Phase I/Phase II Study of Blinatumomab in Pediatric Patients With Relapsed/Refractory Acute Lymphoblastic Leukemia

A. Von Stackelberg, F. Locatelli, G. Zugmaier, R. Handgretinger, T. M. Trippett, Carmelo Rizzari, P. Bader, Louise M. O'Brien, Benoît Brethon, D. Bhojwani, P. G. Schlegel, Arndt Borkhardt, S. R. Rheingold, T. M. Cooper, C. M. Zwaan, Phillip Barnette, C. Messina, G. Michel, Stéphanie Graff-Dubois, K. Q. HuM. Zhu, James A. Whitlock, John L. Gore

Research output: Contribution to journalArticle


Purpose Blinatumomab is a bispecific T-cell engager antibody construct targeting CD19 on B-cell lymphoblasts. We evaluated the safety, pharmacokinetics, recommended dosage, and potential for efficacy of blinatumomab in children with relapsed/refractory B-cell precursor acute lymphoblastic leukemia (BCP-ALL). Methods This open-label study enrolled children /= 3 adverse events were anemia (36%), thrombocytopenia (21%), and hypokalemia (17%). Three patients (4%) and one patient (1%) had cytokine-release syndrome of grade 3 and 4, respectively. Two patients (3%) interrupted treatment after grade 2 seizures. Conclusion This trial, which to the best of our knowledge was the first such trial in pediatrics, demonstrated antileukemic activity of single-agent blinatumomab with complete minimal residual disease response in children with relapsed/refractory BCP-ALL. Blinatumomab may represent an important new treatment option in this setting, requiring further investigation in curative indications.
Original languageUndefined/Unknown
Pages (from-to)4381-4389
Number of pages9
JournalJournal of Clinical Oncology
Issue number36
Publication statusPublished - Dec 20 2016

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