■上皮样血管内皮瘤(EHE)是一种非常罕见的,血管肉瘤,临床表现从惰性到侵袭性。超过50%的患者出现转移性疾病,需要全身治疗,尽管没有专门批准用于EHE的全身疗法。回顾性证据支持mTOR抑制剂(例如西罗莫司)的活性,虽然仅在标签外可用。因此,EHE患者和倡导者正在努力通过收集患者观点和经验的数据来支持有效治疗的批准。
■2023年2月,EHE罕见癌症慈善机构(英国)和EHE基金会(美国),与其他倡导者,对EHE患者关于西罗莫司的使用和可及性的观点和经验进行了调查。调查由20个问题组成,这些问题是为接受治疗的个人设计的,那些接受治疗的人,或者从未接受过药物治疗。在患者社区中广泛推广,在线调查将患者分为三个队列进行分析:肝移植患者,曾服用西罗莫司的非移植患者和未服用西罗莫司的非移植患者.
■该调查评估了来自21个国家/地区的129名患者的反应数据,主要来自美国,英国,澳大利亚,加拿大(70%)。肝脏移植,西罗莫司和非西罗莫司队列为16%,25%和59%,分别。在西罗莫司组中,66%报告治疗持续时间超过一年,16%的人超过五年,说明药物的功效。在非西罗莫司组中,42%的药物未获得,11%的西罗莫司可获得,但由于其超标签状态,未选择用于治疗.总的来说,在所有队列中,87%的患者表示药物的可用性非常重要或非常重要。
调查答复强调了西罗莫司对EHE的活性,以及确保药物标签扩展的重要性,从而为患者提供公平的治疗机会。
UNASSIGNED: Epithelioid hemangioendothelioma (EHE) is an ultra-rare, vascular sarcoma with clinical presentation ranging from an indolent to an aggressive form. Over 50% of patients present with metastatic disease, requiring systemic therapy, although no systemic therapies are specifically approved for EHE. Retrospective evidence supports the activity of mTOR inhibitors (e.g. sirolimus), although available only off-label. EHE patients and advocates are therefore working to support approval of effective treatments by collecting data on patient perspectives and experiences.
UNASSIGNED: In February 2023, the EHE Rare Cancer Charity (UK) and The EHE Foundation (US), with other advocates, conducted a survey of perspectives and experiences of EHE patients regarding the use and accessibility of sirolimus. The survey consisted of 20 questions designed for individuals undergoing treatment, those who had been treated, or had never been treated with the drug. Widely promoted within the patient community, the online survey categorized patients into three cohorts for the analysis: liver transplant patients, non-transplant patients who had ever taken sirolimus and sirolimus-naïve non-transplant patients.
UNASSIGNED: The survey evaluated data from 129 patient responses from 21 countries, mostly from USA, UK, Australia, and Canada (70%). The liver transplant, sirolimus and non-sirolimus cohorts were 16%, 25% and 59%, respectively. In the sirolimus group 66% reported treatment durations exceeding one year, with 16% exceeding five years, indicating the drug\'s efficacy. In the non-sirolimus group, the drug was not available for 42% and for 11% sirolimus was available but not selected for treatment because of its off-label status. Overall, 87% of all patients across all cohorts expressed the importance of the drug\'s availability as hugely or very important.
UNASSIGNED: The survey responses highlight the activity of sirolimus for EHE and the importance of securing a label extension for the drug delivering equitable access to this treatment for patients.