文章：

预测不适合移植的多发性骨髓瘤患者的长期疾病控制：MGUS样特征的影响

Predicting long-term disease control in transplant-ineligible patients with multiple myeloma: impact of an MGUS-like signature

原文发布日期：2019-03-18

DOI: 10.1038/s41408-019-0176-x

类型: Article

开放获取: 是

英文摘要：

Disease control at 5 years would be a desirable endpoint for elderly multiple myeloma (MM) patients, but biomarkers predicting this are not defined. Therefore, to gain further insights in this endpoint, a population of 498 newly diagnosed transplant-ineligible patients enrolled in two Spanish trials (GEM2005MAS65 and GEM2010MAS65), has been analyzed. Among the 435 patients included in this post-hoc study, 18.6% remained alive and progression free after 5 years of treatment initiation. In these patients, overall survival (OS) rate at 10 years was 60.8% as compared with 11.8% for those progressing within the first 5 years. Hemoglobin (Hb) ≥ 12 g/dl (OR 2.74, p = 0.001) and MGUS-like profile (OR 4.18, p = 0.005) were the two baseline variables associated with long-term disease-free survival. Upon including depth of response (and MRD), Hb ≥ 12 g/dl (OR 2.27) and MGUS-like signature (OR 7.48) retained their predictive value along with MRD negativity (OR 5.18). This study shows that despite the use of novel agents, the probability of disease control at 5 years is still restricted to a small fraction (18.6%) of elderly MM patients. Since this endpoint is associated with higher rates of OS, this study provides important information about diagnostic and post-treatment biomarkers helpful in predicting the likelihood of disease control at 5 years.
 

摘要翻译： 

5年疾病控制是老年多发性骨髓瘤（MM）患者理想的治疗终点，但目前尚未明确预测该终点的生物标志物。为此，我们对两项西班牙临床试验（GEM2005MAS65和GEM2010MAS65）中498例新诊断且不适合移植的患者进行回顾性分析。在这项事后研究的435例患者中，18.6%在治疗启动5年后仍保持生存且无进展。这些患者的10年总生存（OS）率为60.8%，而在前5年内疾病进展的患者仅为11.8%。血红蛋白（Hb）≥12g/dl（OR 2.74, p=0.001）和MGUS样状态（OR 4.18, p=0.005）是与长期无病生存相关的两个基线变量。在纳入缓解深度（及MRD状态）分析后，Hb≥12g/dl（OR 2.27）、MGUS样特征（OR 7.48）与MRD阴性（OR 5.18）仍保持预测价值。本研究表明，即使使用新型药物，实现5年疾病控制的概率仍仅限于少数（18.6%）老年MM患者。由于该终点与更高总生存率相关，本研究为诊断及治疗后生物标志物的应用提供了重要信息，有助于预测5年疾病控制的可能性。

原文链接：

Predicting long-term disease control in transplant-ineligible patients with multiple myeloma: impact of an MGUS-like signature