Monga Neerav, Garside Jamie, Gurung Binu, Quigley Joan, O'Donovan Peter, Tapprich Christoph, Nastoupil Loretta, Thieblemont Catherine, Loefgren Christina
Global Market Access and Health Policy, Janssen Global Oncology, 19 Green Belt Dr., Toronto, ON, M3C 1L9, Canada.
HEMAR Janssen, High Wycombe, UK.
Pharmacoecon Open. 2020 Dec;4(4):575-591. doi: 10.1007/s41669-020-00204-z.
Follicular lymphoma (FL) and marginal zone lymphoma (MZL) are types of indolent non-Hodgkin lymphoma (NHL) that develop in the B lymphocytes (also known as B cells).
The aim of this study was to conduct a comprehensive review of studies relating to cost effectiveness, costs and resource use, and health-related quality of life (HRQoL) in patients with FL or MZL.
Three separate systematic reviews were conducted to identify all published evidence on cost effectiveness, costs and resource use, and HRQoL between 2007 and March 2017 using the MEDLINE, MEDLINE in-process, E-pubs ahead of print (Ovid SP), Embase (Ovid SP), NHS EED, and EconLit databases. Select congress proceedings were also searched. Two systematic reviewers independently reviewed titles, abstracts, and full papers against eligibility criteria. Relevant data were extracted into bespoke data extraction templates (DETs) by a single systematic reviewer; these data were then validated for accuracy by a second reviewer against clean copies of the relevant publications.
A total of 25 cost-effectiveness studies (24 in FL; 1 in FL and MZL) met the eligibility criteria. Markov models were the most utilised cost-effectiveness model. US FL studies reported an incremental cost-effectiveness ratio (ICER) of $28,565/QALY for first-line rituximab-cyclophosphamide, vincristine, and prednisone (R-CVP) versus CVP, and $43,000/QALY for second-line obinutuzumab plus bendamustine (G + B) followed by G maintenance versus B. In the UK, ICERs were £1529-10,834/quality-adjusted life-year (QALY) for first-line rituximab + chemotherapy versus chemotherapy, £27,988/QALY for second-line G + B + G-maintenance versus B, and £62,653/QALY for second-line idelalisib versus chemotherapy and/or rituximab. Five costs/resource use and four HRQoL studies were identified in FL, and none in MZL. US mean lifetime costs in first-line patients ranged from $108,000 (rituximab) to $130,300 (rituximab-cyclophosphamide, doxorubicin hydrochloride, vincristine and prednisolone [CHOP]), and from £2185 (watch-and-wait) to £17,054 (chemotherapy) in the UK. In a multinational study, more rituximab-refractory patients receiving G + B + G-maintenance reported a meaningful improvement in total FACT-Lym scores compared with patients receiving B. In the UK, total FACT-Lym scores were meaningfully higher for newly diagnosed patients compared with patients with progression (136.04 vs. 109.7).
We found a small body of evidence of quality of life, and potentially cost-effective treatment options for FL; however, no evidence was reported on MZL specifically. The significant data gaps in knowledge in these diseases demonstrate a marked need for further studies.
滤泡性淋巴瘤（FL）和边缘区淋巴瘤（MZL）是惰性非霍奇金淋巴瘤（NHL）的类型，发生于B淋巴细胞（也称为B细胞）。
本研究的目的是对与FL或MZL患者的成本效益、成本和资源使用以及健康相关生活质量（HRQoL）相关的研究进行全面综述。
进行了三项独立的系统评价，以使用MEDLINE、MEDLINE在研、E-pubs提前印刷版（Ovid SP）、Embase（Ovid SP）、NHS EED和EconLit数据库，识别2007年至2017年3月期间关于成本效益、成本和资源使用以及HRQoL的所有已发表证据。还检索了选定的会议论文集。两名系统评价员根据纳入标准独立审查标题、摘要和全文。一名系统评价员将相关数据提取到定制的数据提取模板（DETs）中；然后由另一名评价员对照相关出版物的清晰副本对这些数据的准确性进行验证。
共有25项成本效益研究（24项关于FL；1项关于FL和MZL）符合纳入标准。马尔可夫模型是最常用的成本效益模型。美国关于FL的研究报告，一线利妥昔单抗-环磷酰胺、长春新碱和泼尼松（R-CVP）与CVP相比，增量成本效益比（ICER）为28,565美元/QALY，二线奥妥珠单抗加苯达莫司汀（G + B）随后G维持与B相比为43,000美元/QALY。在英国，一线利妥昔单抗 + 化疗与化疗相比，ICER为1529 - 10,834英镑/质量调整生命年（QALY），二线G + B + G维持与B相比为27,988英镑/QALY，二线idelalisib与化疗和/或利妥昔单抗相比为62,653英镑/QALY。在FL中确定了5项成本/资源使用研究和4项HRQoL研究，在MZL中未确定。美国一线患者的平均终生成本范围为108,000美元（利妥昔单抗）至130,300美元（利妥昔单抗-环磷酰胺、盐酸多柔比星、长春新碱和泼尼松龙[CHOP]），在英国为2185英镑（观察等待）至17,054英镑（化疗）。在一项跨国研究中，与接受B治疗的患者相比，更多接受G + B + G维持治疗的利妥昔单抗难治性患者报告FACT-Lym总分有有意义的改善。在英国，新诊断患者的FACT-Lym总分与进展期患者相比有显著更高（136.04对109.7）。
我们发现了少量关于FL生活质量以及潜在成本效益治疗方案的证据；然而，未专门报告关于MZL的证据。这些疾病在知识方面的显著数据差距表明明显需要进一步研究。
