Ríos-Tamayo Rafael, Lecumberri Ramón, Cibeira María Teresa, González-Calle Verónica, Alonso Rafael, Domingo-González Amalia, Landete Elena, Encinas Cristina, Iñigo Belén, Blanchard María-Jesús, Alejo Elena, Krsnik Isabel, Gómez-Bueno Manuel, Garcia-Pavia Pablo, Segovia-Cubero Javier, Rosiñol Laura, Lahuerta Juan-José, Martínez-López Joaquín, Bladé Joan
Hospital Universitario Puerta de Hierro Majadahonda, IDIPHISA, CIBERCV, 28222 Madrid, Spain.
Clínica Universidad de Navarra, CCUN, IDISNA, Universidad de Navarra, 31008 Pamplona, Spain.
Cancers (Basel). 2024 Apr 26;16(9):1689. doi: 10.3390/cancers16091689.
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 AL 淀粉样变性是一种具有挑战性的疾病，在日常临床实践中，许多患者被认为身体虚弱。然而，迄今为止尚无研究探讨身体虚弱及其对这些患者预后的影响。我们基于三个与身体虚弱相关的变量构建了一个简单的评分系统来预测死亡率，这三个变量分别是：年龄、东部肿瘤协作组（ECOG）体能状态（<2 与≥2）以及 N 末端脑钠肽前体（NT-proBNP）（<8500 与≥8500 ng/L）。来自西班牙骨髓瘤研究组十个研究点的 416 例连续新诊断患者符合该研究的入选标准。该评分系统在一个转诊中心的推导队列中建立，并在一个多中心队列中进行了外部验证。多变量分析表明，这三个变量是生存的独立预测因素。在两个队列中，该评分系统都能够根据总生存期和早期死亡率区分出四组患者。我们还使用查尔森合并症指数分析了合并症情况，但它在模型中未达到统计学显著性。我们创建了一个列线图，以便轻松估计每个患者在每个时间点的死亡风险。这个评分系统是一种简单、可靠且有效的方法，可在诊断时及整个随访过程中动态评估身体虚弱相关的死亡率。对于身体虚弱的 AL 淀粉样变性患者，最佳治疗方案仍有待确定，但我们建议，对身体虚弱相关风险的评估可以补充当前的分期系统，在这个复杂的临床场景中为临床决策增加价值。
