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Induction Regimens in High-Risk Neuroblastoma: Systematic Review of Response Rates and Toxicities
Samantha D Martin1, Eva C Robinson2, Edie Weller2
1Department of Pediatrics, Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Harvard Medical School, Boston, Massachusetts, USA.
Cancer Medicine
|November 23, 2025
Summary
High-risk neuroblastoma induction therapies using conventional chemotherapy show similar response rates over 30 years. Novel agents are needed to improve outcomes and reduce toxicities in neuroblastoma treatment.
Area of Science:
- Pediatric Oncology
- Clinical Trial Analysis
- Cancer Therapeutics
Background:
- High-risk neuroblastoma (HRNBL) treatment involves various induction therapies.
- Comparative data on response rates and toxicities of HRNBL induction regimens are lacking.
Purpose of the Study:
- To describe and compare key features of HRNBL induction regimens.
- To assess associations between regimen characteristics and end-induction response (EIR).
Main Methods:
- Systematic review of prospective frontline HRNBL therapy trials (PubMed, 1995-2024).
- Included studies reporting end-induction response (EIR) as partial response or better (PR+).
- Analyzed regimen details including agents, doses, duration, and patient numbers.
Main Results:
- 29 publications evaluated 36 HRNBL induction regimens.
- Median study-level EIR rate was 84.4% (weighted average 79.4%), with no improvement over time.
- Anthracycline-containing regimens showed higher EIRs; higher cisplatin dose intensity was linked to lower EIR.
- Only 16.7% of regimens included novel agents; toxic death rate was 0% median.
Conclusions:
- HRNBL induction therapies have predominantly used conventional chemotherapy for 30 years.
- Despite variations in treatment, study-level EIR rates have remained stagnant.
- Incorporating novel agents into future induction regimens is essential for improving EIR and reducing toxicity.
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