Blinatumomab as an Alternative to First-Line Intensive Chemotherapy in Pediatric B-Cell ALL

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In a retrospective study reported in the Journal of Clinical Oncology, Hodder et al found that blinatumomab was effective as a toxicity-sparing alternative to first-line intensive chemotherapy in children and young persons with B-cell acute lymphoblastic leukemia (ALL) who were chemotherapy-intolerant or chemotherapy-resistant.

Study Details

The study involved 105 consecutive pediatric patients aged 1to 24 years with Philadelphia chromosome–positive or –negative B-cell ALL enrolled from multiple sites between February 2018 and February 2023 who received blinatumomab as first-line therapy. Blinatumomab was given as a replacement for postremission intensive chemotherapy to patients with chemotherapy intolerance or resistance. Patients responding to blinatumomab received further chemotherapy (blinatumomab-CT group; n = 85) or first remission hematopoietic stem cell transplantation (blinatumomab-HSCT group; n = 20). Event-free and overall survival among 80 patients in the blinatumomab-CT group were compared to 192 matched controls treated with standard chemotherapy in the UKALL 2003 trial.

Key Findings

Among 85 patients in the blinatumomab-CT group, 70 (82%) received blinatumomab for chemotherapy intolerance. A total of 50 received blinatumomab postinduction and 35 received the agent mid- or postconsolidation. Blinatumomab was well tolerated, with only one patient having a treatment-related grade 3 or 4 adverse event—neurotoxicity—and no grade 3 or 4 cytokine-release syndrome observed. Among 60 patients who were measurable residual disease–positive, 58 (97%) responded to blinatumomab.

Among 80 patients who received blinatumomab-CT vs 192 matched controls, 2-year event-free survival was 95% (95% confidence interval [CI] = 85%–98%) vs 90% (95% CI = 65%–93%); 2-year overall survival was 97% (95% CI = 86%–99%) vs 94% (95% CI = 89%–96%).

Among the 20 patients in the blinatumomab-HSCT group, all responded to blinatumomab, with no grade 3 or 4 neurotoxicity or cytokine-release syndrome observed, and all proceeded to HSCT. Death occurred in three patients in ongoing remission due to HSCT complications, and two patients relapsed.

The investigators concluded: “Blinatumomab is safe and effective in first-line treatment of chemotherapy-intolerant children and young persons with ALL.”

Ajay Vora, MD, of Great Ormond Street Hospital, London, is the corresponding author for the Journal of Clinical Oncology article.

Disclosure: For full disclosures of the study authors, visit

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