Clinico-biological Study/Characterization of Rhabdomyosarcoma in AYA (Adolescents and Young Adults, 15-25-year-old Patients)
试验速览
- 阶段
- 不适用
- 状态
- 进行中（未招募）
- 入组人数
- 113
- 试验地点
- 20
- 主要终点
- Progression-free survival
研究概览
简要总结
Rhabdomyosarcoma (RMS) stands for the most frequent soft tissue sarcoma in children and, adolescents and young adults (AYA, 15-25-year-old population), accounting for approximately half of the whole soft tissue sarcomas in these populations.. Conversely, RMS represents a very small proportion of the soft tissue sarcomas in adults (3%), that is less than 1% of all solid cancers of adults.
To date, previous studies undertaken among the paediatric population have pointed out several prognostic factors such as tumor localisation, tumor invasiveness at diagnosis, tumor size, histological subset, and treatment plans. Age at diagnosis remains an independent prognostic factor.
RMS management is consensual in Europe for paediatric population, essentially based on the protocol RMS 2005 within the framework of the European Paediatric Soft tissue sarcoma Study Group (EpSSG). Care in AYAs remain heterogeneous and are either achieved in paediatric department, according to EpSSG guidelines, or in oncology department, known as "adult unit", depending on ESMO (European Society for Medical Oncology), which are non-specific recommendations for the management of rhabdomyosarcoma.
No consensus has been published yet for RMS in AYA despite the growing interest in cancers in AYA population - topic.supported by the French National Cancer Institute (INCa) - and the increasing network between paediatricians and adult-oncologists. Thus management of RMS in AYA remains patchy/unequal depending on the type of care unit.
Herein, with the support of the Oscar Lambret Center, we aim at assessing and identifying clinico-biological prognostic factors of rhabdomyosarcoma in AYA. Eventually, we hope to offer a standardized treatment to this population. Data collected from medical file will be anonymised in a confidential database of which the recipient is the sponsor of the study.
The ancillary study will aim at characterizing the molecular profile of the difficult-to-classify RMS subtypes (fusiform or pleomorphic subsets) in molecular biology for ambiguous cases.
From a scientific point of view, this study aims at understanding the parameters that may influence the prognosis of RMS in AYAs by evaluating various clinical and biological factors.
Biologically, molecular profiling of RMS in AYA may improve the characterization of this tumour in this age group.
At the clinical level, the completeness of the data collected will lead to a better description of RMS in AYAs. We hope to harmonize their therapeutic management by providing therapeutic adjustments according to population subsets.
Finally, these results could also help to adapt the therapeutic management of AYAs within the framework of the European protocol that is currently under construction, and will involve both children and adults.
详细描述
Rhabdomyosarcoma (RMS) is a rare tumor, with an incidence rate estimated at 4.5-6.9/1,000,000 in paediatric and, adolescent and young adult (AYA, 15 -25 years old) populations. RMS stands for the most frequent soft tissue sarcoma in children and adolescents, accounting for approximately half of the whole soft tissue sarcomas in these populations. Conversely, RMS represents a very small proportion of the soft tissue sarcomas in adults (3%), that is less than 1% of the solid cancers of the adult. To date, previous studies undertaken among the paediatric population have pointed out several prognostic factors such as tumor localisation, tumor invasiveness at diagnosis, tumor size, histological subset, and treatment plans. Age at diagnosis remains an independent prognostic factor. MYOD1 gene expression profile, more common in AYA, also seems to impair the prognosis.
RMS management is consensual in Europe for paediatric population, essentially based on the protocol RMS 2005 within the framework of the European Paediatric Soft tissue sarcoma Study Group (EpSSG). Care in AYAs remain heterogeneous and are either achieved in paediatric department, according to EpSSG guidelines, or in oncology department, known as "adult unit", depending on ESMO (European Society for Medical Oncology), which are non-specific recommendations for the management of rhabdomyosarcoma.
No consensus has been published yet for RMS in AYA despite the growing interest in cancers in AYA population - topic.supported by the French National Cancer Institute (INCa) - and the increasing network between paediatricians and adult-oncologists. Thus management of RMS in AYA remains patchy/unequal depending on the type of care unit.
Principal study
- To evaluate the prognostic value in terms of Progression Free Survival (PFS), in AYA patients with RMS, of clinical factors known as prognostic in children, and of biological factors (MYOD1 mutation in non-alveolar tumor; FOXO1).
- To describe clinical, histological and biological characteristics of RMS in AYA.
- To describe the therapeutic strategy according to the type of department (pediatric, AYA or adults):
- To evaluate the impact on PFS of treatment strategy
- To estimate the Overall Survival (OS) in AYA with RMS
- To estimate the OS curve after relapse for patients who achieved complete remission during first line of treatment
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
- 15 Years 至 25 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 是
入选标准
- •Patients aged from 15 to 25 years old at diagnosis
- •Patients registered in EpSSG RMS 2005 trial, or RRePS / NerSarc / Conticabase databases
- •Over the period from 2006 to 2014
- •Rhabdomyosarcoma histologically proven
- •Localized or metastatic
- •Histological review and molecular biology available
- •No previous treatment except surgery
- •No previous cancer
- •Absence of known serious chronic illness
- •Patient and/or parents information and non-opposition to data collection
排除标准
- •Age over 25 years, or under 15 years
- •Other histologies
结局指标
主要结局
Progression-free survival
时间窗: 3 years after diagnosis
To estimate progression-free survival in AYA patients with RMS, according to clinical factors known as prognostic in children, and of biological factors (MYOD1 mutation in non-alveolar tumor; FOXO1).
次要结局
- Clinical parameters of RMS in AYA(9 months)
- Biological parameters of RMS in AYA(9 months)
- therapeutic strategy(9 months)
- Overall survival (OS)(3 years after diagnosis)