Symptoms, unmet needs, and quality of life in Erdheim-Chester disease: a longitudinal registry-based analysis

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.