Determining the clinical significance of monoclonal gammopathy of undetermined si

确定未确定的单克隆丙种球蛋白病的临床意义

• 批准号: 8114585
• 负责人: Ronald S Go
• 金额: $ 5.91万
• 依托单位: GUNDERSEN LUTHERAN MEDICAL FOUNDATION
• 依托单位国家: 美国
• 财政年份: 2011
• 资助国家: 美国
• 起止时间: 2011-07-01 至 2013-06-30
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/8114585
• 关键词: Accounting; Age; American Society of Clinical Oncology; Biological; Cause of Death; Clinical; Complication; Data; Databases; Diagnosis; Dialysis procedure; Disease; Economic Burden; Fracture; Goals; Health Care Costs; Healthcare Systems; Hematologist; Hypercalcemia; Improve Access; Kidney Diseases; Laboratories; Link; Malignant - descriptor; Malignant Neoplasms; Medical Surveillance; Medicare; Monoclonal Gammapathies; Monoclonal gammopathy of uncertain significance; Multiple Myeloma; Oncologist; Outcome; Patients; Physical Examination; Prevalence; Retrospective Studies; Risk; SEER Program; Symptoms; Testing; United States; Waldenstrom Macroglobulinemia; cancer care; cancer complication; cancer diagnosis; cancer type; clinically significant; cohort; experience; follow-up; patient population; prevent; psychological distress; surveillance strategy

DESCRIPTION (provided by applicant): Multiple myeloma (MM) is uniformly preceded by monoclonal gammopathy of undetermined significance (MGUS). While the biological significance of MGUS has been established, its clinical significance has yet to be determined. MGUS is generally discovered incidentally during the work-up of patients with signs or symptoms suggestive of MM and related malignancies. The prevalence of MGUS is high (3% and 5% of people age over 50 and 70 years, respectively), but the risk of transformation to MM and related malignancies is very low (0.4% per year after taking into account competing causes of death). Yet experts recommend indefinite annual follow- up of MGUS patients with physical examinations and laboratory tests, even though no currently available treatment can prevent malignant transformation and no evidence exists to suggest that such follow-up contributes to better clinical outcomes. In fact, follow-up may cause psychological distress to patients due to cancer anticipation, may represent an enormous economic burden on the health care system (3.6 million people in 2008 harboring MGUS), and may decrease access to cancer care (shortfall of 4000 hematologist- oncologists by 2020 projected by the American Society of Clinical Oncology). Therefore, the clinical significance of MGUS must be determined. Toward that goal, and utilizing the Surveillance Epidemiology and End Results (SEER) program and the linked Medicare data, our study has 2 specific aims. In Aim 1, we will determine whether there is a difference in overall and disease-specific survival between patients with MM and related malignancies who presented de novo and those who had a preceding diagnosis of MGUS. In Aim 2, we will compare the rates of major cancer complications (hypercalcemia, bone fracture, and dialysis- dependent kidney disease) between these 2 groups. In Aim 3, we will determine the impact of various MGUS follow-up strategies on clinical outcome. We will use the Surveillance Epidemiology and End Results (SEER) program and the linked Medicare data. PUBLIC HEALTH RELEVANCE: The findings of our study will determine whether MGUS patients who experience transformation into a malignancy and who have maintained routine follow-up have better clinical outcomes than those who have not been followed. If routine follow-up of MGUS does not result in better outcomes, discontinuing the practice has the potential to relieve the psychological distress of cancer anticipation, to considerably reduce health care cost, and to improve access to cancer care in the United States.

描述（由申请人提供）：多发性骨髓瘤（MM）之前通常会发生未确定意义的单克隆γ病（MGUS）。虽然MGUS的生物学意义已经确立，但其临床意义尚未确定。MGUS通常是在有MM和相关恶性肿瘤体征或症状的患者的检查中偶然发现的。MGUS的患病率很高（分别占50岁和70岁以上人群的3%和5%），但转化为MM和相关恶性肿瘤的风险非常低（考虑到相互竞争的死亡原因后，每年0.4%）。然而，专家建议对MGUS患者进行无限期的年度随访，进行体检和实验室检查，尽管目前没有可用的治疗方法可以预防恶性转化，也没有证据表明这种随访有助于改善临床结果。事实上，由于对癌症的预期，随访可能会给患者带来心理困扰，可能会给医疗保健系统带来巨大的经济负担（2008年有360万人患有MGUS），并可能减少获得癌症护理的机会（美国临床肿瘤学会预计到2020年将缺少4000名血液肿瘤学家）。因此，必须确定MGUS的临床意义。为了实现这一目标，并利用监测流行病学和最终结果（SEER）计划和相关的医疗保险数据，我们的研究有两个具体目标。在Aim 1中，我们将确定MM及相关恶性肿瘤患者的总生存率和疾病特异性生存率是否与先前诊断为MGUS的患者有差异。在Aim 2中，我们将比较两组之间主要癌症并发症（高钙血症、骨折和透析依赖性肾病）的发生率。在Aim 3中，我们将确定各种MGUS随访策略对临床结果的影响。我们将使用监测流行病学和最终结果（SEER）项目和相关的医疗保险数据。