Neoplasms

All Conditions

Nervous System Diseases

Rare Diseases

Neuroblastoma

Esthesioneuroblastoma, Olfactory

Neuroectodermal Tumors, Primitive, Peripheral

Neuroectodermal Tumors

Neuroectodermal Tumors, Primitive

Neoplasms, Neuroepithelial

Neoplasms, Germ Cell and Embryonal

Neoplasms by Histologic Type

Neoplasms, Glandular and Epithelial

Neoplasms, Nerve Tissue

Olfactory Nerve Diseases

Cranial Nerve Diseases

Olfactory Neuroblastoma

Neuroepithelioma

Subjects with confirmed olfactory neuroblastoma.

Marissa B Mallek, R.N.

Charalampos Floudas, M.D.

For more information at the NIH Clinical Center contact National Cancer Institute Referral Office

Background:

Olfactory neuroblastoma (ONB) is a rare cancer. It grows from tissue in the upper part of the nose cavity, related to the sense of smell and can affect a person s sense of smell. Researchers want to better understand the health problems of people with ONB. This may help them design better treatment and supportive care studies.

Objective:

To better understand ONB-the course of the disease, tumor characteristics, response to treatments, and management of the treatment.

Eligibility:

People ages 3 years and older who have ONB. They must enroll in NIH studies #19-C-0016 and #18-DC-0051.

Design:

Participants will be screened with a medical history and medical record review.

Participants do not have to visit NIH.

Participants will give a blood sample. They will complete surveys to assess their emotional and physical wellbeing and needs. Leftover tissue from biopsies and surgeries will be collected.

Participants will take smell tests. They will smell items and answer questions about them.

Participants may take taste tests. They will get plastic taste strips that they will move around their mouth to determine the taste.

Participants may have a physical exam. Their performance status may be assessed.

Participants may give blood, saliva, urine, and nasal secretion samples.

Participants may have computed tomography and/or magnetic resonance imaging scans.

Participants may have one or more tumor biopsies.

Participants will talk to the research team about the results of their medical record/tests evaluation. The team will recommend how to best manage and treat their disease.

Participants may give samples and complete surveys every 12 months. Their medical records will be reviewed every year. They will be monitored for the rest of their life.

...

Background:

Olfactory neuroblastoma (ONB), also known as esthesioneuroblastoma, is a malignant tumor of the nasal cavity believed to arise from the olfactory epithelium. ONB is rare, with a reported incidence of 0.4 per million (approximately 300 cases per year in the United States).

ONB most frequently arises in adults aged 20-60 years. Pediatric ONB is particularly rare, with only small case series or multi-institutional studies reported. However, a Surveillance Epidemiology and End Results (SEER) study of 47 pediatric patients with intranasal malignancy reported ONB as the most frequent pediatric intranasal malignancy (28%).

Due to the location of ONB in the sinonasal tract and anterior skull base, the presenting symptoms are often non-specific, including nasal obstruction and epistaxis, thus patients often present at advanced stages. Furthermore, ONB has a propensity for local invasion and relatively high rates of distant spread most commonly to the neck, lungs, and bones.

Clinical management in adults is generally surgical with adjuvant radiation.

The natural history of pediatric and adult ONB is incompletely understood, and treatment options for recurrent or metastatic ONB are limited. The planned natural history study as part of the NCI POB Rare Tumor Patient Engagement Network (RTPEN) will allow for comprehensive evaluation and recommendations to these patients while longitudinally collecting clinical, epidemiologic, and biological data.

Objective:

To characterize the natural history of ONB including clinical presentation, family history, patterns of disease progression, response to current treatment methods, disease recurrence, and overall survival

Eligibility:

Participants of age \>= 3 years old with histologically documented ONB.

Subjects must also enroll on NIDCD Protocol 18-DC-0051.

Design:

This protocol is intended to characterize the natural history of olfactory neuroblastoma.

Participants will undergo a comprehensive study entry evaluation including acquisition of imaging of tumor sites.

Medical histories will be documented, and participants followed throughout the course of their disease, with particular attention to patterns of disease recurrence and progression, response to therapies, duration of responses, and patient reported outcomes. Specimens will be obtained longitudinally, when feasible, and tumor growth rates will also be assessed throughout the course of the disease, when feasible.

Blood and tumor samples may be obtained at study entry and while on study.

