Incidence of Malignant Peripheral Nerve Sheath Tumors Development in Patients With Neurofibromatosis Type 1 Receiving and Not Receiving Medical Therapies Directed at Plexiform Neurofibromas
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 入组人数
- 296
- 试验地点
- 1
- 主要终点
- risk of MPNST formation
研究概览
简要总结
Background:
NF1 is a genetic syndrome. Tumors appear early in life. Many people with NF1 develop PN. These tumors can become an aggressive cancer called MPNST. People with MPNST may benefit from treatment with a MEK inhibitor (MEKi). Researchers want to learn if there is an increased risk of MPNST formation from MEKi treatment in people with NF1. To do this, they will review data that has been collected in NIH NF1 studies.
Objective:
To describe the characteristics of people who have taken part in NF1 studies at NIH and to compare the risk of MPNST formation in those treated with MEKi or other PN-directed treatment.
Eligibility:
People with NF1 who were seen at NIH from Jan. 1, 1998, to Jan. 1, 2020.
Design:
Participants medical records will be reviewed. Participants who opted out of future use of their data will not be included.
Demographic data, like sex, race, and date of birth, will be collected.
Data about MEKi and non-MEKi treatments will be collected.
Clinical data, such as surgery and treatment details, will be collected.
The differences between all participants who were seen at NIH for any NF1 related study will be compared. Participants will be put into 4 groups:
History of MEKi therapy
Treatment with tumor directed therapy other than MEKi
Treatment with both MEKi and non-MEKi tumor directed therapies
No tumor directed medical therapy
Participants with NF1 who were treated for PN with either a MEKi treatment or a non-MEKi treatment will also be compared.
The study will last for 3 to 6 months.
详细描述
Study Description: This is a retrospective review that includes all patients with NF1 enrolled on natural history and treatment trials at the National Cancer Institute (NCI) from 1/1/1998 to 1/1/2020, excluding patients referred for treatment of MPNST.
Objective
To describe the clinical and demographic characteristics of NF1 study participants who come for evaluation at the NCI and to compare the risk of MPNST formation in patients treated with MEK 1/2 inhibitor (MEKi) or other PN-directed treatment.
Study Population: All participants with NF1 seen at the NIH from 1/1/1998 to 1/1/2020
Description of Sites/Facilities conducting research: This is a single site study initiated by the Pediatric Oncology Branch at the National Cancer Institute
Study Duration: Data abstraction to completion of data analyses will take 3-6 months.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Retrospective
入排标准
- 年龄范围
- 1 Month 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •All participants seen at the NIH from 1/1/1998 to 1/1/2020 with a clinical or laboratory diagnosis of NF1
- •Participants beyond the neonatal period (4 weeks).
排除标准
- •All participants seen at the NIH from 1/1/1998 to 1/1/2020 with a clinical or laboratory diagnosis of NF1 who opted out of future use.
结局指标
主要结局
risk of MPNST formation
时间窗: ongoing
descriptive statistics and frequency distribution of selected variables
次要结局
未报告次要终点