Background: Evidence of the real-world management of dedifferentiated liposarcoma (DDLPS) is limited by the patient size and coding. The objective of this study is to generate consensus expert opinion on locally advanced or metastatic DDLPS diagnosis, treatment, and unmet needs.Methods: A three-round Delphi consensus panel was conducted with 9 DDLPS clinical experts from November to December 2023. Expert panelists were recruited across academic specialty and traditional settings and US regions. The Delphi panel included two rounds of surveys followed by a consensus building workshop. Surveys contained multiple-choice and free response questions, and statements for level of agreement rating. Panelists rated each statement for level of agreement on a 9-point Likert scale. Statements with ≥75% of scores ≥ 7 achieved consensus, and those that did not achieve consensus agreement were modified or removed from subsequent testing. A virtual workshop was held to discuss areas which did not achieve consensus and refine previously agreed upon statements.Results: In total 25 consensus statements were developed by the Delphi panel. Survey 1 achieved 7 consensus statements across the areas of burden, treatment, and unmet needs of DDLPS. Survey 2 generated an additional 10 consensus statements. During the workshop, eight more statements achieved consensus, and four statements were refined for enhanced clarity and precision. The study findings are limited by the number of Delphi panel participants and consensus statements may not be fully representative of clinician perspectives across the US.Conclusions: Consensus areas identified by the Delphi panel help better understand the decision factors for surgical and non-surgical treatments and anticipated utilization. These results could be used to inform both drug development programs as well as care delivery challenges for liposarcoma patients.
背景:去分化脂肪肉瘤(DDLPS)在真实世界中的管理证据受限于患者规模和编码。本研究旨在就局部晚期或转移性DDLPS的诊断、治疗及未满足需求形成专家共识意见。 方法:于2023年11月至12月期间,由9位DDLPS临床专家开展三轮德尔菲共识小组研究。专家组成员来自美国不同学术专科、传统医疗机构及地理区域。德尔菲小组研究包括两轮问卷调查及一轮共识构建研讨会。问卷包含多项选择题、自由回答题及需进行同意度评级的陈述条目。专家采用9点李克特量表对每条陈述的同意程度进行评分。评分≥7的专家比例≥75%的陈述即达成共识,未达成共识的陈述在后续测试中进行了修改或删除。通过线上研讨会讨论未达成共识的领域,并对已达成共识的陈述进行优化完善。 结果:德尔菲小组共形成25条共识声明。第一轮调查在DDLPS的疾病负担、治疗及未满足需求领域达成7项共识;第二轮调查新增10项共识声明;研讨会期间另有8项陈述达成共识,并对4项陈述进行了优化以提高表述的清晰度与精确性。本研究受德尔菲小组参与者数量限制,共识声明可能无法完全代表全美临床医生的观点。 结论:德尔菲小组确定的共识领域有助于更好理解手术与非手术治疗决策因素及预期实施方案。这些结果可为脂肪肉瘤患者的药物研发项目及临床护理实践提供参考依据。