Choi H K, Seeger J D, Kuntz K M
Massachusetts General Hospital, Boston 02114, USA.
Arthritis Rheum. 2000 Oct;43(10):2316-27. doi: 10.1002/1529-0131(200010)43:10<2316::AID-ANR20>3.0.CO;2-6.
Recently, new treatment options for rheumatoid arthritis (RA) patients with an inadequate response to methotrexate (MTX) have become available. Given the wide variability in efficacy and costs among these different treatment options, we sought to determine their cost-effectiveness (CE) in order to guide policy in different cost-constrained settings.
We performed a CE analysis comparing 6 treatment options for patients with MTX-resistant RA: 1) etanercept + MTX, 2) etanercept monotherapy, 3) cyclosporine + MTX, 4) triple therapy (hydroxychloroquine, sulfasalazine, and MTX), 5) continuation of MTX monotherapy, and 6) no second-line agent. A decision model was used with a time horizon of 6 months. We used 2 measures of effectiveness based on published clinical trial data: the American College of Rheumatology 20% response criteria (ACR 20); and a weighted average of proportions of patients achieving responses of ACR 70, ACR 50, and ACR 20 (ACR 70 weighted response [ACR 70WR]). Incremental CE ratios were calculated as the additional cost per patient achieving either outcome, compared with the next least expensive option. To help interpret CE relative to these RA-specific outcomes, we conducted a separate, "reference" CE analysis of MTX use in MTX-naive RA patients, using the same outcomes.
In our reference analysis, MTX therapy for MTX-naive RA cost $1,100 per ACR 20 outcome and $1,500 per ACR 70WR, compared with no second-line agent. In our base-case analysis with either outcome, MTX continuation, cyclosporine + MTX, and etanercept monotherapy cost more, but either were not more efficacious or had a higher incremental CE ratio than the next most expensive option (i.e., they were dominated). Therefore, these options were not cost-effective. The least expensive option, triple therapy, cost 1.3 times more per patient with ACR 20 outcome ($1,500/ACR 20) and 2.1 times more per ACR 70WR ($3,100/ACR 70WR) than MTX therapy for MTX-naive RA. The most efficacious option, the combination of etanercept and MTX, cost 38 times more per patient with ACR 20 outcome ($4,600/ACR 20) and 23 times more per ACR 70WR ($34,800/ACR 70WR) than MTX therapy for MTX-naive RA. Overall, the results of extensive sensitivity analyses did not substantially affect these results.
Our analysis indicates that if 15 mg/week MTX is cost-effective for achieving ACR 20 or ACR 70WR in MTX-naive RA over a 6-month period, then most likely so is triple therapy in MTX-resistant RA. Whether etanercept + MTX is cost-effective depends on whether $34,800/ACR 70WR (or $42,600/ACR 20) over a 6-month period is considered acceptable.
最近，对于对甲氨蝶呤（MTX）反应不足的类风湿关节炎（RA）患者，已有新的治疗选择。鉴于这些不同治疗选择在疗效和成本方面存在很大差异，我们试图确定它们的成本效益（CE），以指导不同成本受限环境下的政策制定。
我们进行了一项成本效益分析，比较了针对MTX抵抗性RA患者的6种治疗选择：1）依那西普+MTX，2）依那西普单药治疗，3）环孢素+MTX，4）三联疗法（羟氯喹、柳氮磺胺吡啶和MTX），5）继续MTX单药治疗，6）不使用二线药物。使用了一个决策模型，时间跨度为6个月。我们基于已发表的临床试验数据使用了2种有效性指标：美国风湿病学会20%反应标准（ACR 20）；以及达到ACR 70、ACR 50和ACR 20反应的患者比例的加权平均值（ACR 70加权反应[ACR 70WR]）。增量成本效益比计算为达到任一结果的每位患者的额外成本，与下一个成本最低的选择相比。为了帮助解释相对于这些RA特异性结果的成本效益，我们使用相同的结果对初治RA患者使用MTX进行了单独的“参考”成本效益分析。
在我们的参考分析中，与不使用二线药物相比，初治RA患者使用MTX治疗达到每个ACR 20结果的成本为1100美元，达到每个ACR 70WR的成本为1500美元。在我们基于任一结果的基础分析中，继续使用MTX、环孢素+MTX和依那西普单药治疗成本更高，但要么疗效不更好，要么增量成本效益比高于下一个成本更高的选择（即它们被主导）。因此，这些选择不具有成本效益。最便宜的选择，三联疗法，达到ACR 20结果时每位患者的成本比初治RA患者使用MTX治疗高1.3倍（1500美元/ACR 20），达到ACR 70WR时高2.1倍（3100美元/ACR 70WR）。最有效的选择，依那西普和MTX联合使用，达到ACR 20结果时每位患者的成本比初治RA患者使用MTX治疗高38倍（4600美元/ACR 20），达到ACR 70WR时高23倍（34800美元/ACR 70WR）。总体而言，广泛的敏感性分析结果并未对这些结果产生实质性影响。
我们的分析表明，如果15毫克/周的MTX在6个月内对初治RA患者达到ACR 20或ACR 70WR具有成本效益，那么三联疗法对MTX抵抗性RA患者很可能也是如此。依那西普+MTX是否具有成本效益取决于6个月内34800美元/ACR 70WR（或42600美元/ACR 20）是否被认为是可接受的。
