Schuler Markus, Richter Stephan, Ehninger Gerhard, Bornhäuser Martin, Hentschel Leopold
Department of Internal Medicine I, University Hospital Carl Gustav Carus, Dresden, Germany.
Department of Internal Medicine II, Helios Hospital Emil von Behring, Berlin, Germany.
BMJ Open. 2017 Jun 30;7(6):e014614. doi: 10.1136/bmjopen-2016-014614.
Even with evolving and expanding therapeutical options for the treatment of advanced sarcomas over recent years, the balance between efficacy and toxicity still remains a major concern. Moreover, the symptom burden in patients with sarcoma remains high compared with other malignant diseases. It is, therefore, crucial to assess treatment effectiveness not only in terms of disease-related outcomes (eg, overall survival) but also from an individual and patient-centred perspective using the assessment of patient-reported outcomes (PROs). By focusing on PROs as a primary study endpoint, we aim to address key issues for patients with advanced soft tissue sarcoma (STS) undergoing palliative treatment.
The protocol of the YonLife study describes a multicentre, cluster-randomised, controlled, open-label proof-of-concept study conducted in patients with advanced or metastatic STS treated with trabectedin in seven German hospitals. The primary objective of the study is to exploratively compare overall quality of life between the patients receiving a multidimensional intervention based on individual PROs and those receiving usual supportive treatment. This complex intervention consists of the (1) electronic assessment of PRO, (2) creation of a case vignette based on PRO and clinical data and (3) treatment suggestions based on the discussion of these vignettes in a regularly meeting expert panel. Additionally, the YonLife trial assesses the applicability of a tablet-based assessment of PROs. Patients' and physicians' acceptance and challenges concerning the implementation process will be evaluated.
The YonLife trial has been approved by the Ethics Committee of the University Hospital Dresden as well as by the relevant institutions of each participating centre before patient enrolment. The findings will be reported via relevant peer-reviewed journals as well as through presentation at international conferences.
NCT02204111, pre-results.
尽管近年来晚期肉瘤的治疗选择不断发展和扩展，但疗效与毒性之间的平衡仍然是一个主要问题。此外，与其他恶性疾病相比，肉瘤患者的症状负担仍然很高。因此，评估治疗效果不仅要从疾病相关的结果（如总生存期）方面进行，还要从个体和以患者为中心的角度，通过评估患者报告的结果（PROs）来进行。通过将PROs作为主要研究终点，我们旨在解决晚期软组织肉瘤（STS）接受姑息治疗患者的关键问题。
YonLife研究方案描述了一项在德国七家医院对接受曲贝替定治疗的晚期或转移性STS患者进行的多中心、整群随机、对照、开放标签的概念验证研究。该研究的主要目的是探索性地比较接受基于个体PROs的多维干预的患者与接受常规支持治疗的患者的总体生活质量。这种复杂的干预包括：（1）PRO的电子评估；（2）基于PRO和临床数据创建病例摘要；（3）在定期召开的专家小组会议上基于这些摘要的讨论提出治疗建议。此外，YonLife试验评估基于平板电脑的PRO评估的适用性。将评估患者和医生对实施过程的接受程度和挑战。
YonLife试验已在患者入组前获得德累斯顿大学医院伦理委员会以及每个参与中心的相关机构的批准。研究结果将通过相关的同行评审期刊以及在国际会议上的报告进行公布。
NCT02204111，预结果。
