Xosptate pediatric use – pro
To date, FLT3 inhibition as a treatment strategy in pediatric AML has been hampered by limited potency and significant toxicity, mainly because of off-target effects. This is the case with FLT3 inhibitors that could be used for pediatric patients, such as sorafenib and midostaurin.14,15 Sorafenib, although deemed effective and more tolerable than expected in pediatric patients, has been associated with significant toxicities. Midostaurin, currently under more extensive evaluation, has shown limited clinical activity based on preliminary data. Gilteritinib, a highly potent and selective oral FLT3 inhibitor, has shown promising results in adults with relapsed/refractory (R/R) FLT3-mutated AML and frontline in combination with induction therapy. This has led to the incorporation of this agent in the new Children’s Oncology Group frontline AAML1831 trial (registered at www.clinicaltrials.gov as #NCT04293562). As such, it remains experimental.
Perl AE Martinelli G et al, Gilteritinib or chemotherapy for relapsed or refractory FLT3-mutated AML. N Engl J Med.2019 381(18): 1728 – 1740
Xospata Prescribing Information 2022
David McCall, et al, Gilteritinib combination therapies in pediatric patient with FLT3-mutated acute myeloid leukemia. Blood Adv (2021) 5 (23): 5215–5219.