Research Institute of Pharmaceutical Science and Technology (RIPST), Ajou University, Suwon, South Korea; Department of Pediatrics, Gangnam Severance Hospital, Yonsei University College of Medicine, Seoul, South Korea.
Department of Pediatrics, Chonnam National University Hwasun Hospital, Chonnam National University Medical School, Hwasun, South Korea.
Thromb Res. 2024 May;237:196-202. doi: 10.1016/j.thromres.2024.03.029. Epub 2024 Apr 13.
The most notable challenge facing hemophilia A treatment is the development of inhibitors against factor VIII, resulting in increased clinical and socioeconomic burdens due to the need for expensive bypassing agents (BPAs). Although immune tolerance induction (ITI) is currently the primary approach for inhibiting and reducing the inhibitors, the lengthy duration of ITI necessitates the continued use of BPA to manage bleeding episodes. In this study, we aimed to obtain real-world evidence on the clinical and economic aspects and associated burdens experienced by patients with hemophilia A with inhibitors undergoing ITI in Korea.
Claims data from January 1, 2007, to December 31, 2020, were used in this study. The study cohort comprised patients with hemophilia A undergoing ITI, who were categorized into three groups: successful, failed, or continuation of ITI. We evaluated clinical and economic burdens, including monthly healthcare visits, medication costs, and total medical expenses.
The study involved 33 cases of ITI across 32 patients. Excluding seven continuation cases where success could not be determined at the observation point, the estimated success rate of ITI was 80.8 %. The median duration of ITI for all patients was 25.7 months. While no significant disparities were noted in the ITI duration between successful and unsuccessful cases (24.51 vs. 25.66 months), substantial discrepancies were observed in the duration of BPA usage (11.10 vs. 25.66 months) and the number of prescribed BPAs (1.79 vs. 2.97).
Successful ITI reduced both clinical and economic burdens, resulting in decreased monthly medication expenses and overall medical costs.
血友病 A 治疗面临的最显著挑战是针对因子 VIII 产生抑制剂，导致由于需要昂贵的旁路制剂（BPA）而增加了临床和社会经济负担。尽管免疫耐受诱导（ITI）是目前抑制和减少抑制剂的主要方法，但 ITI 的漫长持续时间需要继续使用 BPA 来管理出血发作。在这项研究中，我们旨在获得韩国接受 ITI 的血友病 A 伴抑制剂患者的临床和经济方面以及相关负担的真实世界证据。
本研究使用了 2007 年 1 月 1 日至 2020 年 12 月 31 日的索赔数据。研究队列包括接受 ITI 的血友病 A 患者，他们分为三组：成功、失败或继续 ITI。我们评估了包括每月医疗就诊、药物费用和总医疗费用在内的临床和经济负担。
这项研究涉及 32 名患者中的 33 例 ITI。排除观察点无法确定成功的七个继续病例，ITI 的估计成功率为 80.8%。所有患者的 ITI 持续时间中位数为 25.7 个月。虽然成功和不成功病例的 ITI 持续时间没有显著差异（24.51 与 25.66 个月），但 BPA 使用持续时间（11.10 与 25.66 个月）和规定 BPA 的数量（1.79 与 2.97）存在显著差异。
成功的 ITI 减轻了临床和经济负担，导致每月药物费用和总医疗费用降低。
