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Published on: September 1, 2010
Group 4 Medulloblastomas of Early Childhood Treated With High-Dose Chemotherapy- and Craniospinal Irradiation-Sparing
Patricia Orduña1,2, Rose Daynielle Cansanay1, Craig Erker3
1Section of Pediatric Hematology and Bone Marrow Transplantation, Alberta Children's Hospital, Calgary, Alberta, Canada.
Insights
High-dose chemotherapy (HDC) without craniospinal irradiation (CSI) showed a 52.6% relapse rate in young children with Group 4 medulloblastoma (MB), comparable to conventional treatments. Salvage radiotherapy was effective, suggesting this approach may be an alternative to upfront CSI.
Area of Science:
- Pediatric Oncology
- Neuro-oncology
- Cancer Research
Background:
- Group 4 medulloblastoma (MB) is a rare brain tumor in young children.
- Limited data exist on craniospinal irradiation (CSI)-sparing treatment approaches for this population.
Purpose of the Study:
- To evaluate the outcomes of high-dose chemotherapy (HDC) without adjuvant CSI in young children (under 7 years) diagnosed with Group 4 MB.
- To assess the feasibility and efficacy of CSI-sparing strategies in this patient group.
Main Methods:
- A multicenter study included 38 patients younger than 7 years with Group 4 MB.
- Patients were treated with HDC regimens, primarily carboplatin/thiotepa or carboplatin/thiotepa/etoposide, without adjuvant CSI.
- Outcomes including relapse rates, progression-free survival (PFS), and overall survival (OS) were analyzed.
Main Results:
- The 5-year overall survival (OS) was 72.7%, with a 5-year CSI-free OS of 69.7%.
- A relapse rate of 52.6% was observed, with local relapse occurring in 45% of cases.
- Patients receiving upfront gross total resection (GTR) and/or three cycles of HDC showed improved PFS.
- Salvage radiotherapy was administered to 18 patients, with 16 receiving CSI and 2 focal radiotherapy.
Conclusions:
- HDC without upfront CSI in young children with Group 4 MB is associated with a comparable relapse rate to conventional chemotherapy.
- Salvage radiotherapy can effectively treat relapsed disease, with over two-thirds of patients receiving it achieving favorable outcomes.
- This CSI-sparing approach represents a potential alternative treatment strategy for very young children with Group 4 MB, warranting further investigation.
Background:
Group 4 medulloblastoma (MB) is rare in young children. Data on craniospinal irradiation (CSI)-sparing approaches are limited.
Methods:
This multicenter study reported outcomes for patients younger than 7 years old with Group 4 MB treated with high-dose chemotherapy (HDC) without adjuvant CSI.
Results:
Thirty-eight patients were included (26 M/12 F). Median age at diagnosis was 46.4 months (25.9-78), with 24% ≤36 months. Twenty-four patients (63.2%) had localized disease. Fourteen (36.8%) presented with metastatic disease, and 26 (68.4%) underwent gross total resection (GTR). The most used HDC regimens were carboplatin/thiotepa (76.3%) and carboplatin/thiotepa/etoposide (21.1%). Twenty (52.6%) relapses occurred at a median 21.9 months (5-99.8) from diagnosis. Patients with upfront GTR and/or who received three cycles of HDC had better PFS (p = 0.02 and p = 0.002, respectively). Local relapse accounted for 45%. Eighteen patients received salvage therapy with curative intent, all with radiotherapy (16 = CSI, 2 = focal). The CSI dose ranged from 18 to 36 Gy, and 43.7% received ≤23.4 Gy. Patients who underwent salvage surgery and/or chemotherapy were more likely to receive CSI ≤23.4 Gy (p = 0.008 and p = 0.03). The 5-year post-relapse survival and overall survival (OS) were, respectively, 60.3% (95% confidence interval [CI]: 26.9-82) and 72.7% (95% CI: 51.9-85.7). The 5-year CSI-free OS was 69.7% (95% CI: 39.1-87.1).
Conclusion:
High dose chemotherapy for Group 4 MB was associated with a relapse rate of 52.6%, which favorably compares to data reported with conventional chemotherapy. Salvage radiotherapy retrieved more than two-thirds of the patients. Half of the survivors never received radiotherapy. Such an approach may represent an alternative to upfront CSI for young children with Group 4 MB.
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