Forsythe Anna, Kwon Christina S, Bell Timothy, Smith T Alexander, Arondekar Bhakti
Purple Squirrel Economics, New York, NY, USA,
Pfizer Inc, New York, NY, USA.
Clinicoecon Outcomes Res. 2019 Jan 14;11:87-98. doi: 10.2147/CEOR.S187409. eCollection 2019.
AML is a rapidly progressing bone marrow cancer, with poor survival rates compared to other types of leukemia. IC and NIC as well as BSC treatment options are available; however, there is scant published literature on the impact of disease and treatment on the HRQoL in patients receiving NIC.
This study determined the HRQoL among NIC AML patients.
Embase, Medline, Cochrane database, and conference abstracts were searched using the prespecified PICOS criteria from January 2000 to November 2017 for studies reporting HRQoL and patient preference utilities in NIC AML. Studies on patients with RAEB-t MDS, randomized clinical trials (RCTs), prospective observational studies, and patient surveys were included, while systematic reviews and meta-analyses were used for bibliographic searching.
Thirteen records from 12 original studies were identified. These included five records from four RCTs, three prospective studies, four patient survey studies, and one cost-effectiveness analysis. At baseline, NIC AML patients had poor HRQoL scores especially in fatigue (33) and GHS (50) on a 0-100 scale, with higher scores indicating better health. Low baseline HRQoL scores, especially PF and fatigue (<50) were shown to be significant independent predictors of poor survival. Clinical responders demonstrated meaningful improvements, especially in PF and fatigue, along with other health domains after being treated with NIC agents across several studies.
HRQoL is poor for patients with NIC AML; measures such as fatigue and PF at baseline have been identified as independent prognostic factors for overall survival with several studies showing improvement in both domains with treatment. RCTs should incorporate evaluation of treatment impact on patients' PF and fatigue as important measures of effectiveness.
急性髓系白血病(AML)是一种进展迅速的骨髓癌,与其他类型的白血病相比,生存率较低。现有强化化疗(IC)、非强化化疗(NIC)以及最佳支持治疗(BSC)等治疗方案;然而,关于疾病和治疗对接受NIC治疗的患者健康相关生活质量(HRQoL)影响的已发表文献却很少。
本研究确定了接受NIC治疗的AML患者的HRQoL。
使用预先设定的PICOS标准,检索2000年1月至2017年11月期间的Embase、Medline、Cochrane数据库及会议摘要,以查找报告NIC治疗AML患者的HRQoL和患者偏好效用的研究。纳入了有关难治性贫血伴原始细胞过多转变型骨髓增生异常综合征(RAEB-t MDS)患者的研究、随机临床试验(RCT)、前瞻性观察性研究及患者调查,同时使用系统评价和荟萃分析进行文献检索。
从12项原始研究中识别出13条记录。其中包括来自4项RCT的5条记录、3项前瞻性研究、4项患者调查研究以及1项成本效益分析。在基线时,接受NIC治疗的AML患者HRQoL评分较差,尤其是在疲劳(评分为33)和全球健康状况(GHS,评分为50)方面(0至100分制,分数越高表明健康状况越好)。低基线HRQoL评分,尤其是身体功能(PF)和疲劳评分(<50)被证明是生存不良的重要独立预测因素。在多项研究中,临床缓解者在接受NIC药物治疗后,尤其是在PF和疲劳方面,以及在其他健康领域都表现出有意义的改善。
接受NIC治疗的AML患者HRQoL较差;多项研究表明,基线时的疲劳和PF等指标已被确定为总生存的独立预后因素,且治疗后这两个领域均有改善。RCT应将评估治疗对患者PF和疲劳的影响作为有效性的重要指标纳入其中。