Xospata Plus Chemo Safe, Effective for AML Subset

Treatment with Xospata (gilteritinib) was shown in a recent study to be safe and effective when utilized with a chemotherapy regimen in patients with relapsed or refractory acute myeloid leukemia (AML) who have a FLT3 mutation or as single-agent maintenance therapy.

The study, results of which were published in The Journal of Clinical Oncology, determined the recommended regimen to be 120 milligrams of Xospata daily from days four to 17 or eight to 21 of a 7 + 3 induction — which, as explained by the American Cancer Society on its website, would include a pair of chemotherapy drugs: a short infusion of an anthracycline treatment such as idarubicin or daunorubicin on each of the first three days and continuous high-dose cytarabine consolidation for seven days. The authors noted that maintenance therapy with Xospata was well-tolerated.

“These results demonstrated the safety and tolerability of (Xospata) incorporated into an induction and consolidation chemotherapy regimen and as single-agent maintenance therapy for patients with newly diagnosed FLT3-mutant AML,” the authors wrote. “The data herein provide an important framework for the design of randomized trials comparing (Xospata) with other FLT3 inhibitors.”

In this study, there were 58 participants — 36 of whom had FLT3 mutations — evaluable for responses to the 120 milligram a day dose. Patients with FLT3-mutated AML showed a composite complete response rate of 89%, with 83% as conventional complete responses. Of note, these responses were all attained in one induction cycle, according to the authors, who noted that the median overall survival time (the time following treatment that a patient is still alive) was 46.1 months.

Xospata was “well-tolerated” in that context, the authors wrote, with a median time of 40 days for blood count recovery during induction.

According to the study’s introduction, the current standard of care for patients with newly diagnosed FLT3-mutated AML is induction and consolidation chemotherapy with midostaurin. Researchers noted that prolonged maintenance therapy with this approach is often poorly tolerated and does not often result in an overall survival benefit.

Xospata was approved by the Food and Drug Administration (FDA) in 2018 as the first treatment of adults with relapsed or refractory AML who have a FLT3 mutation that is detected by an FDA-approved test, LeukoStrat CDx FLT3 Mutation Assay.

In a press release issued at the time of the approval, Xospata was touted as “a new, highly effective and well-tolerated treatment option … for a group of truly high-risk patients who, until today, had no specific therapies available beyond chemotherapy to treat their disease” said Dr. Alexander Perl, an associate professor of hematology-oncology in the Perelman School of Medicine at the University of Pennsylvania and the Abramson Cancer Center.

Common side effects with Xospata reported at the time of its approval included muscle and joint pains, transaminase increase, fatigue, fever, noninfectious diarrhea, shortness of breath, swelling, rash, pneumonia, nausea, stomatitis (painful swelling and sores inside the mouth), cough, headache, low blood pressure, dizziness and vomiting.

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