PILOT STUDY OF GERIATRIC ASSESSMENTS IN SENIOR ADULTS WITH MULTIPLE MYELOMA

描述（由申请人提供）：老年多发性骨髓瘤（MM）患者的数量将在未来20年翻一番，但在基线老年综合征对老年骨髓瘤患者的治疗选择、毒性和结局的影响的知识方面存在根本性差距。迄今为止，没有MM研究包括全面的老年评估或评估治疗对老年领域的影响。该项目的长期目标是1)将老年评估纳入老年MM患者的护理中，以便临床医生可以预测MM治疗引起的毒性和功能下降；2)通过检查MM治疗在老年人中的实际有效性，为临床医生和患者提供决策方便。该项目的目的是比较接受高剂量治疗/自体干细胞移植（HDT/ASCT）的MM患者与未接受高剂量治疗/自体干细胞移植（HDT/ASCT）的MM患者中老年综合征的患病率，并确定哪些患者容易出现功能状态下降。中心假设是，基线时至少有一种IADL存在合并症或依赖的患者不太可能接受HDT/ASCT的强化治疗，并且在随访6个月后，合并症患者更有可能在IADL中出现残疾。这项研究的基本原理是，了解基线老年因素对治疗决策的影响，并确定哪些患者有功能衰退的风险，将允许对有功能衰退风险的老年MM患者进行更个性化的决策和有针对性的干预。本研究将通过两个具体目标来实现：1)探讨新诊断的老年多发性骨髓瘤的基线老年学评估与初始治疗选择之间的关系；2)确定基线老年综合征是否预测新诊断多发性骨髓瘤的老年人随访6个月时功能状态的下降。目的1将在一项前瞻性先导队列研究中完成，研究对象为30名65岁以上的近期在Siteman癌症中心/华盛顿大学就诊的MM患者。患者将完成癌症与衰老研究组的老年评估工具；将评估基线老年评估与初始治疗策略（HDT/ASCT vs非移植）之间的关系。在Aim 2中，参加Aim 1的队列将在随访3个月和6个月时进行重复的老年病学评估；将确定功能下降的危险因素。这项拟议研究的贡献是重要的，因为它是连续研究的第一步，旨在比较HDT/ASCT与非移植治疗策略在疾病控制和治疗对生存质量的影响方面的有效性。该研究具有创新性，因为它预测了即将发生的范式转变，即癌症正在成为一种慢性疾病，使用MM作为恶性肿瘤模型来研究老年综合征、治疗选择、癌症治疗的长期毒性和生存之间的相互关系。

DESCRIPTION (provided by applicant): The number of older adults with multiple myeloma (MM) will double in the next two decades, yet there is a fundamental gap in knowledge of the impact of baseline geriatric syndromes on treatment selection, toxicity and outcomes in older adults with MM. No MM study to date has included comprehensive geriatric assessments or assessed the impact of therapy on geriatric domains over time. The long-term goals of this project are 1) to integrate geriatric assessments into the care of senior adults with MM so that clinicians may predict toxicity and functional decline due to MM therapy and 2) to facilitate decision-making for clinicians and patients by examining the real-world effectiveness of MM therapy in older adults. The objectives of this project are to compare the prevalence of geriatric syndromes in MM patients who receive high dose therapy/autologous stem cell transplant (HDT/ASCT) versus those who do not, and to identify which patients are vulnerable to decline in functional status. The central hypotheses are that patients with comorbidities or dependence in at least one IADL at baseline will be less likely to undergo intense therapy with HDT/ASCT and that patients with comorbidities will be more likely to develop disability in their IADLs after 6 months of followup. The rationale for the proposed research is that understanding the impact of baseline geriatric factors on treatment decisions and identifying which patients are at risk for functional decline will allow more personalized decision-making and targeted interventions for older MM patients at risk for decline. The objectives will be accomplished through two specific aims: 1) To explore the relationship between baseline geriatric assessments and initial treatment selection in older adults with newly diagnosed multiple myeloma; and 2) To determine whether baseline geriatric syndromes predict decline in functional status at 6 months of follow-up in older adults with newly diagnosed multiple myeloma. Aim 1 will be accomplished in an prospective pilot cohort study of 30 patients over the age of 65 with recently diagnosed MM seen at Siteman Cancer Center/ Washington University. Patients will complete the Cancer and Aging Research Group geriatric assessment tool; associations between baseline geriatric assessments and initial treatment strategy (HDT/ASCT vs nontransplant) will be evaluated. For Aim 2, the cohort enrolled in Aim 1 will undergo repeat geriatric assessments at 3 and 6-months of follow-up; risk factors for functional decline will be identified. The contribution of this propsed research is significant because it is the first step in a continuum of research expected to compare the effectiveness of HDT/ASCT to non-transplant treatment strategies both in terms of disease-control and the impact of therapy on quality of survival. The proposed research is innovative because it anticipates an impending paradigm shift wherein cancer is becoming a chronic illness, using MM as a model malignancy to study the interrelationships among geriatric syndromes, treatment selection, long-term toxicities of cancer treatment and survival.