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Selinexor in Refractory Oligoblastic AML: Phase II Trial Findings

By: Susan Reckling
Posted: Tuesday, September 1, 2020

Effective treatments for patients with high-risk myelodysplastic syndromes are sorely needed, with no standard therapy currently available. A recent single-center, single-arm phase II trial explored the use of the selective inhibitor of nuclear export selinexor in patients with oligoblastic acute myeloid leukemia (AML) or myelodysplastic syndromes (MDS) whose disease was refractory to hypomethylating agents. According to Virginia M. Klimek, MD, of Memorial Sloan Kettering Cancer Center, New York, and colleagues, this novel targeted therapy demonstrated activity, with adverse events being manageable with supportive care alone. Their early study findings were published in The Lancet Haematology.

“We therefore propose that further development of XPO1 inhibitors for [MDS] is warranted and recommend including more patients with splicing factor mutations in future trials to confirm this genotype as a predictor of response,” commented the investigators.

A total of 25 patients were enrolled in this study between September 2014 and March 2018. All patients had high-risk MDS or oligoblastic AML, which was defined as blasts between 20% and 30%. The dosage regimen consisted of 60 mg given orally twice a week for 2 weeks, followed by 1 week off.

Of the 23 evaluable patients, the overall response rate was 26% (95% confidence interval [CI] = 10%–48%) in 6 patients with marrow complete remission. An additional 12 patients (52%; 95% CI = 31%–73%) achieved stable disease. In regards to toxicity, the most common grade 3 or 4 adverse events were thrombocytopenia (8 of 25 patients) and hyponatremia (5 of 25 patients). No drug-related serious adverse events nor treatment-related deaths were reported.

“Further studies are needed to compare selinexor with supportive care alone and to identify patient subgroups that might benefit the most from selinexor treatment,” concluded the study authors.

Disclosure: For full disclosures of the study authors, visit thelancet.com.



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