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Updated: Mar 10, 2026
In Ovo Xenografting of Patient-Derived Acute Lymphoblastic Leukemia (ALL) Cells (PDX-ALL)
Published on: August 1, 2025
E2A-PBX1 exhibited a promising prognosis in pediatric acute lymphoblastic leukemia treated with the CCLG-ALL2008
Yixin Hu1, Hailong He1, Jun Lu1
1Department of Hematology and Oncology, The Children's Hospital of Soochow University, Suzhou, People's Republic of China.
Insights
Pediatric acute lymphoblastic leukemia (ALL) patients with E2A-PBX1 gene expression showed improved outcomes with the CCLG-ALL2008 protocol. This intensified treatment led to lower minimal residual disease (MRD) and better event-free survival (EFS).
Area of Science:
- Pediatric Oncology
- Hematologic Malignancies
- Molecular Diagnostics in Leukemia
Background:
- E2A-PBX1 gene fusion is a specific subtype of pre-B-cell acute lymphoblastic leukemia (ALL).
- Understanding the prognosis and treatment response in pediatric ALL with specific genetic markers is crucial for optimizing therapy.
Purpose of the Study:
- To evaluate the clinical prognosis of pediatric patients diagnosed with E2A-PBX1-positive acute lymphoblastic leukemia (ALL).
- To assess the effectiveness of the CCLG-ALL2008 treatment protocol in this specific patient cohort.
Main Methods:
- A cohort of 349 Chinese pediatric patients with pre-B-cell ALL were analyzed.
- Patients were stratified into E2A-PBX1 positive (n=20) and negative (n=223) groups.
- Clinical characteristics, minimal residual disease (MRD), and 5-year survival outcomes (EFS, RFS, OS) were compared.
Main Results:
- E2A-PBX1 fusion transcript was found in 5.7% of patients.
- E2A-PBX1 positive patients were younger and had more inferior karyotypes.
- These patients showed favorable treatment response with lower MRD levels (TP1) and significantly improved 5-year event-free survival (95.0% vs 66.3%).
Conclusions:
- Pediatric patients with E2A-PBX1-positive ALL demonstrated a beneficial response to the risk-based CCLG-ALL2008 intensified treatment protocol.
- The protocol resulted in lower MRD levels and improved event-free survival, despite the absence of favorable initial diagnostic characteristics.
Objective:
The objective of this study was to observe the prognosis of pediatric patients with E2A-PBX1-positive acute lymphoblastic leukemia (ALL) from the treatment with the CCLG-ALL2008 protocol.
Design And Methods:
Three hundred and forty-nine Chinese pediatric patients with pre-B-cell ALL were enrolled in this study from December 2008 to September 2013. Of these, 20 patients with E2A-PBX1 expression and 223 without the gene expression were stratified into two cohorts. Clinical and biological characteristics and 5-year event-free survival (EFS), relapse-free survival (RFS), and overall survival (OS) were analyzed and compared between these two groups.
Results:
The E2A-PBX1 fusion transcript was detected in 20 of 349 (5.7%) patients. Compared with the gene-negative subgroup, patients with E2A-PBX1 were younger in age but did not show significant differences in white blood cell (WBC) count or gender distribution at primary diagnosis. Moreover, there were more inferior karyotypes detected in the E2A-PBX1 subgroup (P=0.035). With the CCLG-ALL2008 treatment protocol, patients with E2A-PBX1 showed a favorable treatment response with lower minimal residual disease (MRD) levels (<10-4) at time point 1 (TP1, P=0.039) but no superior steroid response or histological remission. We also observed a promising survival outcome, with a 5-year EFS reaching 95.0%±4.9% versus 66.3%±3.9% in the gene-negative group (P=0.039). However, we did not find significant differences in RFS (P=0.061) and OS (P=0.113).
Conclusion:
Our data provided clinical observation of Chinese pediatric patients. Patients with E2A-PBX1-positive ALL benefited well from the CCLG-ALL2008 protocol, a risk-based intensified treatment trial, with lower levels of MRD and longer RFS duration though they had no favorable characteristics at primary diagnosis.
