Department of Pediatric Oncology, Dana-Farber/Children's Hospital Cancer Care, Harvard Medical School, Boston, Massachusetts 02115, USA.
Pediatr Blood Cancer. 2010 Jul 1;54(7):879-84. doi: 10.1002/pbc.22396.
Decision analysis was used to clarify differences in survival and complication rates comparing surgery alone versus surgery plus chemotherapy for Stage I, favorable histology Wilms tumor patients.
A state transition model was used to simulate treatment with nephrectomy-only, nephrectomy with adjuvant vincristine (VCR) or with vincristine plus dactinomcyin (NWTS Regimen EE4A). Rates of relapse and complications of therapy were obtained from the literature. In sensitivity analysis, the model was probed for the value(s) at which the treatment of choice changes.
The overall survival (OS) is essentially the same for patients treated with any of the three strategies (OS(Nephrectomy) = 98.8%; OS(EE4A) = 98.8%; OS(VCR) = 98.6%). Rates of serious long-term complications in the surviving population are also similar across treatment strategies (nephrectomy = 1.4%; VCR = 1.2%; EE4A = 0.3%). Both the progression and salvage rates after nephrectomy-only would have to be much worse than expected for nephrectomy-only to be an unacceptable strategy.
The differences in overall survival and rates of long-term complications between the three different initial strategies were negligible in the model. Based on this analysis, it was decided by the Children's Oncology Group that it was acceptable to continue to include nephrectomy without adjuvant chemotherapy as an experimental arm of the low risk Wilms tumor protocol with stringent eligibility criteria and close follow-up. Decision analysis can have a role in clinical trial design by making the tradeoffs between strategies more explicit. The robustness of these conclusions can be tested by widely varying the underlying assumptions.
通过决策分析，比较了单纯手术与手术加化疗治疗 I 期、组织学有利的 Wilms 肿瘤患者的生存和并发症发生率的差异。
使用状态转移模型模拟单纯肾切除术、肾切除术加辅助长春新碱（VCR）或长春新碱加放线菌素 D（NWTS 方案 EE4A）的治疗。从文献中获得了复发率和治疗并发症的数据。在敏感性分析中，模型探测了治疗选择发生变化的价值。
接受三种治疗策略的患者的总生存率（OS）基本相同（肾切除术 OS = 98.8%；EE4A OS = 98.8%；VCR OS = 98.6%）。在接受治疗的存活人群中，严重长期并发症的发生率在治疗策略之间也相似（肾切除术 = 1.4%；VCR = 1.2%；EE4A = 0.3%）。只有在单纯肾切除术后的进展和挽救率比预期差得多的情况下，单纯肾切除术才是不可接受的策略。
在模型中，三种不同初始策略之间的总生存率和长期并发症发生率的差异可以忽略不计。基于这项分析，儿童肿瘤学组决定，继续将无辅助化疗的肾切除术作为低危 Wilms 肿瘤方案的实验臂是可以接受的，前提是严格的入选标准和密切随访。决策分析可以通过更明确地权衡策略在临床试验设计中发挥作用。通过广泛改变基本假设，可以检验这些结论的稳健性。
