Myneni Saket, Tang Linda, Akbari Hanan, Mayne Raquel, Ahmed A Karim, Kazemi Foad, Dea Nicolas, Zwagerman Nathan T, Su Shirley Y, Choby Garret, Wang Eric W, Redmond Kristin J, McKean Erin L, Snyderman Carl H, Rowan Nicholas R, Mukherjee Debraj
Department of Neurosurgery, Johns Hopkins University, Baltimore, MD, USA.
School of Public Health, Johns Hopkins Bloomberg, Baltimore, MD, USA.
J Neurooncol. 2025 May;173(1):157-166. doi: 10.1007/s11060-025-04974-6. Epub 2025 Feb 19.
Chordomas are rare malignant tumors arising from the embryological notochord that present most frequently in the lumbosacral spine, followed by the skull base, with an overall 1/1,000,000 incidence. These tumors and their treatment significantly affect quality of life (QOL) due to intricate anatomical locations and aggressive treatment regimens. Despite these challenges, there are currently no disease-specific patient-reported outcome (PRO) surveys for chordomas. We aimed to develop a tool to assess QOL in patients with skull base chordomas (sbCs).
Twenty-seven patients who underwent sbC resection were interviewed on QOL throughout their care. Grounded-theory analysis of interview transcripts generated 7 themes. We developed an initial survey with 79 items from existing general and anatomic-specific QOL assessment tools addressing these themes. Ten chordoma providers and 10 new patients completed an anonymous Qualtrics survey, rating items' relevance on a 5-point Likert scale to validate survey content. An a priori cutoff of > 3.0 was used for significant relevance. Mean relevance scores for each item were compared between providers and patients as well as between skull base respondents and 5 control patients with lumbar spine and sacral chordomas, using two-sided Mann-Whitney U-tests.
Seventy-four items reached the relevance threshold. These were consolidated to create the final 42-item Skull Base Chordoma Patient Reported Outcome Survey (sbCPROS). Providers significantly overvalued items related to the themes of pain (73%), sleep changes (60%), and sensory & motor symptoms (43%) relative to patients. Ten items were more relevant to skull base patients than patients with spinal tumors (p < 0.05).
The authors developed a novel patient-centered, disease-specific PRO instrument to assess change in QOL for sbC patients over time. sbCPROS may provide significant insight into the delivery of high quality care for patients with sbCs and guide patient-physician discussions about care decision-making.
脊索瘤是一种罕见的恶性肿瘤，起源于胚胎脊索，最常发生于腰骶部脊柱，其次是颅底，总体发病率为1/1,000,000。由于其复杂的解剖位置和积极的治疗方案，这些肿瘤及其治疗会显著影响生活质量（QOL）。尽管存在这些挑战，但目前尚无针对脊索瘤的特定疾病患者报告结局（PRO）调查。我们旨在开发一种工具来评估颅底脊索瘤（sbC）患者的生活质量。
对27例行sbC切除术的患者在整个治疗过程中进行了生活质量访谈。对访谈记录进行扎根理论分析，得出7个主题。我们从现有的一般和解剖学特定生活质量评估工具中开发了一个包含79个条目的初始调查问卷，以解决这些主题。10名脊索瘤医疗服务提供者和10名新患者完成了一项匿名的Qualtrics调查，使用5点李克特量表对条目的相关性进行评分，以验证调查内容。显著相关性的先验临界值设定为> 3.0。使用双侧曼-惠特尼U检验比较医疗服务提供者和患者之间以及颅底受访者与5名腰椎和骶骨脊索瘤对照患者之间每个条目的平均相关性得分。
74个条目达到相关性阈值。这些条目经过整合，形成了最终的42条目颅底脊索瘤患者报告结局调查问卷（sbCPROS）。相对于患者，医疗服务提供者对与疼痛（73%）、睡眠变化（60%）以及感觉和运动症状（43%）主题相关的条目评估明显过高。10个条目对颅底患者比脊柱肿瘤患者更具相关性（p < 0.05）。
作者开发了一种新颖的以患者为中心、针对特定疾病的PRO工具，以评估sbC患者随时间推移的生活质量变化。sbCPROS可能为sbC患者提供高质量护理的显著见解，并指导患者与医生关于护理决策的讨论。
