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Treatment response of CNS high-grade neuroepithelial tumors with MN1 alteration
Lorena V Baroni1, Carlos Rugilo2, Fabiana Lubieniecki3
1Service of Hematology/Oncology, Hospital JP Garrahan, Buenos Aires, Argentina.
Abstract:
Central nervous system high-grade neuroepithelial tumor with MN1 alteration (CNS HGNET-MN1) is a rare recently described entity. Fourteen CNS HGNET-MN1 patients were identified using genome-wide methylation arrays/RT-PCR across seven institutions. All patients had surgery (gross total resection: 10; subtotal resection: four) as initial management followed by observation alone in three patients, followed by radiotherapy in eight patients (focal: five; craniospinal: two; CyberKnife: one) and systemic chemotherapy in three patients. Seven patients relapsed; five local and two metastatic, despite adjuvant radiotherapy, of which three died. Treatment of CNS HGNET-MN1 remains a major treatment challenge despite aggressive surgical resections and upfront radiotherapy, warranting new approaches to this rare malignancy.
Insights
Central nervous system high-grade neuroepithelial tumor with MN1 alteration (CNS HGNET-MN1) is a rare malignancy. Current treatments involving surgery and radiotherapy present challenges, indicating a need for novel therapeutic strategies.
Area of Science:
- Neuro-oncology
- Molecular Pathology
- Pediatric Oncology
Background:
- Central nervous system high-grade neuroepithelial tumor with MN1 alteration (CNS HGNET-MN1) is a recently identified rare entity.
- Understanding the clinical characteristics and treatment outcomes of CNS HGNET-MN1 is crucial for developing effective management strategies.
Purpose of the Study:
- To characterize the clinical presentation, treatment modalities, and outcomes of patients diagnosed with CNS HGNET-MN1.
- To evaluate the efficacy of current treatment approaches, including surgery and radiotherapy, for CNS HGNET-MN1.
Main Methods:
- Retrospective analysis of 14 patients diagnosed with CNS HGNET-MN1 across seven institutions.
- Utilized genome-wide methylation arrays and RT-PCR for diagnosis.
- Documented surgical interventions, radiotherapy regimens, and chemotherapy use.
Main Results:
- All 14 patients underwent surgical resection (10 gross total, 4 subtotal).
- Adjuvant treatments included observation (3), radiotherapy (8), and chemotherapy (3).
- Seven patients relapsed (5 local, 2 metastatic) despite aggressive treatment, with three deaths observed.
Conclusions:
- CNS HGNET-MN1 presents a significant treatment challenge, with high rates of relapse even after aggressive multimodal therapy.
- Current treatment paradigms, including extensive surgery and upfront radiotherapy, are insufficient for durable control.
- Novel therapeutic approaches are urgently required to improve outcomes for patients with this rare CNS malignancy.
