Results from a phase I/II single-arm trial suggest that blinatumomab may have an antileukemic effect in children with relapsed/refractory B-cell precursor acute lymphoblastic leukemia (BCP-ALL).
ALL is a progressive cancer of the blood and bone marrow that, although rare in adults, is the most common type of cancer in children. Between 15% and 20% of patients diagnosed with ALL will experience relapse. The prognosis for these patients is extremely poor, as only 40-50% achieve post-relapse survival.
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The open-label trial enrolled 49 patients in phase I and 44 patients in phase II. All patients were under the age of 18 years and diagnosed with relapsed or refractory BCP-ALL. The primary endpoints were maximum-tolerated dosage for the phase I trial and complete remission within the first two treatment cycles for the phase II trial.
Overall, of the 70 patients who received the recommended dosage of blinatumomab, 27 patients (39%) achieved complete remission within the first two cycles. Fourteen of these patients also achieved minimal residual disease response.
Further, treatment with blinatumomab was also generally well tolerated. The most frequent grade 3 or higher adverse events reported by patients who received the recommended dosage were anemia (36%), thrombocytopenia (21%), febrile neutropenia (17%), hypokalemia (17%) and neutropenia (17%). The most common adverse events overall were pyrexia (80%), anemia (41%), nausea (33%) and headache (30%).
From these results researchers concluded that blinatumomab demonstrated antileukemic activity in patients with BCP-ALL and that the drug may represent a promising new treatment option in this needed setting. More investigation on the drug for other indications is needed.
"Pediatric patients with relapsed or refractory Ph- B-cell precursor ALL are in critical need of new treatment options," added Sean E. Harper, MD, executive vice president of Research and Development at Amgen, the company that produces blinatumomab. "The publication of this data in the Journal of Clinical Oncology provides clinical evidence of the potential of BLINCYTO in this patient population and underscores the significance of the recent regulatory approval for use of BLINCYTO in these patients."
