Introduction: Multiple studies have demonstrated the effectiveness of pharmacokinetic (PK)-guided individualized prophylaxis with human coagulation factor VIII (FVIII) compared with standard prophylaxis, but no studies have evaluated the economics of PK-guided prophylaxis in China. Hence, we conducted this study to assess the cost-effectiveness of PK-guided prophylaxis with recombinant FVIII (rFVIII) versus standard prophylaxis in Chinese adult patients with severe hemophilia A. Methods: A discrete event simulation model was developed to simulate 10,000 patients with hemophilia A who received rFVIII treatment over a 1-year time horizon. The standard prophylaxis rFVIII dose was 30 IU/kg by intravenous injection. The PK-guided prophylaxis dosage was adjusted for each patient to maintain FVIII trough level at 1-5 IU/dL. Dosing interval for both approaches was kept fixed at 48 h. The health outcomes included annual joint bleed rate (AJBR) and quality-adjusted life years (QALYs). The model considered the costs of drug. Incremental cost-effectiveness ratio (ICER) was estimated and scenario analysis was performed.Results: A total of 94.3% of patients receiving PK-guided individualized prophylaxis achieved the goal of maintaining the trough concentration at 1-5 IU/dL compared with 62.7% on standard prophylaxis. AJBR and QALYs gained in PK-guided and standard prophylaxis were 1.527 vs 1.601, and 0.8384 vs 0.8383, respectively. Costs of drug prophylaxis and costs of treatment for bleeding events in PK-guided prophylaxis (148,641.47 USD; 4546.43 USD) were lower than those in standard prophylaxis (159,620.93 USD; 4753.39 USD). An average saving of USD 11,186.47 was obtained by the PK-guided approach. The prophylaxis treatment scenarios were the most influential factors. Conclusion: PK-guided individualized prophylaxis appeared to be a dominant treatment compared with standard prophylaxis, with slightly higher QALYs but lower total costs.

Background: There is a myriad of microwave ablation (MWA) systems used in clinical settings worldwide for the management of liver cancer that offer a variety of features and capabilities. However, an analysis on which features and capabilities result in the most favorable efficacy and safety results has never been completed due to a lack of head-to-head comparisons. The aim of this study is to compare single-antenna and multiple-antenna MWA using radiofrequency ablation (RFA) as a common comparator in the treatment of very-early, early hepatocellular carcinoma (HCC) and ≤5 cm liver metastases. Methods: This network meta-analysis was performed according to PRISMA guidelines. PubMed, Cochrane, and Web of Science databases were searched for comparative studies. Complete ablation (CA) rate, local tumor progression-free (LTPF) rate, overall survival (OS), and major complication rate were assessed. Subgroup analyses were further performed based on synchronous or asynchronous MWA generators and tumor size (<2 cm or ≥2 cm). Results: Twenty-one studies (3424 patients), including 3 randomized controlled trials (RCTs) and 18 observational studies, met eligibility criteria. For CA, LTPF and major complications, as compared to single-antenna MWA, multiple-antenna MWA had relative risks (RRs) of 1.051 (95% CI: 0.987–1.138), 1.099 (95% CI: 0.991–1.246), and 0.605 (95% CI: 0.193–1.628), respectively. For 1-year and 3-year OS, as compared to single-antenna MWA, multiple-antenna MWA had odds ratios (ORs) of 0.9803 (95% CI: 0.6772–1.449) and 1.046 (95% CI: 0.615–1.851), respectively. Subgroup analysis found synchronized multi-antenna MWA was associated with significantly better LTPF by 22% (RR: 1.22, 95% CI 1.068, 1.421), and 21.4% (RR: 1.214, 95% CI 1.035, 1.449) compared with single-antenna MWA, and asynchronous multiple-antenna MWA, respectively, with more evident differences in larger tumors (≥2 cm). Conclusion: Multi-antenna and single-antenna MWA showed similar effectiveness for local treatment of liver tumors, but synchronous multi-antenna MWA exhibited better LTPF compared to other MWA approaches, particularly for larger liver tumors (≥2 cm). Large-scale RCTs should be further conducted.