作者: Katherine Tarlock , Bill Chang , Todd Cooper , Thomas Gross , Sumit Gupta
DOI: 10.1002/PBC.25437
关键词:
摘要: Background FLT3/ITD is associated with poor outcomes in adult and pediatric acute myeloid leukemia (AML). Allogeneic hematopoietic stem cell transplantation (HSCT) can improve cure rates, however relapse still common. Recent studies demonstrate the activity of FLT3 inhibitors, including sorafenib, targeting underlying mutation. Procedure We conducted a retrospective study 15 patients FLT3/ITD+ AML treated sorafenib within 18 months after receiving HSCT. Sorafenib was administered either as prophylaxis considered at very high risk for (n = 6) or time disease recurrence (n = 9). Results Sorafenib initiated median 100 days post Overall, 11/15 (73%) experienced medically significant toxicities. Among who toxicity, 6/11 (55%) received treatment doses above what later determined to be maximum tolerated dose leukemia. Importantly, did not appear exacerbate graft versus host disease. Our findings suggest that may particular efficacy minimal residual (MRD); all MRD immediately prior transplant emergence post-HSCT are alive remain complete remission 48 HSCT. Conclusions Our case series suggests administration feasible tolerable early Ongoing prospective controlled needed further define dosing period determine optimal context this approach. Pediatr Blood Cancer 2015;62:1048–1054. © 2015 Wiley Periodicals, Inc.