Clinical benefit of eculizumab in patients with no transfusion history in the International Paroxysmal Nocturnal Haemoglobinuria Registry
Clinical benefit of eculizumab in patients with no transfusion history in the International Paroxysmal Nocturnal Haemoglobinuria Registry
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DOI：
10.1111/imj.13523
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发表时间：
2017-09-01
影响因子：
2.1
通讯作者：
Rosse, Wendell F.
中科院分区：
文献类型：
--
作者：
Almeida, Antonio M.;Bedrosian, Camille;Rosse, Wendell F.
背景：Eculizumab可减少阵发性睡眠性血红蛋白尿症(PNH)患者的血管内溶血，改善疾病症状。目的：在真实环境中，研究Eculizumab对溶血性PNH(乳糖酶脱氢酶(LDH)和Eulizumab；=1.5正常上限)且无红细胞输血史的患者的疗效。方法：来自国际PNH登记的三个人群：(I)未输血，未经治疗；(Ii)未输血，eculizumab治疗和(Iii)输血，eculizumab治疗(>；)=在Eulizumab启动前6个月内输血1次)。经多元线性回归分析，未输血患者接受尤利珠单抗治疗的LDH(U/L)从基线到6个月的平均绝对值变化与未接受治疗的患者相比。次要结果是从基线到最后一次可用评估的慢性病治疗(FAIT)-疲劳和欧洲癌症研究与治疗组织生活质量问卷(EORTC-QLQ)-C30疲劳评分的平均变化。结果：研究人群包括(I)144名未输血、未治疗的患者；(Ii)45名未输血、接受尤鲁珠单抗治疗的患者；(Iii)105名已输血、接受尤利珠单抗治疗的患者。在这些患者中，分别有136/144、43/45和99/105存在HDA。与未经治疗的患者相比，未输血、经治疗的患者乳酸脱氢酶绝对值下降幅度更大(-1318.8比-39.4；P
Background: Eculizumab reduces intravascular haemolysis and improves disease symptoms in patients with paroxysmal nocturnal haemoglobinuria (PNH).Aims: To characterise, in a real-world setting, the effect of eculizumab in patients with haemolytic PNH (lactase dehydrogenase (LDH) >= 1.5 upper limit of normal) and no history of red blood cell transfusion, including those with high disease activity (HDA).Methods: Three populations from the International PNH Registry were studied: (i) non-transfused, untreated; (ii) non-transfused, eculizumab-treated and (iii) transfused, eculizumab-treated (>= 1 transfusions in 6 months prior to eculizumab initiation). Using multivariate linear regression, the primary outcome was mean absolute change from baseline to 6 months in LDH (U/L) in non-transfused patients who were treated with eculizumab versus those who remained untreated. Secondary outcomes were mean changes in functional assessment of chronic illness therapy (FACIT)-Fatigue and European Organisation for Research and Treatment of Cancer Quality of Life Questionnaire (EORTC-QLQ)-C30 Fatigue scores from baseline to last available assessment.Results: The study population included (i) 144 non-transfused, untreated patients; (ii) 45 non-transfused, eculizumab-treated patients and (iii) 105 transfused, eculizumabtreated patients. Of these, 136/144, 43/45 and 99/105 had HDA respectively. Compared with untreated patients, non-transfused, treated patients had greater absolute reduction in LDH (-1318.8 vs -39.4; P