Cogle Christopher R, Kurtin Sandra E, Bentley Tanya G K, Broder Michael S, Chang Eunice, Megaffin Scott, Fruchtman Steven, Petrone Michael E, Mukherjee Sudipto
Division of Hematology and Oncology, Department of Medicine, College of Medicine, University of Florida, Gainesville, Florida, USA
University of Arizona, Tucson, Arizona, USA.
Oncologist. 2017 Apr;22(4):379-385. doi: 10.1634/theoncologist.2016-0211. Epub 2017 Mar 10.
Although hypomethylating agents (HMAs) are effective and approved therapies for patients with myelodysplastic syndromes (MDS), many patients do not benefit from treatment, and nearly all ultimately stop responding to HMAs. The incidence and cost burden of HMA failure are unknown yet needed to appreciate the magnitude and significance of such failure.
We analyzed a de-identified dataset of over 5 million individuals with private health insurance in the U.S. to estimate MDS incidence, prevalence, and treatments. Based on MDS provider interviews, a conceptual model of MDS patient management was constructed to create a new, claims-relevant and drug development-relevant definition of HMA treatment failure. This algorithm was used to define resource encumbrance of MDS patients in whom HMA treatment failed.
We estimated an MDS incidence rate of ∼70 cases per 100,000 enrollees per year and a prevalence of 155 cases per 100,000 enrollees. The proportion of MDS patients receiving HMA treatment was low (∼3%), and treatment was typically initiated within 1 year of the first MDS claim. Notably, HMA-treated individuals were older and had more comorbidities than the overall MDS cohort. Total health care costs of managing MDS patients after HMA failure were high (∼$77,000 during the first 6 months) and were driven primarily by non-pharmacy costs.
This study quantifies for the first time the burden of significant unmet need in caring for MDS patients following HMA treatment failure. 2017;22:379-385 U.S.-based treatment patterns among MDS patients demonstrate the significant clinical, financial, and health care burden associated with HMA failure and call for active therapies for this patient population.
尽管低甲基化药物（HMAs）是治疗骨髓增生异常综合征（MDS）患者的有效且已获批准的疗法，但许多患者并未从治疗中获益，而且几乎所有患者最终都会对HMAs不再产生反应。HMA治疗失败的发生率和成本负担尚不清楚，但了解此类失败的程度和重要性很有必要。
我们分析了美国超过500万拥有私人医疗保险的个人的去识别数据集，以估计MDS的发病率、患病率和治疗情况。基于对MDS医疗服务提供者的访谈，构建了一个MDS患者管理的概念模型，以创建一个与索赔相关且与药物开发相关的HMA治疗失败的新定义。该算法用于定义HMA治疗失败的MDS患者的资源负担。
我们估计MDS的发病率约为每年每10万名参保者中有70例，患病率为每10万名参保者中有155例。接受HMA治疗的MDS患者比例较低（约3%），且治疗通常在首次提出MDS索赔后的1年内开始。值得注意的是，接受HMA治疗的个体比整个MDS队列中的个体年龄更大，合并症更多。HMA治疗失败后管理MDS患者的总医疗费用很高（前6个月约77,000美元），且主要由非药房费用驱动。
本研究首次量化了HMA治疗失败后照顾MDS患者时重大未满足需求的负担。2017年；22:379 - 385美国MDS患者的治疗模式表明，与HMA治疗失败相关的临床、财务和医疗负担重大，需要为这一患者群体提供积极的治疗方法。
