Schöffski P, Jones R L, Agulnik M, Blay J Y, Chalmers A, Italiano A, Pink D, Stacchiotti S, Valverde C, Vincenzi B, Wagner M J, Maki R
Department of General Medical Oncology, University Hospitals Leuven, Leuven; Laboratory of Experimental Oncology, KU Leuven, Leuven; Department of Oncology, Leuven Cancer Institute, Leuven, Belgium.
Sarcoma Unit, The Royal Marsden, London; The Institute of Cancer Research, London, UK.
ESMO Open. 2024 Jul;9(7):103487. doi: 10.1016/j.esmoop.2024.103487. Epub 2024 Jun 28.
Locally advanced (unresectable) or metastatic dedifferentiated liposarcoma (DDLPS) is a common presentation of liposarcoma. Despite established diagnostic and treatment guidelines for DDLPS, critical clinical gaps remain driven by diagnostic challenges, symptom burden and the lack of targeted, safe and effective treatments. The objective of this study was to gather expert opinions from Europe and the United States on the management, unmet needs and expectations for clinical trial design as well as the value of progression-free survival (PFS) in this disease. Other aims included raising awareness and educate key stakeholders across healthcare systems.
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.
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.
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.
局部晚期（不可切除）或转移性去分化脂肪肉瘤（DDLPS）是脂肪肉瘤的常见表现形式。尽管已有DDLPS的诊断和治疗指南，但由于诊断挑战、症状负担以及缺乏靶向、安全有效的治疗方法，关键的临床差距依然存在。本研究的目的是收集欧洲和美国专家对于DDLPS的管理、未满足的需求、临床试验设计的期望以及无进展生存期（PFS）在该疾病中的价值的意见。其他目标包括提高医疗系统中关键利益相关者的认识并对其进行教育。
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。专家们以9分制李克特量表对每个陈述进行评分。共识性意见被定义为≥75%的专家对某一陈述的评分≥7分。在共识会议上讨论了修订后的陈述。
在55条预定义陈述中，就疾病负担、治疗模式、未满足的需求、PFS的价值及其与总生存期（OS）的关联以及交叉试验设计等方面达成了43条共识。12条陈述被降优先级或与其他陈述合并。没有专家不同意的陈述。
本研究构成了首个关于DDLPS的国际德尔菲小组。其旨在探讨KOL对DDLPS疾病负担和未满足需求的看法、PFS的价值及其向OS获益的潜在转化，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、未满足需求及临床试验期望方面达成了一致。提高对DDLPS相关关键临床差距的认识有助于改善患者预后并支持创新治疗的开发。
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