Fondazione IRCCS Istituto Nazionale Tumori, Medical Oncology 2, Milan, Italy.
EHE Rare Cancer Charity, London, United Kingdom.
PLoS One. 2024 Aug 12;19(8):e0308387. doi: 10.1371/journal.pone.0308387. eCollection 2024.
Epithelioid hemangioendothelioma (EHE) is an ultra-rare sarcoma, marked by distinctive molecular and pathological features and with a variable clinical behavior. Its natural history is still partially understood, reliable prognostic and predictive factors are lacking and many questions are still open on the optimal management. In the context of EURACAN, a prospective registry specifically dedicated to EHE was developed and launched with the aim of providing, through high-quality prospective data collection, a better understanding of this disease.
Registry-based cohort study including only new cases of patients with a pathological and molecularly confirmed diagnosis of EHE.
To improve the understanding of EHE natural history, validate and identify new prognostic and predictive factors, clarify the activity and efficacy of currently available treatment options, describe treatment pattern.
Settings and participantsIt is an hospital-based registry established in centers with expertise in EHE including adult patients with a new pathological and molecularly confirmed diagnosis of EHE starting from the 1st December 2023. The characteristics of each patient in the facility who meets the above-mentioned inclusion criteria will be collected prospectively and longitudinally with follow-up at cancer progression and / or cancer relapse or patient death. It is a secondary use of data which will be collected from the clinical records. The data collected for the registry will not entail further examinations or admissions to the facility and/or additional appointments to those normally provided for routine patient follow-up. VariablesFull details on patients and disease features, treatment and outcome will be collected, according to common clinical practice guidelines developed and shared with all the contributing centers. In addition, data on potential confounders (e.g. comorbidity; functional status etc.) will also be collected. Statistical methodsThe data analyses will include descriptive statistics and analytical analyses. Multivariable Cox's proportional hazards model and Hazard ratios (HR) for all-cause or cause-specific mortality will be used to determine independent predictors of overall survival, recurrence and progression.
The registry has been joined by 21 sarcoma reference centers across EU and UK, covering 10 countries. Patients' recruitment started in December 2023. The estimated completion date is December 2033 upon agreement on the achievement of all the registry objectives. The already established collaboration and participation of EHE patient's associations involved in the project will help in promoting the registry and fostering accrual.
上皮样血管内皮细胞瘤（EHE）是一种极为罕见的肉瘤，具有独特的分子和病理特征，临床表现多样。其自然病史仍不完全清楚，缺乏可靠的预后和预测因素，许多问题仍未解决，包括最佳治疗方案。EURACAN 组织开发并启动了一项专门针对 EHE 的前瞻性登记研究，旨在通过高质量的前瞻性数据收集，更好地了解这种疾病。
本研究为基于登记的队列研究，仅纳入经病理和分子学证实的 EHE 新病例。
提高对 EHE 自然病史的认识，验证和确定新的预后和预测因素，阐明现有治疗方案的疗效，描述治疗模式。
研究场所和参与者：该登记研究在具有 EHE 专业知识的中心开展，纳入 2023 年 12 月 1 日起新诊断为 EHE 的成年患者。对符合上述纳入标准的每个患者的特征进行前瞻性和纵向收集，随访至癌症进展和/或癌症复发或患者死亡。该研究将二次使用从临床记录中收集的数据，登记不会导致对该场所的进一步检查或入院，也不会导致对常规患者随访的额外预约。
将根据制定并与所有参与中心共享的常见临床实践指南，全面收集患者和疾病特征、治疗和结局等详细信息。此外，还将收集潜在混杂因素（如合并症；功能状态等）的数据。
数据分析将包括描述性统计和分析性分析。多变量 Cox 比例风险模型和全因或特定原因死亡率的风险比（HR）将用于确定总生存、复发和进展的独立预测因素。
该登记研究已在欧盟和英国的 21 个肉瘤参考中心开展，涵盖 10 个国家。患者招募已于 2023 年 12 月开始。预计在达成登记研究所有目标时，于 2033 年 12 月完成。该项目已建立合作关系，参与的 EHE 患者协会将有助于促进登记研究和增加患者入组。
