Paroxysmal nocturnal hemoglobinuria treated with eculizumab in Wakayama, Japan: a retrospective analysis
Paroxysmal nocturnal hemoglobinuria treated with eculizumab in Wakayama, Japan: a retrospective analysis
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日本和歌山市用依库丽单抗治疗阵发性睡眠性血红蛋白尿症的回顾性分析
DOI：
10.11406/rinketsu.61.605
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发表时间：
2020
期刊：
影响因子：
--
通讯作者：
西川 彰則
中科院分区：
文献类型：
--
作者：
田村 志宣;花岡 伸佳;園木 孝志;古家 美昭;堀 善和;弘井 孝幸;山下 友佑;大岩 健洋;村田 祥吾;蒸野 寿紀;西川 彰則
目前，人源化的抗C5单抗eculizumab被广泛用于治疗阵发性睡眠性血红蛋白尿(PNH)，因为它能抑制血管内溶血，从而提高生活质量。然而，在某些情况下，这种治疗是难治的，或者与脑膜炎双球菌脑膜炎有关。还没有发表针对地区的分析，目前关于地区特异性和遗传因素的信息有限。我们在此介绍了一项在日本和歌山市的医院接受eculizumab治疗的8名PNH患者的回顾性研究结果。这些患者的中位年龄为77岁(范围23-88岁)。6名患者有再生障碍性贫血的并发症，4名患者有血栓病史，2名患者有溶血性发作。治疗前血清乳酸脱氢酶中位数为1192IU/L(范围755~1525IU/L)。5例患者在开始治疗后1个月内血清LDH水平恢复正常，PNH相关症状消失。在三名对eculizumab无效的患者中发现了C5基因突变。
Currently, the humanized anti-C5 monoclonal antibody, eculizumab, is widely used for treating paroxysmal nocturnal hemoglobinuria (PNH) due to its effects on suppression of intravascular hemolysis and resulting improvement in quality of life. However, in some cases, this treatment is refractory or is associated with meningococcal meningitis. No region-specific analyses have been published, and currently, information on region specificity and genetic factors is limited. We present here the results of a retrospective study involving eight patients with PNH who were treated with eculizumab in our hospital in Wakayama, Japan. The median age of these patients was 77 (range 23-88) years. Six patients had a complication of aplastic anemia, four patients had a history of thrombosis, and two experienced hemolytic episodes. Before initiating eculizumab treatment, the median serum LDH level was 1,192 IU/l (range 755-1,525 IU/l). Serum LDH levels normalized in five patients within a month of initiating therapy and PNH-related symptoms disappeared. C5 gene mutations were identified in the three patients who did not respond to eculizumab.