Short-course blinatumomab for refractory/relapse precursor B acute lymphoblastic leukemia in children

Front Pediatr. 2023 Aug 4:11:1187607. doi: 10.3389/fped.2023.1187607. eCollection 2023.

Abstract

Objective: To evaluate the clinical efficacy and safety of a short course of blinatumomab in children with refractory or relapsed precursor B-cell acute lymphoblastic leukemia (R/R-BCP-ALL).

Methods: The clinical data of 33 R/R BCP-ALL children aged 0-18 years who underwent a short course of blinatumomab (14 days) between August 2021 and November 2022 were retrospectively collected and analyzed.

Results: Among 33 patients with BCP-ALL, 26 achieved complete remission (CR), with a total remission rate of 78.8% (26/33). The duration of remission was approximately 14 days. Of the 7 children without CR, 5 were still in remission at 28 days. In 11 patients with refractory disease and 22 with recurrence, the remission rates were 90.9% (10/11) and 72.7% (16/22), respectively. The overall survival (OS) rates of the 26 patients with CR and seven patients without CR were 96.1% and 57.1% (p = 0.002), respectively, and the disease-free survival (DFS) rates were 96.1% and 42.9% (p < 0.001), respectively. Among the 26 patients with CR, 15 underwent bridging hematopoietic stem cell transplantation (HSCT) and 11 did not receive HSCT; with OS rates of 93.3% and 100% (p = 0.40) and DFS rates of 93.3% and 100% (p = 0.400), respectively. The OS for all patients was 87.9% (29/33) and the DFS was 84.8% (28/33). There were 18 cases (54.5%) of cytokine release syndrome (CRS), 2 cases (6.1%) of severe CRS (all grade 3), 1 case (3.0%) of immune effector cell-associated neurotoxicity syndrome (ICANS), 0 cases (0%) of ICANS ≥ grade 3, and no deaths caused by treatment.

Conclusions: Short-term follow-up revealed a high R/R BCP-ALL remission rate in children treated with a short course of blinatumomab. The toxicity was low and controllable. No significant short-term survival benefits were observed after bridging HSCT with blinatumomab. In developing countries, a short course of blinatumomab can achieve satisfactory outcomes, while reducing household costs and saving medical resources.

Keywords: acute lymphoblastic leukemia; blinatumomab; children; hematopoietic stem cell transplantation; refractory/relapse; short-course.

Grants and funding

This study was supported by the Medical Research Project of Jiangsu Provincial Health and Family Planning Commission (Key Project ZD2021006), National Natural Science Foundation of China (Nos. 81770193, 82100229, and 81970163), Jiangsu Project (No. BE2021654), Suzhou Enterprise Technology Innovation’s project, Suzhou Key Laboratory of Childhood Leukemia (SZS201615).