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）在不确定的显着性（MGUS）的单克隆性伽马病（MGUS）之前均匀。尽管已经建立了MGU的生物学意义，但其临床意义尚未确定。通常在有症状或症状的患者进行MM和相关恶性肿瘤的患者的检查过程中偶然发现了MGU。 MGU的患病率很高（分别超过50岁和70岁的人的3％和5％），但是转变为MM和相关恶性肿瘤的风险非常低（考虑到竞争性死亡原因，每年0.4％）。然而，专家建议无限期进行体格检查和实验室测试的MGU患者的年度随访，尽管目前没有可用的治疗可以防止恶性转化，并且没有证据表明这种随访会导致更好的临床结果。实际上，随访可能会因癌症的预期引起患者的心理困扰，可能代表医疗保健系统的巨大经济负担（2008年有360万人携带MGU），并可能减少获得癌症护理的机会（到2020年，美国临床肿瘤学会预测的4000名血液学家 - 肿瘤学家的短缺）。因此，必须确定MGU的临床意义。为了实现这一目标，并利用监视流行病学和最终结果（SEER）计划以及链接的Medicare数据，我们的研究具有2个具体目标。在AIM 1中，我们将确定MM和相关恶性肿瘤的患者和对MGU进行过诊断的患者的整体和疾病特异性生存率是否存在差异。在AIM 2中，我们将比较这两组之间重大癌症并发症（高钙血症，骨骼骨折和透析肾脏疾病）的发生率。在AIM 3中，我们将确定各种MGU随访策略对临床结果的影响。我们将使用监视流行病学和最终结果（SEER）计划以及链接的Medicare数据。 公共卫生相关性：我们的研究结果将确定经历转变为恶性肿瘤并保持常规随访的MGUS患者是否比没有受到遵循的患者更好。如果MGU的常规随访不会导致更好的结果，则停止这种做法有可能减轻癌症预期的心理困扰，大大降低医疗保健成本，并改善美国获得癌症护理的机会。