Cancer Res Treat.  2024 Apr;56(2):652-664. 10.4143/crt.2023.865.

Long-term Outcomes of Protocol-Based Treatment for Newly Diagnosed Medulloblastoma

• Ahn WK
• Hahn SM
• Yoon HI
• Lee J
• Park EK
• Shim KW
• Kim DS
• Suh CO
• Kim SH
• Lyu CJ
• Han JW

• KMID: 2554354
• DOI: http://doi.org/10.4143/crt.2023.865

Abstract

Purpose
The Korean Society of Pediatric Neuro-Oncology (KSPNO) conducted treatment strategies for children with medulloblastoma (MB) by using alkylating agents for maintenance chemotherapy or tandem high-dose chemotherapy (HDC) with autologous stem cell rescue (ASCR) according to the risk stratification. The purpose of the study was to assess treatment outcomes and complications based on risk-adapted treatment and HDC.
Materials and Methods
Fifty-nine patients diagnosed with MB were enrolled in this study. Patients in the standard-risk (SR) group received radiotherapy (RT) after surgery and chemotherapy using the KSPNO M051 regimen. Patients in the high-risk (HR) group received two and four chemotherapy cycles according to the KSPNO S081 protocol before and after reduced RT for age following surgery and two cycles of tandem HDC with ASCR consolidation treatment.
Results
In the SR group, 24 patients showed 5-year event-free survival (EFS) and overall survival (OS) estimates of 86.7% (95% confidence interval [CI], 73.6 to 100) and 95.8% (95% CI, 88.2 to 100), respectively. In the HR group, more infectious complications and mortality occurred during the second HDC than during the first. In the HR group, the 5-year EFS and OS estimates were 65.5% (95% CI, 51.4 to 83.4) and 72.3% (95% CI, 58.4 to 89.6), respectively.
Conclusion
High intensity of alkylating agents for SR resulted in similar outcomes but with a high incidence of hematologic toxicity. Tandem HDC with ASCR for HR induced favorable EFS and OS estimates compared to those reported previously. However, infectious complications and treatment-related mortalities suggest that a reduced chemotherapy dose is necessary, especially for the second HDC.

Keyword

Medulloblastoma; High-dose chemotherapy; Autologous stem cell rescue; Children

Figure

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  Fig. 1. The Korean Society of Pediatric Neuro-Oncology (KSPNO) protocol scheme for medulloblastoma. The KSPNO M051 protocol for the standard-risk group (A) and the KSPNO S081 protocol for the high-risk group (B). CSRT, craniospinal radiotherapy; CTx, chemotherapy; HD, high-dose; RTx, radiotherapy.

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  Fig. 2. Event-free survival and overall survival for patients according to the risk group (A, B) and molecular subgroup (C, D).

Reference

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