Marathe Priya H, Reiner Anne S, Bossert Dana, Sigler Allison M, Fournier Deanna, Brewer Kathleen, Goyal Gaurav, Atkinson Thomas M, Mao Jun J, Panageas Katherine S, Diamond Eli L
Department of Pediatrics, Memorial Sloan Kettering Cancer Center, New York, NY.
Department of Epidemiology and Biostatistics, Memorial Sloan Kettering Cancer Center, New York, NY.
Blood Adv. 2025 Sep 9;9(17):4415-4424. doi: 10.1182/bloodadvances.2024015659.
Measurement of patient-reported outcomes (PROs) and health-related quality of life (HrQOL) are crucial for comprehensive, patient-centered cancer care. Both PROs and HrQOL have been understudied in patients with Erdheim-Chester disease (ECD), a rare cancer with protean manifestations, dense symptomatology, and frequent diagnostic delay. We sought to evaluate the longitudinal evolution of symptom burden and unmet supportive care needs in patients with ECD, and to identify associations between these PROs and HrQOL. A registry-based cohort of patients with ECD completed a PRO battery including the Functional Assessment of Cancer Therapy-General (FACT-G) and other validated PRO measures. Descriptive statistics were used to characterize the distribution of PROs and FACT-G scores; PROs were modeled by univariable linear regression with FACT-G total score as the dependent variable at (1) registry enrollment and (2) 12-month time points. Changes in FACT-G total score (the difference between the 12-month and enrollment scores) were correlated with changes in PROs using univariable linear regression analysis. In 158 patients, mean total FACT-G was 70.8, lower than observed across multiple cancer cohorts. Higher levels of pain and fatigue, presence of neurologic symptoms, and greater number of unmet needs were all associated with worse HrQOL. Improvement in pain, fatigue, and unmet needs over 12 months was significantly associated with improvement in HrQOL. In patients with ECD, HrQOL is substantially diminished, even when considering other patients with cancer. Mitigation of symptoms and addressing unmet supportive care needs represent opportunities for intervention to improve HrQOL in ECD.
患者报告结局（PROs）和健康相关生活质量（HrQOL）的测量对于全面的、以患者为中心的癌症护理至关重要。在 Erdheim-Chester 病（ECD）患者中，PROs 和 HrQOL 均未得到充分研究，ECD 是一种罕见癌症，具有多样的表现、密集的症状学且诊断经常延迟。我们试图评估 ECD 患者症状负担和未满足的支持性护理需求的纵向演变，并确定这些 PROs 与 HrQOL 之间的关联。一个基于登记处的 ECD 患者队列完成了一组 PRO 评估，包括癌症治疗功能评估通用版（FACT-G）和其他经过验证的 PRO 测量。描述性统计用于描述 PROs 和 FACT-G 评分的分布；在（1）登记入组时和（2）12 个月时间点，以 FACT-G 总分作为因变量，通过单变量线性回归对 PROs 进行建模。使用单变量线性回归分析，将 FACT-G 总分的变化（12 个月和入组时分数的差值）与 PROs 的变化进行关联。在 158 名患者中，FACT-G 总分平均为 70.8，低于多个癌症队列的观察值。更高水平的疼痛和疲劳、神经症状以及更多未满足需求均与更差的 HrQOL 相关。12 个月内疼痛、疲劳和未满足需求的改善与 HrQOL 的改善显著相关。在 ECD 患者中，即使与其他癌症患者相比，HrQOL 也显著降低。减轻症状和满足未满足的支持性护理需求是改善 ECD 患者 HrQOL 的干预机会。
