Department of Surgery, University of Virginia School of Medicine, Charlottesville
Department of Gastroenterology, University of Virginia School of Medicine, Charlottesville
JAMA Surg. 2018 Mar 1;153(3):225-232. doi: 10.1001/jamasurg.2017.4587.
The 2012 international consensus guidelines defined a subcategory of pancreatic branch duct intraductal papillary mucinous neoplasms with “worrisome features,” which may be followed with close surveillance. However, given the poor prognosis of invasive malignancy, the role of early, upfront resection requires further investigation.
To compare the utility of upfront resection vs long-term surveillance. We hypothesized that surveillance of these cystic neoplasms would offer greater long-term utility.
DESIGN, SETTING, AND PARTICIPANTS: A Markov decision analysis model was constructed to estimate and compare 2 management strategies: early resection and long-term surveillance. Estimates for the utility of outcomes, probabilities of transitions between disease states, and probabilities of surgical morbidity were derived from a literature review of articles published between 1997 and 2014. The comparative effectiveness model’s variable estimates were based on data published predominantly by high-volume, tertiary referral centers. Model probability variable estimates were derived from large, retrospective, single-institution reports. For utility variables, estimates derived from studies using standard-gamble or time-tradeoff methods were given greater weight.
Expected utility was measured in terms of quality-adjusted life years using 3% annual discount. Probabilistic and 1-way sensitivity analyses were performed to assess the potential effects of uncertainty in estimates of key model variables.
Early resection yielded 11.63 quality-adjusted life-years during 20 years of follow-up compared with 11.06 for surveillance. Probabilistic sensitivity analysis indicated that resection has a 94% likelihood of being more effective than surveillance. Early resection obtained greater utility only if each of the following criteria are met: life expectancy is at least 18 years, surgical mortality is less than 4.3%, and baseline preoperative utility is at least 0.78. Additional drivers of the model outcomes include the rate of progression from worrisome to high-risk features and the likelihood of finding cancer on resection for neoplasms with high-risk stigmata.
Early resection compares favorably with surveillance in the management of branch duct intraductal papillary mucinous neoplasms with worrisome features. However, careful consideration of patient factors and surgeon outcomes is imperative.
2012 年国际共识指南定义了胰腺分支胰管内乳头状黏液性肿瘤的一个亚类，具有“令人担忧的特征”，可能需要密切监测。然而，鉴于侵袭性恶性肿瘤的预后较差，早期进行手术切除的作用需要进一步研究。
比较早期切除与长期监测的效果。我们假设这些囊性肿瘤的监测将提供更大的长期效果。
设计、设置和参与者：构建了一个马尔可夫决策分析模型，以估计和比较两种管理策略：早期切除和长期监测。结果的效用、疾病状态之间转移的概率和手术发病率的概率是从 1997 年至 2014 年发表的文章的文献综述中得出的。比较有效性模型的变量估计基于主要来自高容量、三级转诊中心的数据。模型概率变量估计来自大型回顾性单一机构报告。对于效用变量，使用标准博弈或时间权衡方法得出的估计值被赋予更大的权重。
使用 3%的年度贴现率，以质量调整生命年来衡量预期效用。进行了概率和单向敏感性分析，以评估模型关键变量估计不确定性的潜在影响。
在 20 年的随访期间，早期切除获得了 11.63 个质量调整生命年，而监测则获得了 11.06 个。概率敏感性分析表明，切除的效果比监测更有效，可能性为 94%。只有满足以下所有条件，早期切除才能获得更大的效果：预期寿命至少为 18 年，手术死亡率小于 4.3%，并且基线术前效用至少为 0.78。模型结果的其他驱动因素包括从令人担忧的特征到高危特征的进展率，以及在具有高危特征的肿瘤中发现癌症的可能性。
早期切除与监测在处理具有令人担忧特征的分支胰管内乳头状黏液性肿瘤方面具有优势。然而，必须仔细考虑患者因素和外科医生的结果。
