Division of Pediatric Urology, Texas Children's Hospital, Houston, TX, USA.
Institute for Pediatric Urology, New York Presbyterian Hospital, Weill Cornell Medical College, New York, NY, USA.
J Pediatr Urol. 2022 Jun;18(3):314-319. doi: 10.1016/j.jpurol.2022.01.021. Epub 2022 Feb 7.
Treatment options for refractory neurogenic detrusor overactivity (NDO) in children include botulinum toxin type A (BTX-A) and augmentation cystoplasty (AC). Although BTX-A is accepted in contemporary pediatric urologic practice, cost and long-term outcomes data for BTX-A are limited relative to the gold standard, AC. The purpose of this study was to compare the projected 10-year costs of AC versus BTX-A.
We performed a cost analysis from the payer perspective by computationally modeling treatment sequences by a Markov model. In the model, we used probabilities derived from published sources, and costs obtained at a tertiary medical center. The base case was a pediatric patient with refractory NDO. In the model, we assumed biannual BTX-A treatments. Treatment costs over 10 years were compared between immediate AC versus bridging therapy with BTX-A. Using the computational model, we simulated 100,000 instances of 10-year treatment cost for each of the two treatment modalities. The costs for the two treatment approaches were then compared using t-test and Wilcoxon test.
The projected median and mean 10-year cost of immediately AC were $51,798.72 (95% CI [$51,798.72, $327,483.80]) and $123,473.4 (SD: $98,085.23) respectfully, while the projected median and mean 10-year cost of bridging therapy with BTX-A prior to proceeding to AC as needed were $74,552.46 (95% CI [$53,188.56, $309,913.07]) and $124,858.80 (SD: $84,495.35) (p < 0.001).
For a typical index pediatric patient with NDO, bridging therapy with intravesical BTX-A is associated with an increased cost compared to immediate AC over a ten-year period.
治疗儿童难治性神经源性逼尿肌过度活动(NDO)的选择包括肉毒杆菌毒素 A 型(BTX-A)和膀胱扩大术(AC)。虽然 BTX-A 在当代小儿泌尿科实践中被接受,但相对于金标准 AC,BTX-A 的成本和长期结果数据有限。本研究的目的是比较 AC 与 BTX-A 的 10 年预计成本。
我们从支付者的角度通过马尔可夫模型进行成本分析,对治疗方案进行计算机建模。在模型中,我们使用来自已发表来源的概率和在三级医疗中心获得的成本。基础病例是一名患有难治性 NDO 的儿科患者。在模型中,我们假设每两年进行一次 BTX-A 治疗。在这两种治疗方法之间比较了 10 年治疗费用。使用计算模型,我们对每一种治疗方法的 10 年治疗成本进行了 100,000 次模拟。然后使用 t 检验和 Wilcoxon 检验比较两种治疗方法的成本。
立即行 AC 的预计中位数和平均 10 年费用分别为 51798.72 美元(95%CI[51798.72 美元,327483.80 美元])和 123473.4 美元(SD:98085.23 美元),而需要时行 BTX-A 桥接治疗后再行 AC 的预计中位数和平均 10 年费用分别为 74552.46 美元(95%CI[53188.56 美元,309913.07 美元])和 124858.80 美元(SD:84495.35 美元)(p<0.001)。
对于典型的 NDO 索引儿科患者,与即刻 AC 相比,在 10 年内,腔内 BTX-A 桥接治疗与 AC 相比会增加成本。