Divisions of Pediatric Hospital Medicine and
Clinical Excellence Research Center, Stanford University, Stanford, California.
Hosp Pediatr. 2020 Mar;10(3):257-265. doi: 10.1542/hpeds.2019-0153.
Neuromuscular scoliosis (NMS) can result in severe disability. Nonoperative management minimally slows scoliosis progression, but operative management with posterior spinal fusion (PSF) carries high risks of morbidity and mortality. In this study, we compare health and economic outcomes of PSF to nonoperative management for children with NMS to identify opportunities to improve care.
We performed a cost-effectiveness analysis. Our decision analytic model included patients aged 5 to 20 years with NMS and a Cobb angle ≥50°, with a base case of 15-year-old patients. We estimated costs, life expectancy, quality-adjusted life-years (QALYs), and incremental cost-effectiveness from published literature and conducted sensitivity analyses on all model inputs.
We estimated that PSF resulted in modestly decreased discounted life expectancy (10.8 years) but longer quality-adjusted life expectancy (4.84 QALYs) than nonoperative management (11.2 years; 3.21 QALYs). PSF costs $75 400 per patient. Under base-case assumptions, PSF costs $50 100 per QALY gained. Our findings were sensitive to quality of life (QoL) and life expectancy, with PSF favored if it significantly increased QoL.
In patients with NMS, whether PSF is cost-effective depends strongly on the degree to which QoL improved, with larger improvements when NMS is the primary cause of debility, but limited data on QoL and life expectancy preclude a definitive assessment. Improved patient-centered outcome assessments are essential to understanding the effectiveness of NMS treatment alternatives. Because the degree to which PSF influences QoL substantially impacts health outcomes and varies by patient, clinicians should consider shared decision-making during PSF-related consultations.
神经肌肉性脊柱侧凸（NMS）可导致严重残疾。非手术治疗可最大限度地减缓脊柱侧凸的进展，但后路脊柱融合术（PSF）的手术治疗具有较高的发病率和死亡率。在这项研究中，我们比较了 PSF 与非手术治疗对 NMS 患儿的健康和经济结果，以确定改善治疗的机会。
我们进行了成本效益分析。我们的决策分析模型包括年龄在 5 至 20 岁之间、Cobb 角≥50°的 NMS 患者，并以 15 岁患者为基础病例。我们根据已发表的文献估计了成本、预期寿命、质量调整生命年（QALY）和增量成本效益，并对所有模型输入进行了敏感性分析。
我们估计 PSF 导致患者预期寿命略有缩短（10.8 年），但生活质量调整后预期寿命较长（4.84 QALY），而非手术治疗则为（11.2 年；3.21 QALY）。PSF 每位患者的费用为 75400 美元。在基本假设下，PSF 每获得一个 QALY 的成本为 50100 美元。我们的研究结果对生活质量（QoL）和预期寿命很敏感，如果 PSF 显著提高 QoL，则 PSF 更具优势。
在 NMS 患者中，PSF 是否具有成本效益取决于 QoL 改善的程度，当 NMS 是导致虚弱的主要原因时，改善程度较大，但 QoL 和预期寿命的有限数据使得无法进行明确评估。需要改进以患者为中心的结果评估，以了解 NMS 治疗选择的有效性。由于 PSF 对 QoL 的影响程度对健康结果有很大影响，且因患者而异，因此临床医生应在与 PSF 相关的咨询中考虑共同决策。
