Sorafenib for FLT3+ AML

Sorafenib (Nexavar,) has been approved for the treatment of metastatic renal cancer and advanced hepatocellular carcinoma. Among other biological actions, is the ones against FLT3-RTK and FLT3-ITD, suggesting that it may have a role in treating AML cases that have FLT3 mutations. Sorafenib in a phase 1 clinical trial on 16 patients with AML was found to be active in 6 of the 7 Flt3-ITD–positive patients. However, treatment duration was short (21-70 days), and no durable responses were reported. Notably, a complete molecular remission has recently been reported in a patient relapsing after stem cell transplantation (SCT).

In summary, there is a high degree of interest in the use of sorefenib with other drugs or alone for FLT+ AML but corroborating literature is still very preliminary and at the level of case reports. For this reason, it is still experimental at this time.

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