Divisions of Pediatric Hematology and Oncology, University of California, Irvine and CHOC Children's Specialists, Irvine, CA 92868, USA.
Department of Real-World Data Oncology-Hematology, F Hoffmann-La Roche Ltd., Basel, CH-4070, Switzerland.
J Comp Eff Res. 2022 Jul;11(10):717-728. doi: 10.2217/cer-2021-0278. Epub 2022 May 10.
To determine if emicizumab was channeled to clinically complex people with hemophilia A upon approval. Claims data (16 November 2017, through 31 December 2019) from US-based insurance databases were analyzed to compare the clinical complexity of people with hemophilia A initiating emicizumab with matched individuals receiving factor VIII (FVIII) episodically or prophylactically. People with hemophilia A with evidence of previous bypassing agent use (indicating FVIII inhibitors) were excluded. Outcomes included bleeding events, arthropathy, pain, comorbidities and healthcare costs. A larger proportion of emicizumab users had bleeding events, comorbidities and arthropathy and greater healthcare costs in the year prior to starting emicizumab compared with FVIII users. Claims-based data limitations prevent an absolute conclusion. Nevertheless, emicizumab users appear more clinically complex than FVIII users, suggesting post-approval channeling.
为了确定emicizumab 在获得批准后是否被用于治疗具有临床复杂性的血友病 A 患者。 分析了来自美国保险数据库的索赔数据（2017 年 11 月 16 日至 2019 年 12 月 31 日），以比较开始使用 emicizumab 的血友病 A 患者与接受因子 VIII（FVIII）按需或预防性治疗的匹配个体的临床复杂性。 排除了有先前旁路剂使用证据（表明存在 FVIII 抑制剂）的血友病 A 患者。 结果包括出血事件、关节炎、疼痛、合并症和医疗保健费用。 在开始使用 emicizumab 之前的一年中，与 FVIII 使用者相比，emicizumab 使用者有更多的出血事件、合并症和关节炎，并且医疗保健费用更高。 基于索赔的数据限制无法得出绝对结论。 尽管如此，emicizumab 使用者似乎比 FVIII 使用者更具临床复杂性，这表明批准后存在渠道转移。
