文章：

冰岛筛查、治疗或预防多发性骨髓瘤（iStopMM）：一项针对意义未明单克隆丙种球蛋白病的人群筛查研究及随访策略的随机对照试验

Iceland screens, treats, or prevents multiple myeloma (iStopMM): a population-based screening study for monoclonal gammopathy of undetermined significance and randomized controlled trial of follow-up strategies

原文发布日期：2021-05-17

DOI: 10.1038/s41408-021-00480-w

类型: Article

开放获取: 是

英文摘要：

Monoclonal gammopathy of undetermined significance (MGUS) precedes multiple myeloma (MM). Population-based screening for MGUS could identify candidates for early treatment in MM. Here we describe the Iceland Screens, Treats, or Prevents Multiple Myeloma study (iStopMM), the first population-based screening study for MGUS including a randomized trial of follow-up strategies. Icelandic residents born before 1976 were offered participation. Blood samples are collected alongside blood sampling in the Icelandic healthcare system. Participants with MGUS are randomized to three study arms. Arm 1 is not contacted, arm 2 follows current guidelines, and arm 3 follows a more intensive strategy. Participants who progress are offered early treatment. Samples are collected longitudinally from arms 2 and 3 for the study biobank. All participants repeatedly answer questionnaires on various exposures and outcomes including quality of life and psychiatric health. National registries on health are cross-linked to all participants. Of the 148,704 individuals in the target population, 80 759 (54.3%) provided informed consent for participation. With a very high participation rate, the data from the iStopMM study will answer important questions on MGUS, including potentials harms and benefits of screening. The study can lead to a paradigm shift in MM therapy towards screening and early therapy.
 

摘要翻译： 

意义未明的单克隆丙种球蛋白病是多发性骨髓瘤的前期病变。基于人群的MGUS筛查可识别多发性骨髓瘤早期治疗的候选者。本研究介绍了冰岛骨髓瘤筛查、治疗与预防项目——这是首个包含随访策略随机试验的基于人群的MGUS筛查研究。项目招募1976年前出生的冰岛居民，在冰岛医疗系统采血时同步收集血液样本。MGUS阳性参与者被随机分入三个研究组：第一组不进行主动联系，第二组遵循现行指南随访，第三组采用更密集的随访策略。疾病进展者将接受早期治疗。研究纵向收集第二、三组的样本存入生物样本库，所有参与者需反复填写涵盖生活质量、精神健康等多维度指标的问卷，并与国家健康登记系统进行数据关联。目标人群148,704人中，80,759人（54.3%）签署知情同意书。凭借极高的参与率，iStopMM研究数据将解答关于MGUS筛查潜在获益与危害的重要问题，可能推动多发性骨髓瘤治疗模式向筛查与早期治疗转变。

原文链接：

Iceland screens, treats, or prevents multiple myeloma (iStopMM): a population-based screening study for monoclonal gammopathy of undetermined significance and randomized controlled trial of follow-up strategies