Introduction: Patients with transplant-ineligible (TIE) multiple myeloma (MM) have high rates of symptom burden. The aim of this study was to develop and validate a prognostic model to predict symptoms in patients with TIE MM. Methods: In this population-based, retrospective cohort study, using multiple administrative health care databases linked using a unique encrypted patient identifier in Ontario, Canada, symptoms were identified using the patient self-reported Edmonton Symptom Assessment System (ESAS) at each clinic visit. The primary outcome was the presence of moderate-to-severe (ESAS score 4–10) symptoms (specifically symptoms of pain, tiredness, depression, and impaired well-being) within one year from the index date. Using the entire cohort, a multivariable logistic regression model with baseline covariates was developed to predict the risk of experiencing each of the above symptoms, categorized as moderate to severe within 1 year post-index date. Internal validation of the model was assessed via bootstrap validation methods. Results: A total of 1535 TIE adults with MM met the inclusion criteria. The median age was 75, with 25.2% of patients aged 80 years or older. In the multivariate analysis, baseline symptoms continued to be most associated with future symptom burden. Baseline severe pain (OR 9.84, 95% CI 6.29–15.7) was most associated with patients experiencing moderate–severe pain one year post-index date. Similarly, baseline severe tiredness (OR 17.34, 95% CI 9.00–33.42), baseline severe depression (OR 28.07, 95% CI 15.96–49.38), and baseline severely impaired well-being (OR 4.12, 95% CI 2.30–7.37) were the biggest predictors of patients experiencing moderate–severe tiredness, depression, and impaired well-being, respectively, at one year after the index date. Conclusions: Patients with MM experience persisting symptoms of pain, tiredness, depression, and impaired well-being, with baseline symptoms being the biggest predictor of future symptom burden.
引言：不适合移植的多发性骨髓瘤患者症状负担发生率较高。本研究旨在开发并验证一个预测此类患者症状的预后模型。方法：这项基于人群的回顾性队列研究利用加拿大安大略省通过独特加密患者标识符链接的多个行政医疗数据库，在每次门诊就诊时通过患者自报的埃德蒙顿症状评估系统识别症状。主要结局指标为索引日期后一年内出现中重度症状（ESAS评分4-10分），具体包括疼痛、疲劳、抑郁和健康状况受损症状。基于完整队列数据，我们建立了包含基线协变量的多变量逻辑回归模型，用于预测索引日期后一年内出现上述各项中重度症状的风险。通过自助法验证对模型进行了内部验证。结果：共1535名不适合移植的成年多发性骨髓瘤患者符合纳入标准。中位年龄75岁，25.2%的患者年龄≥80岁。多变量分析显示，基线症状与未来症状负担的相关性最为显著。基线重度疼痛患者出现中重度疼痛的比值比最高（OR 9.84，95% CI 6.29-15.7）。同样，基线重度疲劳（OR 17.34，95% CI 9.00-33.42）、基线重度抑郁（OR 28.07，95% CI 15.96-49.38）和基线健康状况严重受损（OR 4.12，95% CI 2.30-7.37）分别是预测索引日期后一年出现相应中重度症状的最强预测因子。结论：多发性骨髓瘤患者持续存在疼痛、疲劳、抑郁和健康状况受损等症状，其中基线症状是预测未来症状负担最重要的指标。
肿瘤(癌症)患者之家