Eculizumab is effective in managing patients with paroxysmal nocturnal hemoglobinuria (PNH). In South Korea, the financial support for eculizumab therapy is extended by the National Health Insurance Services (NHIS) only to patients with high-risk PNH for approximately 10 years. In this study, we performed a nationwide analysis of the real-world efficacy of eculizumab therapy in patients diagnosed with PNH between January 1, 2002, and December 31, 2016, by using the NHIS database. Patients treated with eculizumab (the eculizumab-treated group) exhibited a significantly higher survival rate than patients not treated with eculizumab (the eculizumab-untreated group), with 4-year survival rates after propensity score matching of 98.31% and 79.67%, respectively (p = 0.0489). The mean red blood cell (RBC) transfusion units per 12 months after eculizumab therapy were significantly lower than that before eculizumab therapy (5.75 units vs. 12.28 units, p < 0.0001). The median time for the first transfusion in the eculizumab-treated group was significantly longer than that in the eculizumab-untreated group. The 4-year transfusion-independence rate for the eculizumab-treated group was significantly higher than that for the eculizumab-untreated group (20.81% vs. 10.24%, p = 0.078). There was no significant difference between the two groups in the incidence of new documented complications related to PNH. In conclusion, eculizumab therapy for patients with high-risk PNH may effectively improve the survival rate and reduce the transfusion requirement. Paradoxically, eculizumab-treated patients with severe PNH exhibit a higher survival rate than eculizumab-untreated patients with less severe PNH.

依库珠单抗可有效治疗阵发性夜间血红蛋白尿 (PNH) 患者。在韩国，国民健康保险服务 (NHIS) 仅向高危 PNH 患者提供对依库珠单抗治疗的财政支持，时间约为 10 年。在这项研究中，我们使用 NHIS 数据库对依库珠单抗治疗在 2002 年 1 月 1 日至 2016 年 12 月 31 日期间诊断为 PNH 的患者的真实世界疗效进行了全国性分析。接受依库珠单抗治疗的患者（依库珠单抗治疗组）的生存率显着高于未接受依库珠单抗治疗的患者（依库珠单抗未治疗组），倾向评分匹配后的 4 年生存率分别为 98.31% 和 79.67%。磷 = 0.0489）。依库珠单抗治疗后每 12 个月的平均红细胞 (RBC) 输血单位显着低于依库珠单抗治疗前（5.75 单位与 12.28 单位，p  < 0.0001）。依库珠单抗治疗组首次输血的中位时间显着长于依库珠单抗未治疗组。依库珠单抗治疗组的 4 年输血独立性显着高于依库珠单抗未治疗组（20.81% vs. 10.24%，p = 0.078）。在与 PNH 相关的新记录并发症发生率方面，两组之间没有显着差异。综上所述，依库珠单抗治疗高危PNH患者可有效提高生存率，减少输血需求。矛盾的是，依库珠单抗治疗的重度 PNH 患者比依库珠单抗未治疗的重度 PNH 患者表现出更高的生存率。