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Updated: Jun 24, 2025

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Published on: February 5, 2020
Efficacy of anti-PD-1 monotherapy for recurrent or metastatic olfactory neuroblastoma
Yuta Hoshi1,2, Tomohiro Enokida1, Shingo Tamura3
1Department of Head and Neck Medical Oncology, National Cancer Center East Hospital, Kashiwa, Japan.
Background:
Olfactory neuroblastoma (ONB) is a rare malignant tumor of the head and neck. Due to its rarity, standard systemic therapy for this condition has yet to be established. In particular, the use of immune checkpoint inhibitors (ICIs) for the recurrent or metastatic (R/M) ONB population remains unclear.
Methods:
We retrospectively evaluated 11 patients with R/M ONB who received any systemic chemotherapy at two Japanese institutions (National Cancer Center Hospital East and Kyushu Medical Center) between January 2002 and March 2022 and analyzed outcomes by use of anti-PD-1 antibody (nivolumab or pembrolizumab) monotherapy.
Results:
Of the 11 patients, 6 received ICI (ICI-containing treatment group) and the remaining 5 were treated with systemic therapy but not including ICI (ICI-non-containing treatment group). Overall survival (OS) was significantly longer in the ICI-containing group (median OS: not reached vs. 6.4 months, log-rank p-value: 0.035). The fraction of ICI systemic therapy in the entire treatment period of this group reached 85.9%. Four patients (66.7%) in the ICI-containing treatment group experienced immune-related adverse events (irAE), with grades of 1/2. No irAE of grade 3 or more was seen, and no patient required interruption or discontinuation of treatment due to toxicity.
Conclusion:
ICI monotherapy appears to be effective and to contribute to prolonged survival in R/M ONB.
Insights
Immune checkpoint inhibitors (ICIs) show promise for recurrent or metastatic olfactory neuroblastoma (ONB). Patients receiving ICI therapy experienced significantly longer overall survival compared to those without ICIs.
Area of Science:
- Oncology
- Head and Neck Cancer Research
- Immunotherapy
Background:
- Olfactory neuroblastoma (ONB) is a rare head and neck malignancy.
- Standard systemic therapies for ONB are not well-established.
- The efficacy of immune checkpoint inhibitors (ICIs) in recurrent or metastatic (R/M) ONB is unclear.
Purpose of the Study:
- To evaluate the effectiveness of ICIs in patients with R/M ONB.
- To compare outcomes between ICI-containing and ICI-non-containing treatment groups.
Main Methods:
- Retrospective analysis of 11 R/M ONB patients from two Japanese institutions.
- Patients received systemic chemotherapy between 2002 and 2022.
- Outcomes were analyzed based on the use of anti-PD-1 antibody monotherapy (nivolumab or pembrolizumab).
Main Results:
- Six patients received ICI-containing treatment; five did not.
- The ICI-containing group showed significantly longer overall survival (median OS: not reached vs. 6.4 months, p=0.035).
- ICI therapy was well-tolerated, with manageable immune-related adverse events (irAEs) of grade 1/2 in 66.7% of patients.
Conclusions:
- ICI monotherapy appears effective for R/M ONB.
- ICIs may contribute to prolonged survival in this patient population.
- Further research is warranted to establish ICIs as a standard treatment.
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