A Natural History Study of Children and Adults With Olfactory Neuroblastoma

.All IPD recorded in the medical record will be shared with intramural investigators upon request. @@@@@@@@@@@@In addition, all large scale genomic sequencing data will be shared with subscribers to dbGaP.

To characterize the natural history of ONB including clinical presentation, family history, patterns of disease progression, response to current treatment methods, disease recurrence, and overall survival.

National Cancer Institute (NCI)

Antineoplastic Agents

All Drugs and Chemicals

Antirheumatic Agents

Etoposide

Gemcitabine

Cyclophosphamide

Cisplatin

Etoposide phosphate

Immunosuppressive Agents

Immunologic Factors

Antineoplastic Agents, Alkylating

Alkylating Agents

Antimetabolites

Enzyme Inhibitors

Induction chemotherapy based on pathological molecular subtypes of olfactory neuroblastoma.

① According to the immunohistochemical results of the pathological report, patients with Ki-67 index ≥ 25% were treated with two weeks of gemcitabine+platinum based chemotherapy regimen;

② According to the immunohistochemical results of the pathological report, patients with Ki-67 index \<25% were treated with a basic chemotherapy regimen of cyclophosphamide+etoposide+cisplatin (CEP regimen);

According to the progression of the patient's disease, the combination of other chemotherapy/small molecule drugs/targeted drugs can be considered;

When the Ki67% index of the tumor is ≥ 25%, patients were treated with the GP regimen for induction chemotherapy; When the Ki67% index of the tumor is \< 25%, patients were treated with the CEP regimen for induction chemotherapy.

Gemcitabine+Cisplatin(GP); Cyclophosphamide+Etoposide+Cisplatin(CEP)

Xiaole Song, MD

Jingyi Yang, MD

Hongmeng Yu, Dr

Xiaole Song, Dr

Xicai Sun, Dr

Jingyi Yang, Dr

Fu Chen, Dr

Hongmeng Yu, Prof.

Xicai Sun, MD

The goal of this clinical trial is to learn about the induction chemotherapy efficacy in olfactory neuroblastoma. The main question it aims to answer is: wether olfactory neuroblastoma patients with different pathology subtypes apply to different induction chemotherapy schemes. Participants will be treated with different chemotherapy schemes, to evaluate the tumor remission rate and long term survival.

This study included patients with olfactory neuroblastoma who were pathologically diagnosed and met the criteria. According to molecular profiles, two different induction chemotherapy schemes were used to evaluate the tumor remission rate.

Efficacy and Safety of Induction Chemotherapy for Olfactory Neuroblastoma (ESICON)

Efficacy and Safety of Induction Chemotherapy for Olfactory Neuroblastoma: a Prospective Umbrella Clinical Study

Study Protocol, Informed Consent Form (ICF), Clinical Study Report (CSR) and Analytic Code were to be shared on request.

According to RECIST1.1 criteria, complete remission (CR) and partial remission (PR)

pCR rate refers to the probability that no tumor cells was found during pathological diagnosis of tumor sample of surgical treatment after induction chemotherapy, in all patients who have been enrolled in the group.

During the surgery after induction chemotherapy, 4-6 surgical margins were retrieved for each patient. The rate of surgical pathology negative margin refers to the probability that pathological examination showed no tumor cells in all surgical margins, among all patients who have been enrolled in the group.

The 2-year OS rate refers to the proportion of alive patients 2 years after enrollment, analyzed using the Kaplan Meier method.

Refers to the incident rate of side effects related to the prescribed treatment during the induction chemotherapy and the whole follow-up duration. Treatment-related side effects were evaluated using the Common Terminology Criteria for Adverse Events (CTCAE) version 5.

Study title	NCT ID	Conditions	Interventions	Eligibility	Organization
A Natural History Study of Children and Adults With Olfactory Neuroblastoma	NCT04755205	Olfactory Neuro... Esthesioneurobl...		3 Years - 120 Years	National Institutes of Health Clinical Center (CC)
Efficacy and Safety of Induction Chemotherapy for Olfactory Neuroblastoma (ESICON)	NCT06255210	Olfactory Neuro...	Gemcitabine+Cis...	18 Years -	Eye & ENT Hospital of Fudan University
Efficacy and Safety of Induction Chemotherapy for Olfactory Neuroblastoma (ESICON)	NCT06255210	Olfactory Neuro...	Gemcitabine+Cis...	18 Years -	Eye & ENT Hospital of Fudan University

Spots Global Cancer Trial Database for olfactory neuroblastoma