Batt Katharine, Schultz Bob G, Caicedo Jorge, Hollenbeak Christopher S, Agrawal Neha, Chatterjee Sagnik, Bullano Michael
Department of Hematology and Medical Oncology, Wake Forest Baptist School of Medicine, Winston-Salem, NC, USA.
Outcomes Research, Takeda Pharmaceuticals U.S.A., Inc., Lexington, MA, USA.
Curr Med Res Opin. 2022 Oct;38(10):1685-1693. doi: 10.1080/03007995.2022.2105072. Epub 2022 Aug 3.
Factor VIII (FVIII) replacement and emicizumab have demonstrated efficacy for prevention of bleeds among patients with hemophilia A (PwHA) compared to on-demand (OD) use. Evidence investigating clinical outcomes and healthcare costs of non-inhibitor PwHA switching from prophylaxis with FVIII concentrates to emicizumab has not been well-established within large real-world datasets. This study aimed to investigate billed annualized bleed rates (ABR) and total cost of care (TCC) among non-inhibitor PwHA switching from FVIII-prophylaxis to emicizumab-prophylaxis.
This retrospective, observational study was conducted using IQVIA PharMetrics Plus, a US administrative claims database. The date of first claim for emicizumab was defined as the index date. OD patients and inhibitor patients were excluded. Bleeds were identified using a list of 535 diagnosis codes. Bayesian models were developed to estimate the probability ABR worsens and TCC increases after switching to emicizumab. Wilcoxon rank-sum tests were used to test statistical significance of changes in ABR and TCC after switch.
Among the 121 identified patients, the difference in mean ABR between FVIII-prophylaxis (0.68 [SD = 1.28]) and emicizumab (0.55 [SD = 1.48]) was insignificant ( = .142). The mean annual TCC significantly increased for patients switching from FVIII-prophylaxis ($518,151 [SD = $289,934]) to emicizumab ($652,679 [SD = $340,126]; < .0001). The Bayesian models estimated a 21.0% probability of the ABR worsening and a 99.9% probability of increasing TCC after switch.
This study found that in male non-inhibitor PwHA, switching from FVIII prophylaxis to emicizumab incurs substantial cost increase with no significant benefit in ABR. This evidence may help guide providers, payers, and patients in shared decision-making conversations around best treatment options.
与按需使用相比，因子VIII（FVIII）替代疗法和艾美赛珠单抗已证明对预防甲型血友病患者（PwHA）出血有效。在大型真实世界数据集中，尚未充分确立关于非抑制性PwHA从FVIII浓缩物预防转换为艾美赛珠单抗预防的临床结局和医疗成本的证据。本研究旨在调查从FVIII预防转换为艾美赛珠单抗预防的非抑制性PwHA的年度计费出血率（ABR）和总护理成本（TCC）。
本回顾性观察性研究使用美国管理索赔数据库IQVIA PharMetrics Plus进行。艾美赛珠单抗首次索赔日期定义为索引日期。排除按需治疗患者和抑制物患者。使用535个诊断代码列表识别出血情况。开发贝叶斯模型以估计转换为艾美赛珠单抗后ABR恶化和TCC增加的概率。使用Wilcoxon秩和检验来检验转换后ABR和TCC变化的统计学显著性。
在121名确定的患者中，FVIII预防组（0.68 [标准差 = 1.28]）和艾美赛珠单抗组（0.55 [标准差 = 1.48]）的平均ABR差异不显著（P = 0. .142）。从FVIII预防组（518,151美元[标准差 = 289,934美元]）转换为艾美赛珠单抗组（652,679美元[标准差 = 340,126美元]）的患者平均年度TCC显著增加（P < 0.0001）。贝叶斯模型估计转换后ABR恶化的概率为(21.0%)，TCC增加的概率为(99.9%)。
本研究发现，在男性非抑制性PwHA中，从FVIII预防转换为艾美赛珠单抗会导致成本大幅增加，而ABR无显著益处。这一证据可能有助于指导医疗服务提供者、支付方和患者围绕最佳治疗方案进行共同决策对话。
