Dramatic response to targeted therapy in an aggressive olfactory neuroblastoma: illustrative case
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Saksham Gupta1, Wenya Linda Bi1, Donald J Annino2
1Center for Skull Base and Pituitary Surgery, Department of Neurosurgery, and.
Background:
Olfactory neuroblastomas are rare sinonasal tumors that arise from the olfactory epithelium. The authors presented a case of an olfactory neuroblastoma with extensive cranial invasion that demonstrated dramatic response to sorafenib, a tyrosine kinase inhibitor.
Observations:
A 54-year-old man with history of prostate cancer and melanoma presented with left-sided proptosis and was found to have a 6.5-cm Kadish stage D olfactory neuroblastoma with cranial invasion that was refractory to chemotherapy and everolimus. However, it demonstrated dramatic response to sorafenib, causing extensive skull base defects that prompted operative repair. Genomic analysis of the tumor revealed mutations in TSC1 and SUFU. The patient developed disease progression with liver metastases 35 months after starting sorafenib, prompting a change to lenvatinib. He experienced progression of his olfactory neuroblastoma 10 months following this change and died in hospice 1 month later.
Lessons:
The authors reviewed the clinical presentation and management of a large olfactory neuroblastoma with dramatic response to sorafenib. They highlighted prior uses of targeted therapy in the management of refractory olfactory neuroblastoma within the context of current standard treatment regimens. Targeted therapies may play a vital role in the management of refractory olfactory neuroblastoma.
Insights
Olfactory neuroblastoma, a rare sinonasal tumor, showed a significant response to sorafenib, a targeted therapy, even when resistant to other treatments. This case highlights the potential of targeted therapies for refractory olfactory neuroblastoma.
Area of Science:
- Oncology
- Genetics
- Pharmacology
Background:
- Olfactory neuroblastoma is a rare sinonasal tumor originating from the olfactory epithelium.
- This study focuses on a specific case of advanced olfactory neuroblastoma with extensive cranial invasion.
Observation:
- A 54-year-old male patient with refractory Kadish stage D olfactory neuroblastoma demonstrated a dramatic response to sorafenib.
- Genomic analysis revealed mutations in TSC1 and SUFU genes within the tumor.
- The patient experienced disease progression after 35 months on sorafenib, leading to treatment with lenvatinib.
Findings:
- Sorafenib, a tyrosine kinase inhibitor, induced a significant tumor response in a patient with refractory olfactory neuroblastoma.
- The patient's tumor exhibited mutations in TSC1 and SUFU, potentially influencing treatment response.
- Despite initial success, the tumor eventually progressed, necessitating a change in targeted therapy.
Implications:
- Targeted therapies, such as sorafenib, show promise in managing refractory olfactory neuroblastoma.
- Genomic profiling may guide treatment decisions for patients with rare sinonasal tumors.
- Further research is warranted to explore the role of targeted agents in olfactory neuroblastoma treatment regimens.
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