Comparison of an Initial Active Follow-up and Therapeutic Care on Functional Outcome and Quality of Life in Patients With Head and Neck Paragangliomas
试验速览
- 阶段
- 不适用
- 状态
- 尚未招募
- 入组人数
- 122
- 试验地点
- 8
- 主要终点
- Time to functional outcome deterioration
研究概览
简要总结
Head and neck paragangliomas (HNPGs) are predominantly non-secreting, benign and slow-growing tumors. Although most patients remain asymptomatic, up to 30% develop symptoms related to local tumor growth and fewer than 5-10% develop metastatic disease. Surgery has long been considered the standard treatment, while radiotherapy and active surveillance represent alternative management strategies. Treatment-related morbidity and impaired quality of life have been reported in patients with HNPGs; however, the impact of immediate intervention compared with an initial active surveillance strategy has not been prospectively evaluated. This multicenter randomized controlled trial aims to compare active surveillance with immediate intervention in patients with newly diagnosed carotid or vagal paragangliomas. The study hypothesis is that an initial active surveillance strategy increases the time before functional outcome deterioration, particularly ENT-related symptoms, while not resulting in a higher complication rate when treatment is subsequently performed. Eligible patients will undergo baseline assessment including a specialized ENT examination to evaluate cranial nerve function and cervical MRI (or contrast-enhanced CT when MRI is contraindicated) to confirm tumor location, size, and the absence of lymphadenopathy or atypical imaging features. After providing written informed consent, participants will be centrally randomized in a 1:1 ratio, stratified by study center and tumor location, to either active surveillance or immediate intervention. Patients assigned to active surveillance will undergo regular clinical and radiological follow-up, with treatment initiated only if disease progression or symptom development warrants intervention. Patients assigned to immediate intervention will receive surgery or radiotherapy according to multidisciplinary team recommendations and local practice, within six months after randomization. Patients who decline participation in the randomized trial, as well as those who are not eligible for randomization, may be offered participation in a parallel observational study collecting clinical, imaging, treatment and outcome data according to routine practice. Patient quality of life will also be assessed throughout the study (experimental and observational) using validated questionnaires.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Patients ≥ 18 years-old,
- •Diagnosis of carotid or vagal HNPG with a largest diameter more than 10 mm
- •Diagnosis confirmed by imaging reading (MRI of CT scan) from less than 6 months,
- •Study eligibility validated in multidisciplinary discussion,
- •Patient followed in the reference center,
- •Preliminary written informed consent before any study-specific intervention
排除标准
- •Patient with initial nerve palsy due to the evaluated HNPG,
- •Patient with non-typical presentation suggestive of aggressiveness (tumor pain, atypical imaging, suspicious lymphadenopathy),
- •Malignant HNPG identified by extra cervical lesion on metabolic imaging,
- •More than one HNPG at inclusion
- •Secreting HNPG defined as plasma or urine metanephrine or normetanephrine more than 2 times ULN,
- •Patient already treated with cervical radiotherapy,
- •Patient already treated with systemic therapy for another pheochromocytoma or paraganglioma,
- •Patient with another evolutive disease or other condition resulting on a life expectancy of less than 5 years at the investigator's discretion,
- •Patient unable or unwilling to be treated at the study center
- •Patients currently enrolled in another interventional study including investigational medicinal products or device,
- •Female patients who are pregnant, lactating or women of child-bearing potential without highly effective methods of contraception
- •Persons deprived of their liberty by a judicial or administrative decision
- •Persons under psychiatric care
- •Persons admitted to a health or social institution for purposes other than research
- •Adults subject to a legal protection measure (guardianship, curatorship)
- •Persons not affiliated to a social security scheme or beneficiaries of a similar scheme
研究组 & 干预措施
Active follow-up
干预措施: Active follow-up (Other)
Treatment
Patients will be treated by surgery or radiation. The choice between these 2 options is left to the discretion of the investigator and the patient.
干预措施: Surgery (Procedure)
Treatment
Patients will be treated by surgery or radiation. The choice between these 2 options is left to the discretion of the investigator and the patient.
干预措施: Radiation (Radiation)
结局指标
主要结局
Time to functional outcome deterioration
时间窗: From baseline to 5 years maximum
Time to functional outcome deterioration is defined as the delay between inclusion and functional outcome deterioration. Patients without functional deterioration are censored at their last functional assessment. Functional outcome deterioration is defined as a decrease in at least 12 points (or 8.6%) of the FACT H\&N score since inclusion. The FACT-H\&N score ranges from 0 to 148. The higher the score, the better the quality of life.
次要结局
- Evolution of the functional outcomes between the groups(Baseline, 1 year and 5 years)
- Quality of life (QoL)(Baseline, 1 year and 5 years)
- Anxiety(Baseline, 1 year and 5 years)
- Factors associated with a significant quality of life(Baseline, 1 year, 5 years)
- Factors associated with functional outcomes(Baseline, 1 year, 5 years)
- Factors associated with anxiety deterioration(Baseline, 1 year, 5 years)
- Switch proportion, reason and time to therapeutic intervention(From baseline to study end)
- Progression/recurrence rate(From baseline to study end)
- Surgery complication rate(At 1 year and 5 years)
- Radiation therapy complication rate(At 1 year and 5 years)
- Disease progression(At 1 year and 5 years)
- Proportion of distant metastasis(At 5 years)