Real-world efficacy and safety of blinatumomab across three clinical settings in pediatric B-cell acute lymphoblastic leukemia: a single-center retrospective cohort study.
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BACKGROUND: Blinatumomab, a CD19/CD3 bispecific T-cell engager, is used in pediatric B-cell acute lymphoblastic leukemia (B-ALL) across several clinical settings. Real-world data remain limited for patients receiving blinatumomab because of chemotherapy intolerance or treatment delay. We described treatment responses and adverse events across three clinical settings in a single-center cohort. METHODS: This single-center retrospective cohort included patients aged ≤18 years with B-ALL who received blinatumomab at Anhui Provincial Children's Hospital between September 2021 and September 2025. The cohort comprised patients with relapsed/refractory (R/R) disease, complete remission (CR) with measurable residual disease (MRD) positivity (CR-MRD+), or CR with MRD negativity (CR-MRD-) treated because of chemotherapy intolerance or treatment delay. Patients with isolated central nervous system relapse without bone marrow involvement or who discontinued treatment before completing one planned cycle were excluded. Clinical data were retrospectively abstracted from medical records. Blinatumomab was administered by continuous intravenous infusion with dose escalation from 5 μg/m2/day (<45 kg) or 9 μg/m2/day (≥45 kg) to 15 μg/m2/day. Cycles lasted 14 or 28 days according to clinical condition and tolerance. The follow-up cutoff was November 30, 2025. Efficacy outcomes were described by subgroup, and adverse events were summarized across 54 treatment cycles. RESULTS: Forty-nine patients (26 males and 23 females; median age, 5 years; range, 0.5-17.0 years) received 54 blinatumomab treatment cycles. Fourteen patients had R/R disease, of whom 12 achieved CR (85.7%). All 16 patients in the CR-MRD+ group achieved MRD negativity after treatment. In the CR-MRD- group, post-treatment MRD was available for 18 patients, all of whom remained MRD negative; one patient had no post-treatment MRD assessment and was recorded as lost to follow-up. Across 54 treatment cycles, cytokine release syndrome (CRS) was recorded in 35 cycles (64.8%; all grade 1-2), infections in 20 cycles (37.0%), and neutropenia in 20 cycles (37.0%). Two deaths were recorded by the follow-up cutoff. CONCLUSIONS: In this single-center retrospective cohort, treatment responses and adverse events varied across three clinically distinct pediatric B-ALL settings. These descriptive findings do not establish comparative treatment efficacy and should be interpreted in light of the small sample size, clinical heterogeneity, cycle-level safety summaries, and incomplete follow-up in one patient.