METHODS: An international panel of 12 sarcoma key opinion leaders (KOLs) was recruited. The study consisted of two rounds of surveys with pre-defined statements. Experts scored each statement on a 9-point Likert scale. Consensus agreement was defined as ≥75% of experts scoring a statement with ≥7. Revised statements were discussed in a consensus meeting.
RESULTS: Consensus was reached on 43 of 55 pre-defined statements across disease burden, treatment paradigm, unmet needs, value of PFS and its association with overall survival (OS), and cross-over trial design. Twelve statements were deprioritised or merged with other statements. There were no statements where experts disagreed.
CONCLUSIONS: This study constitutes the first international Delphi panel on DDLPS. It aimed to explore KOL perception of the disease burden and unmet need in DDLPS, the value of PFS, and its potential translation to OS benefit, as well as the relevance of a cross-over trial design for DDLPS therapies. Results indicate an alignment across Europe and the United States regarding DDLPS management, unmet needs, and expectations for clinical trials. Raising awareness of critical clinical gaps in relation to DDLPS can contribute to improving patient outcomes and supporting the development of innovative treatments.
方法:招募了一个由12名肉瘤关键意见领袖(KOL)组成的国际小组。该研究包括两轮带有预定义陈述的调查。专家以9分的李克特量表对每个陈述进行评分。共识被定义为≥75%的专家对陈述评分≥7。在协商一致会议上讨论了订正声明。
结果:关于疾病负担的55项预定义陈述中的43项达成了共识,治疗范式,未满足的需求,PFS的价值及其与总生存期(OS)的关系,和交叉试验设计。12个语句被取消优先级或与其他语句合并。没有专家不同意的陈述。
结论:本研究构成了第一个关于DDLPS的国际Delphi小组。它旨在探索KOL对DDLPS中疾病负担和未满足需求的看法,PFS的值,以及它潜在的转化为操作系统的好处,以及DDLPS治疗交叉试验设计的相关性。结果表明,欧洲和美国在DDLPS管理方面保持一致,未满足的需求,和对临床试验的期望。提高对与DDLPS相关的关键临床差距的认识可以有助于改善患者预后并支持创新治疗方法的开发。