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复发和/或难治性大 B 细胞淋巴瘤健康相关生活质量结局及相关效用值的系统文献综述

英文原题:A Systematic Literature Review of Health-Related Quality of Life Outcomes and Associated Utility Values in Relapsed and/or Refractory Large B Cell Lymphoma.

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A Systematic Literature Review of Health-Related Quality of Life Outcomes and Associated Utility Values in Relapsed and/or Refractory Large B Cell Lymphoma.

PubMed 2024/01/10(内容时间) Pharmacoecon Open Q2 · IF 2.4(JCR 2025)

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研究概要

本 SLR 中综合的证据为理解复发/难治性 LBCL 的 HRQOL 证据提供了全面的认识。

中文摘要

随着治疗选择不断增加,关于复发/难治性(R/R)大B细胞淋巴瘤(LBCL)的健康相关生活质量(HRQOL)结局及相关效用值,尚缺乏综合汇总,难以为医疗政策和新旧治疗产品的决策者评估提供依据。这些评估会直接影响患者和医生可选择的治疗方案。

开展系统文献综述(SLR),了解R/R LBCL治疗的HRQOL证据并识别相关健康效用值。

检索并筛选2003年1月1日至2022年5月2日发表的文献。依据预先制定的人群、干预、对照、结局和研究设计标准筛选研究,由两名独立评审者完成;并按照英国国家卫生与临床优化研究所(NICE)的卫生技术评估建议进行证据质量评估。纳入的治疗包括嵌合抗原受体(CAR)T细胞产品(lisocabtagene maraleucel、axicabtagene ciloleucel、tisagenlecleucel)、新型疗法(selinexor、nivolumab、polatuzumab vedotin和bendamustine)、挽救治疗及rituximab。

综述纳入33项报告HRQOL的独立研究,其中包括15项报告健康状态效用值的经济学研究、9项临床试验、7份卫生技术评估报告,以及各1项情景研究和时点调查。CAR-T 细胞治疗在二线及三线或更后线治疗中均可改善一般或淋巴瘤特异性HRQOL指标。CAR-T 治疗期间的效用值为0.50至0.74。缓解/无进展生存期的效用值(0.70至0.90)和疾病进展期的效用值(0.39至0.59)在研究间相近。新型疗法的无进展生存期效用值为0.83,疾病进展期为0.39至0.71。挽救化疗治疗期间的效用值为0.63至0.67。

本SLR综合证据全面呈现了R/R LBCL的HRQOL情况。文献中的多种效用值来源显示,不同治疗方式下患者HRQOL结局存在差异。CAR-T 治疗R/R LBCL与健康效用值改善相关。新型疗法和挽救疗法的结果不一。随着更多新疗法应用于这一患者群体,还需要更多数据为治疗决策提供依据。

展开英文摘要原文

In this ever-expanding treatment landscape, there is a lack of consolidated health-related quality of life (HRQOL) outcomes and utility reports in relapsed or refractory (R/R) large B cell lymphoma (LBCL) to inform health care policy and decision-maker assessments for both old and new products. These assessments can have a direct effect on what treatment options are available to patients and physicians.

A systematic literature review (SLR) was performed to understand the HRQOL evidence for treatments in R/R LBCL and identify associated health utility values.

The SLR searched and screened literature published from 1 January 2003 to 2 May 2022. Studies were screened based on Population, Intervention, Comparator, Outcome, Study design criteria established a priori and were assessed by two independent reviewers; quality assessments of the evidence were performed in accordance with health technology assessment recommendations from the National Institute for Health and Care Excellence. Several types of therapies were included, such as chimeric antigen receptor (CAR) T cell products (lisocabtagene maraleucel, axicabtagene ciloleucel, tisagenlecleucel), novel therapies (selinexor, nivolumab, polatuzumab vedotin, and bendamustine), salvage therapies, and rituximab.

The review identified 33 unique studies reporting HRQOL, including 15 economic studies that reported health state utility values, 9 clinical trials, 7 health technology assessment reports, and 1 each of a vignette-based study and a point-in-time survey. Improvements in general and/or lymphoma-specific HRQOL measures were observed with CAR T cell therapy in both the second-line and third-line or later settings. On-treatment utility values for CAR T cell therapies ranged from 0.50 to 0.74. Values for remission/progression-free survival (0.70-0.90) and for disease progression (0.39-0.59) were similar across studies. For novel therapies, utility values were 0.83 for progression-free survival and ranged from 0.39 to 0.71 for disease progression. On-treatment utility values for salvage chemotherapy ranged from 0.63 to 0.67.

Overall, the evidence synthesized in this SLR provides a comprehensive understanding of the HRQOL evidence in R/R LBCL. This article identified several sources for utility values in the published literature showing variation in the HRQOL outcomes for patients across a variety of therapeutics. Treatment of R/R LBCL with CAR T cell therapies was associated with improvement in health utility values. Mixed results were found for novel therapies and salvage therapies. More data are needed as new therapies are used in this patient population to inform treatment decision-making.

论文信息

作者
Liu FF、Bartlett M、Craigie S
单位
Bristol Myers Squibb, 3401 Princeton Pike, Lawrence Township, Princeton, NJ, 08648, USA. feifei.liu@bms.com.United States
文献类型
系统综述
期刊
PharmacoEconomics - open2024 Mar
原文标识
PubMed 38198111 · DOI 10.1007/s41669-023-00464-5