Abstract：
This study aims to evaluate the efficacy, safety, and long-term cost-effectiveness of fixed-dose busulfan (Bu) administration and pharmacokinetically (PK) guided adjustment of Bu dose for patients who underwent hematopoietic stem cell transplantation. The efficacy and safety of both dosing strategies were compared using a systematic review and meta-analysis. A Markov model was used in estimating relevant cost and health outcomes from the perspective of the health system. The primary outcomes of interest were lifetime cost, quality adjusted life-years (QALYs) gained, and incremental cost-effectiveness ratio (ICER) in dollar per QALY gained. Results showed that progression-free survival and overall survival in the PK-guided group were higher than that in the fixed-dose group, and the PK-guided group was associated with low non-relapse mortality and relapse rate. In contrast to safety, the incidence of acute graft-versus-host disease (GVHD) was the same in the two groups (P > 0.05). Cost-effectiveness analysis showed that the QALY of the PK-guided group (12.8135 QALYs and $582,475.07) increased by 2.0609 relative to that in the fixed-dose group (10.7526 QALYs and $562,833.20), and the ICER was $9530.72/QALY. One-way and probability sensitivity analyses confirmed the reliability of the results. In conclusion, the PK-guided approach has higher efficacy and is safer.
摘要：
本研究旨在评估疗效，安全,对于接受造血干细胞移植的患者，固定剂量白消安（Bu）给药和药代动力学（PK）指导调整Bu剂量的长期成本效益。使用系统评价和荟萃分析比较了两种给药策略的有效性和安全性。从卫生系统的角度使用马尔可夫模型来估计相关成本和健康结果。利息的主要结果是终生成本，获得的质量调整生命年(QALYs)，和每QALY增量成本效益比(ICER)。结果显示，PK引导组的无进展生存期和总生存期均高于固定剂量组，PK指导组的非复发死亡率和复发率较低.与安全相比，两组急性移植物抗宿主病(GVHD)发生率相同(P>0.05)。成本-效果分析表明，PK指导组的QALY（12.8135QALYs和$582,475.07）相对于固定剂量组（10.7526QALYs和$562,833.20）增加了2.0609，ICER为9530.72美元/QALY。单向和概率敏感性分析证实了结果的可靠性。总之,PK指导方法疗效更高,安全性更高.
