Blinatumomab for treating pediatric B-lineage acute lymphoblastic leukemia: A retrospective real-world study

Wu, Ying; Liu, Yanming; Fan, Jia; Qi, Peijing; Lin, Wei; Yang, Jie; Liu, Huiqing; Wang, Xiaoling; Zheng, Huyong; Wang, Tianyou; Zhang, Ruidong

doi:10.3389/fped.2022.1034373

Cited by 4 publications

(1 citation statement)

References 26 publications

(37 reference statements)

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“…Trials are underway testing the benefit of Blina in first-line treatment of CYP with B-ALL (ClinicalTrials.gov identifiers: NCT03643276, NCT03914625), but as an addition rather than a replacement for intensive phases of chemotherapy backbones. Our results and those of other small case series [25][26][27][28] in patients with chemotherapy intolerance provide a strong argument for testing the latter strategy for all CYP with B-ALL.…”

Section: Discussionsupporting

confidence: 66%

Blinatumomab for First-Line Treatment of Children and Young Persons With B-ALL

Hodder,

Mishra,

Enshaei

et al. 2024

JCO

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PURPOSE We tested whether blinatumomab (Blina) is effective as a toxicity-sparing alternative to first-line intensive chemotherapy in children and young persons (CYP) with B-ALL who were chemotherapy-intolerant or chemotherapy-resistant. METHODS Data were collected for consecutive CYP (age 1-24 years) with Philadelphia chromosome–positive or Philadelphia chromosome–negative B-ALL who received Blina as first-line therapy. Blina was given as replacement for postremission intensive chemotherapy to patients with chemotherapy intolerance or resistance. Blina responders received further chemotherapy (Blin-CT) or first remission hematopoietic stem-cell transplant (Blin-HSCT) if indicated. Event-free survival (EFS) and overall survival (OS) of the Blin-CT group were compared with those of matched controls treated with standard chemotherapy in the UKALL 2003 trial. Events were defined as death, relapse, or secondary cancer. RESULTS From February 2018 to February 2023, 105 patients were treated, of whom 85 were in the Blin-CT group and 20 were in the Blin-HSCT group. A majority of Blin-CT patients received Blina for chemotherapy intolerance (70 of 85, 82%), and the group had a higher-risk profile than unselected patients with B-ALL. Blina was well tolerated with only one patient having a grade 3/4–related toxicity event, and of the 60 patients who were minimal residual disease–positive pre-Blina, 58 of 60 (97%) responded. At a median follow-up of 22 months, the 2-year outcomes of the 80 matched Blin-CT group patients were similar to those of 192 controls (EFS, 95% [95% CI, 85 to 98] v 90% [95% CI, 65 to 93] and OS, 97% [95% CI, 86 to 99] v 94% [95% CI, 89 to 96]). Of the 20 in the HSCT group, three died because of transplant complications and two relapsed. CONCLUSION Blina is safe and effective in first-line treatment of chemotherapy-intolerant CYP with ALL.

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Section: Discussionsupporting

confidence: 66%

Blinatumomab for First-Line Treatment of Children and Young Persons With B-ALL

Hodder,

Mishra,

Enshaei

et al. 2024

JCO

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Blinatumomab

2023

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Leukemia burden impacts the efficacy and toxicity of blinatumomab in pediatric B-cell acute lymphoblastic leukemia

Yan,

Hu,

Gao

et al. 2024

Preprint

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Background Blinatumomab has demonstrated its efficacy and safety in pediatric patients with B-cell acute lymphoblastic leukemia (B-ALL). The objective of this analysis was to describe the responses and toxicities of blinatumomab in pediatric patients with different leukemic burdens in the bone marrow. Methods We enrolled patients aged 0-18 years who were diagnosed with CD19-positive B-ALL and treated with blinatumomab between January 2021 and May 2023 from 14 centers in China. Results A total of 307 patients were enrolled in this analysis. The complete remission (CR) rate was 72.1% among 61 patients with ≥5% blasts(non-complete remission, NCR group), of whom 90.9% achieved minimal residual disease (MRD) negativity. Among 93 patients with <5% blasts but multiparametric flow cytometry MRD (MFC-MRD) positive(MRD+ group), 96.8% achieved MRD negativity. Of the 153 MFC-MRD negative patients(MRD- group), 60.0% and 65.5% turned quantitative polymerase chain reaction MRD (qPCR-MRD) or next-generation sequencing MRD (NGS-MRD) negative, respectively. Additionally, Patients in the MRD+ and MRD- groups had significantly better outcomes than those in the NCR group, with 30-month OS rates of 91.6% (95% CI: 0.857-0.979), 95.3% (95% CI: 0.915-0.993), and 77.6% (95% CI: 0.674-0.894), respectively (P<0.001), and 30-month RFS rates of 90.7% (95% CI: 0.847-0.972), 93.3% (95% CI: 0.890-0.979), and 64.4% (95% CI: 0.495-0.837), respectively (P<0.001). There was no statistically significant difference in OS between the patients who achieved MFC-MRD negativity in the NCR group and those in the MRD+ group, with 30-month OS rates of 85.7% (95% CI: 0.745-0.987) and 93.2% (95% CI: 0.881-0.986), respectively (P=0.270). In this study, 41% of patients experienced grade ≥3 adverse events (AEs), with hematological toxicity being the most common (32.9%). The severe adverse events, such as cytokine release syndrome (CRS) and neurotoxicity, occurred at a low rate, particularly grade ≥3, at 3.6% and 2.6%, respectively. Conclusion Overall, these results indicate that blinatumomab is effective and well-tolerated. Patients with a lower leukemia burden before blinatumomab administration tend to have better overall survival and relapse-free survival with fewer AEs.

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Blinatumomab for treating pediatric B-lineage acute lymphoblastic leukemia: A retrospective real-world study

Cited by 4 publications

References 26 publications

Blinatumomab for First-Line Treatment of Children and Young Persons With B-ALL

Blinatumomab for First-Line Treatment of Children and Young Persons With B-ALL

Blinatumomab

Leukemia burden impacts the efficacy and toxicity of blinatumomab in pediatric B-cell acute lymphoblastic leukemia

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