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急性髓系白血病治疗领域的未满足临床需求和疾病经济负担。

Unmet clinical needs and economic burden of disease in the treatment landscape of acute myeloid leukemia.

机构信息

Department of Leukemia at The University of Texas, Houston, TX; MD Anderson Cancer Center, Houston, TX. Email:

Department of Leukemia at The University of Texas, Houston, TX; MD Anderson Cancer Center, Houston, TX.

出版信息

Am J Manag Care. 2018 Aug;24(16 Suppl):S347-S355.

Abstract

Acute myeloid leukemia (AML) is the most common form of acute leukemia in adults in the United States, with approximately 19,520 new cases estimated for 2018. Despite advances in the management of hematologic malignancies, the development of novel targeted and immune therapies, and improvements in supportive care, the overall outcome for patients with AML remains poor due to several factors, including increased frequency in the older population, poor response to chemotherapy, high relapse rates, and limited effective therapy options in relapsed patients. In addition, AML presents a substantial clinical and financial burden attributable in part to the heterogenic characteristics at presentation, such as varied age distributions and cytogenetic and molecular abnormalities, coupled with prolonged hospitalizations, high rates of infectious complications, and need for allogeneic stem cell transplants. Several unmet needs exist in AML. For instance, more-effective, less-toxic treatments are urgently needed because many patients with AML are not candidates for standard induction therapy. AML is also characterized by high rates of relapsed/refractory disease; therefore, strategies to reduce relapse are important. Other unmet needs center on poor quality of life from disease- and therapy-related toxicities and inadequate psychosocial support frequently experienced by many patients with AML and by their caregivers. This review will discuss these issues and additional challenges faced both by patients with AML and by caregivers and medical personnel who work in the AML setting.

摘要

急性髓系白血病(AML)是美国成年人中最常见的急性白血病形式,估计 2018 年有大约 19,520 例新发病例。尽管在血液恶性肿瘤的治疗、新型靶向和免疫治疗的开发以及支持性护理的改善方面取得了进展,但由于多种因素,包括老年人群中发病率的增加、对化疗的反应不佳、高复发率以及复发患者有效治疗选择有限,AML 患者的总体预后仍然较差。此外,AML 还带来了巨大的临床和经济负担,部分原因是其表现出的异质性特征,例如不同的年龄分布、细胞遗传学和分子异常,加上住院时间延长、感染并发症发生率高以及需要异体干细胞移植。AML 存在一些未满足的需求。例如,迫切需要更有效、毒性更低的治疗方法,因为许多 AML 患者不符合标准诱导治疗的条件。AML 还以高复发/难治性疾病率为特征;因此,减少复发的策略很重要。其他未满足的需求主要集中在疾病和治疗相关毒性导致的生活质量差,以及许多 AML 患者及其护理人员经常经历的心理社会支持不足。这篇综述将讨论这些问题以及 AML 患者和在 AML 环境中工作的护理人员和医务人员面临的其他挑战。

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