treatment.Conclusions Multisystem infant LCH with BRAFV600E-mutation had a high response rate when treated with frontline dabrafenib,and the drug-related side effects were tolerable.However,the recurrence rate was high.To achieve long-term remission,prolonged course of dabrafenib or subsequent chemotherapy need to be further explored.
维普
万方数据