Soliris（eculizumab，依库珠单抗）
生产商：Alexion 制药
日前，美国FDA正式批准Alexion制药公司的Soliris（又名Eculizumab）用于治疗阵发性睡眠性血红蛋白尿症（PNH），这是目前为止获得FDA批准的唯一一个用于治疗这种罕见血液系统疾病的药物。 阵发性睡眠性血红蛋白尿症是一种以血管内溶血、静脉血栓以及骨髓衰竭为主要表现的后天获得性溶血性疾病，一般多见于成人。患者通常会出现反复的血红蛋白尿，甚至持久的贫血。同时，按照病情严重程度的不同，还会表现出疼痛、体虚乏力、输血需求加大、凝血增多、致死性中风、心脏病以及肠道疾病等。 Soliris是一种特异性针对C5补体的重组人源性单克隆抗体。研究表明：尽管Soliris无法彻底治愈PNH，但是它通过与C5补体结合，阻止补体终末复合物的活化，从而延长了红细胞的寿命，起到治疗PNH的效果。 然而，研究也发现：由于Soliris阻断了机体正常的免疫系统，因此增加了患者对感染（特别是脑膜炎球菌感染）的敏感性。在Soliris的临床实验中，脑膜炎感染是患者表现出的最主要的副作用。为了预防这一副作用，在一项临床实验中，196名PNH患者都在给予Soliris前预先注射了脑膜炎疫苗。 为了解决这一严重的副作用，相关人员已经制定了一项Soliris特殊危险管理计划。这一危险管理计划主要包括：（1）在产品标签以及包装盒上特别警示患者在使用Soliris前应该预先注射脑膜炎球菌疫苗，并在产品标签上列出详尽的患者用药说明；（2）对医生开展有关Soliris使用方面的教育培训。 目前，PNH在美国的患病率非常低，只有百万分之一。因此，Soliris是被FDA作为罕用药物批准的，而依照美国《罕用药物法案》的规定，获得批准生产这一罕用药物的Alexion制药公司将享有长达七年的对Soliris的市场独占权。

IMPORTANT SAFETY INFORMATION

WARNING: SERIOUS MENINGOCOCCAL INFECTION

SOLIRIS® increases the risk of meningococcal infections.

Patients should be vaccinated with a meningococcal vaccine at least 2 weeks prior to receiving the first dose of SOLIRIS. Revaccinate according to current medical guidelines for vaccine use.

Patients should be monitored closely for early signs of meningococcal infections and evaluated immediately if infection is suspected, and treated with antibiotics if necessary.

2007年FDA批准治疗罕见血液病的新药Eculizumab
美国FDA批准治疗罕见血液病阵发性夜间血尿症(PNH)的新药Eculizumab（或叫Soliris）。PNH可导致残疾或早期死亡。这种药物分子中含有的一种活性成份是美国以前未上市的成份。这种药物治疗与输血不同。

PNH是一种成年人疾病。红细胞不正常。一旦这些不正常的红细胞在血流中出现，就可能出现溶血。可导致贫血。根据严重程度不同，患者要忍受不同程度的疼痛，疲劳和虚弱－这就需要对患者频繁输血。Soliris适用于降低溶血率。

Soliris生产企业Alexion。

人源型抗C5单克隆抗体Eculizumab

Eculizumab(h5G1.1)是抑制末端补体成分活化的重组人源型单克隆抗体,其能特异性地键合到人末端补体蛋白C5,通过抑制人补体C5向C5a和C5b的裂解以阻断炎症因子C5a的释放及C5b-9的形成.临床前研究表明该抗体对C5有高度亲和力,能阻断C5a和C5b-9的形成,并保护哺乳动物细胞不受C5b-9介导的损伤。
About SOLIRIS
SOLIRIS® (eculizumab) is the first and only medication that is FDA approved specifically to treat PNH.
SOLIRIS treats the underlying cause of the signs and symptoms of PNH (chronic hemolysis). In clinical trials, patients who were treated with SOLIRIS experienced the following benefits:

Significant reduction in red blood cell destruction (chronic hemolysis). This reduction in hemolysis began in the first week and was sustained over the long term
Significant improvements in anemia, including the complete elimination of the need for transfusions or a significant reduction in the number of transfusions required
Marked improvements in fatigue
Significant impact on overall quality of life
Fewer blood clots with SOLIRIS treatment as compared to the same period of time prior to treatment
 
The effect of withdrawal of anticoagulant therapy during SOLIRIS treatment has not been established. Therefore, treatment with SOLIRIS should not alter anticoagulant management.

Because SOLIRIS blocks part of your immune system, you may be at an increased risk for certain infections during SOLIRIS treatment. You will need to have all recommended immunizations and vaccines before you start treatment with SOLIRIS.
SOLIRIS is generally well tolerated. The most common side effects are headache, runny nose, back pain, and nausea.

Taking SOLIRIS
SOLIRIS® (eculizumab) is given as an IV infusion at the doctor's office or a special infusion center. The infusion usually lasts about 35 minutes.
You will receive your SOLIRIS infusion once a week for the first 4 weeks (600-mg induction dose). Beginning on the 5th week, you will receive a slightly higher dosage of SOLIRIS every two weeks (900-mg maintenance dose).

For best results

It's important not to miss any treatments. SOLIRIS only controls hemolysis as long as you take it
Make sure your doctor knows about any other drugs you are taking
Talk with your doctor before you take any vitamins, over-the-counter drugs, or natural supplements, as these may affect your therapy as well
To find out how to begin SOLIRIS therapy and how the OneSource™ Program will guide you each step of the way, please contact an Alexion Case Manager.

OneSource is a team of registered nurses at Alexion dedicated to assisting you with your questions about SOLIRIS treatment. This team is available to help facilitate medical insurance coverage and funding options.

Eculizumab可以安全、有效地用于ＰＮＨ病人

2月5日《新英格兰医学杂志》（N Engl J Med 2004;350:537-538,552-559）上发表的一项报告表明，Eculizumab――一种阻断终末补体成份激活的抗体，可以安全地治疗阵发性睡眠性血红蛋白尿症（PNH，引起溶血，因此需要输血）。

PNH起因于一种基因突变，以致血细胞对补体系统的破坏更为敏感，引起溶血和血红蛋白尿，通常在夜晚加重，因为此时血液PH的变化让补体更加活跃。英国利兹教学医院的希尔曼（Peter Hillmen）和同事检测了11位病人用eculizumab治疗（每周输液600mg，连续4周，然后每半周输900mg，超过8周）的效果。

治疗减少了溶血，表现为乳酸脱氢酶下降（p = 0.002），PNH型红细胞的百分比降低（p = 0.005）。在开始eculizumab治疗前，每位病人平均每月需要输2.1单位的血，治疗后显著下降到只需0.6单位。其它有显著意义的临床益处包括：血红蛋白尿的发作下降96%，生活品质改善。尽管试验期间所有病人至少都有一次不良事件发生，但都不是eculizumab引起的。

“这些结果不仅证实PNH病人的溶血性贫血与补体有关，而且也指出本病可能还有其它治疗方法”，本杂志的副总编斯奇沃斯（Robert S. Schwartz）在相关评论中说，但本研究还没回答某些问题，如eculizumab应用多长时间，以及本药是否可改善骨髓功能。