Systemic AL amyloidosis is a challenging disease for which many patients are considered frail in daily clinical practice. However, no study has so far addressed frailty and its impact on the outcome of these patients. We built a simple score to predict mortality based on three frailty-associated variables: age, ECOG performance status (<2 vs. ≥2) and NT-proBNP (<8500 vs. ≥8500 ng/L). Four-hundred and sixteen consecutive newly diagnosed patients diagnosed at ten sites from the Spanish Myeloma Group were eligible for the study. The score was developed in a derivation cohort from a referral center, and it was externally validated in a multicenter cohort. Multivariate analysis showed that the three variables were independent predictors of survival. The score was able to discriminate four groups of patients in terms of overall survival and early mortality in both cohorts. Comorbidity was also analyzed with the Charlson comorbidity index, but it did not reach statistical significance in the model. A nomogram was created to easily estimate the mortality risk of each patient at each time point. This score is a simple, robust, and efficient approach to dynamically assess frailty-dependent mortality both at diagnosis and throughout follow-up. The optimal treatment for frail AL amyloidosis patients remains to be determined but we suggest that the estimation of frailty-associated risk could complement current staging systems, adding value in clinical decision-making in this complex scenario.
系统性轻链型淀粉样变性是一种具有挑战性的疾病，在日常临床实践中许多患者被视为衰弱状态。然而，目前尚无研究探讨衰弱及其对这类患者预后的影响。我们基于三个与衰弱相关的变量构建了一个简易评分系统用于预测死亡率：年龄、ECOG体能状态（<2分 vs. ≥2分）和NT-proBNP水平（<8500 vs. ≥8500 ng/L）。本研究纳入了西班牙骨髓瘤工作组十个中心连续收治的416例新诊断患者。该评分系统在转诊中心的推导队列中建立，并在多中心队列中进行外部验证。多变量分析显示这三个变量均为生存期的独立预测因子。该评分在两个队列中均能有效区分四组不同总生存期和早期死亡风险的患者群体。研究同时采用查尔森合并症指数评估合并症情况，但该指标在模型中未达到统计学显著性。我们构建了列线图以便动态评估各时间点的个体死亡风险。该评分系统为动态评估诊断时及随访期间衰弱相关死亡率提供了一种简便、稳健且高效的方法。虽然衰弱型轻链淀粉样变性患者的最佳治疗方案仍有待确定，但我们建议衰弱相关风险评估可补充现有分期系统，为此复杂临床情境中的诊疗决策提供附加价值。
A Simple Frailty Score Predicts Survival and Early Mortality in Systemic AL Amyloidosis
肿瘤(癌症)患者之家