Global Survey of Trilateral Retinoblastoma: Incidence and Outcomes
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 发起方
- 入组人数
- 4,351
- 试验地点
- 1
- 主要终点
- Number of participants (retinoblastoma patients) that will be diagnosed with trilateral retinoblastoma (incidence)
研究概览
简要总结
Patients with heritable retinoblastoma are at risk to develop an intracranial brain tumor, which is often fatal. The investigators intend to look at the incidence and survival of trilateral retinoblastoma (which is a brain tumor that can either be located in the pineal gland or elsewhere in the brain) in retinoblastoma patients globally. All retinoblastoma patients from participating centers will be included. The investigators hypothesize that the apparent incidence of trilateral retinoblastoma (especially the usually later diagnosed pineal trilateral retinoblastoma) in low-income countries will be lower because of low chances of surviving the ocular tumors at about 50% and also because of possible under-diagnosis. Therefore, as retinoblastoma care improves in low-income countries the incidence of (pineal) trilateral retinoblastoma might go up. Knowledge about incidence and survival can help improve health practices in parts of the world where this might be needed. Therefore this global study firstly aims 1) to evaluate survival after trilateral retinoblastoma and factors influencing survival and 2) to evaluate incidence of trilateral retinoblastoma by country income level. The study will run from 2024 through 2027.
详细描述
This study will adhere to the Strengthening the Reporting of Observational Studies in Epidemiology (STROBE) statement for cohort studies and to the Guidelines for Accurate and Transparent Health Estimates Reporting (GATHER) statement. The study is designed as a prospective cohort study of retinoblastoma patients diagnosed in one year from as many as possible worldwide treatment centers. The methods of patient recruitment are partly similar to previous global retinoblastoma studies. Data will be extracted from a the Global Retinoblastoma study follows up patients who will present with retinoblastoma in 2024-2027.
All unique trilateral retinoblastoma patients (cases) and all other retinoblastoma patients (controls) with will be included in the incidence analysis. For survival analysis follow-up data will be required.
After initial submission of case data by participating centers three years after the start of the study information on follow-up, metastasis, eye globe salvage, laterality, diagnosis of trilateral retinoblastoma and survival outcome will be available. At a later stage participating centers that reported trilateral retinoblastoma cases will be contacted again for additional information. All trilateral retinoblastoma patients will be included in the analysis, regardless if additional information is provided.
The investigators will request the following additional data:
- location (pineal, supra-/parasellar, other location (e.g., 3rd ventricle)),
- treatment for trilateral retinoblastoma,
- if not clear from initial report: cause of death (trilateral retinoblastoma, side effect of treatment, other),
- details on (germline) RB1 mutation.
- country of residence,
- family history of retinoblastoma,
- was patient symptomatic or asymptomatic when trilateral retinoblastoma was diagnosed (if symptomatic: what where the symptoms? If asymptomatic: how was the tumor diagnosed),
- information on trilateral retinoblastoma confirmation (e.g., biopsy, CSF, treatment response, disease progression),
- treatment for trilateral retinoblastoma,
- was the treatment with intent to cure (active treatment) or was the intent palliative,
- maximum diameter (mm) of tumor,
- wat trilateral retinoblastoma metastasized at the time of diagnosis,
- was this case published (if yes: where?),
- also missing data during the initial submission phase will be requested again.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Prospective
入排标准
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •patients diagnosed with retinoblastoma
排除标准
- 未提供
结局指标
主要结局
Number of participants (retinoblastoma patients) that will be diagnosed with trilateral retinoblastoma (incidence)
时间窗: 6 years
The proportion of patients that be diagnosed with trilateral retinoblastoma in the included cohort of retinoblastoma patients
Overall and event-free survival after being diagnosed with trilateral retinoblastoma
时间窗: 6 years
The overall and event free survival of patients diagnosed with trilateral retinoblastoma in the included cohort of retinoblastoma patients.
次要结局
未报告次要终点
研究者
Marcus de Jong
Principal Investigator
Amsterdam UMC, location VUmc