Application of a scoring system in Japanese patients diagnosed with atypical hemolytic uremic syndrome to assess the relationship between the score and clinical responses to eculizumab.
Application of a scoring system in Japanese patients diagnosed with atypical hemolytic uremic syndrome to assess the relationship between the score and clinical responses to eculizumab.
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DOI：
10.1186/s12959-023-00489-0
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发表时间：
2023-04-18
影响因子：
3.1
通讯作者：
Matsumoto, Masanori
中科院分区：
文献类型：
--
作者：
Wada, Hideo;Teranishi, Hirofumi;Shimono, Akihiko;Kato, Noritoshi;Maruyama, Shoichi;Matsumoto, Masanori
关键词：
非典型溶血性尿毒综合征（阿胡斯）是由补体失调引起的，并且通常通过排除血栓性微血管病（TMA）的其他病症来诊断。依库珠单抗是一种末端补体抑制剂，自2013年以来已在日本被批准用于阿胡斯治疗。最近，发表了一个评分系统来支持阿胡斯的诊断。在本文中，我们修改了该评分系统以将其应用于诊断患有阿胡斯并用依库珠单抗治疗的患者，并评估了评分与对依库珠单抗的临床应答之间的关联。本分析纳入了188例临床诊断为阿胡斯、接受依库珠单抗治疗并入组上市后监测（PMS）的日本患者。原始评分系统中的一些参数替换为PMS中收集的临床相似参数，以修改该系统，以下称为TMA/阿胡斯评分，范围为-15至20分。还评估了依库珠单抗开始后90天内的治疗反应，并探索了治疗反应与TMA发作时计算的TMA/阿胡斯评分之间的关系。中位（范围）TMA/阿胡斯评分为10（3-16）。受试者工作特征曲线分析显示，预测依库珠单抗治疗反应的TMA/阿胡斯评分的截止值估计为10，阴性预测值表明≥ 5分适合考虑评估依库珠单抗治疗反应; 185例（98%）患者≥ 5分，3例（2%）患者< 5分。在≥ 5分的患者中，96.1%的患者部分缓解，31.1%的患者完全缓解。< 5分的3例患者中有1例符合部分缓解标准。在存活者和非存活者之间没有观察到TMA/阿胡斯评分的显著差异，表明该评分不适合预测结果（即，存活/死亡）。几乎所有临床诊断为阿胡斯的患者评分≥ 5分，并对依库珠单抗有反应。TMA/阿胡斯评分系统可以成为阿胡斯临床诊断和C5抑制剂治疗应答概率的支持工具。本研究按照药物PMS良好实践指南（MHLW 2004年第171号部级法令）进行。在线版本包含补充材料，可通过10.1186/s12959-023-00489-0获得。
Atypical hemolytic uremic syndrome (aHUS) is caused by complement dysregulation and is generally diagnosed by exclusion from other disorders of thrombotic microangiopathy (TMA). Eculizumab, a terminal complement inhibitor, has been approved for aHUS treatment since 2013 in Japan. Recently, a scoring system was published to support diagnosis of aHUS. Herein we modified this scoring system to apply it to patients diagnosed with aHUS and treated with eculizumab, and assessed the association between the score and clinical responses to eculizumab. One hundred eighty-eight Japanese patients who were clinically diagnosed with aHUS, treated with eculizumab, and enrolled in post-marketing surveillance (PMS) were included in this analysis. Some of parameters in the original scoring system were replaced with clinically similar parameters collected in the PMS to modify the system, hereafter referred to as the TMA/aHUS score, which ranges from -15 to 20 points. Treatment responses within 90 days after eculizumab initiation were also assessed, and the relationship between treatment response and TMA/aHUS scores calculated at TMA onset was explored. The median (range) TMA/aHUS score was 10 (3–16). Receiver operating characteristic curve analysis showed that the cutoff value of TMA/aHUS score to predict treatment response to eculizumab was estimated as 10, and negative predictive value indicated that ≥ 5 points was appropriate to consider assessing the treatment response to eculizumab; 185 (98%) patients had ≥ 5 points and 3 (2%) had < 5 points. Among the patients with ≥ 5 points, 96.1% showed partial response and 31.1% showed complete response. One of the three patients with < 5 points met partial response criteria. No significant difference in the TMA/aHUS scores was observed between survivors and non-survivors, suggesting that the score was not appropriate to predict the outcome (i.e., survival/death) in patients treated with eculizumab. Almost all patients clinically diagnosed with aHUS scored ≥ 5 points and responded to eculizumab. The TMA/aHUS score system could become a supporting tool for the clinical diagnosis of aHUS and probability of response to treatment with a C5 inhibitor. This study was conducted as per good PMS practice guidelines for drugs (MHLW Ministerial Ordinance No. 171 of 2004). The online version contains supplementary material available at 10.1186/s12959-023-00489-0.
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影响因子：
2.3
作者：
Ito S;Hidaka Y;Inoue N;Kaname S;Kato H;Matsumoto M;Miyakawa Y;Mizuno M;Okada H;Shimono A;Matsuda T;Maruyama S;Fujimura Y;Nangaku M;Kagami S
通讯作者：
Kagami S
DOI：
10.1182/asheducation-2018.1.530
发表时间：
2018-11-01
影响因子：
3
作者：
Chiasakul, Thita;Cuker, Adam
通讯作者：
Cuker, Adam
影响因子：
5.7
作者：
Wada, Hideo;Matsumoto, Takeshi;Yamashita, Yoshiki
通讯作者：
Yamashita, Yoshiki
DOI：
10.1182/asheducation-2016.1.217
发表时间：
2016-12-01
影响因子：
3
作者：
Afshar-Kharghan, Vahid
通讯作者：
Afshar-Kharghan, Vahid
影响因子：
7.2
作者：
Glas, AS;Lijmer, JG;Bossuyt, PMM
通讯作者：
Bossuyt, PMM