最近開發(fā)了一種方法治療多發(fā)性夜間血紅蛋白尿(PNH)，這是一種罕見的致殘和危及生命的血液疾病，特征為慢性紅細(xì)胞破壞或者溶血。

溶血會導(dǎo)致PHN患者出現(xiàn)下列1種或者多種癥狀:嚴(yán)重貧血，嫉妒乏力，周期性疼痛，呼吸困難，肺源性高血壓，間歇性黑尿(血紅蛋白尿)，腎臟疾病，生

命質(zhì)量下降，血液凝集(血栓)。PNH經(jīng)常危及患者生命，據(jù)估計(jì)診斷后PNH患者中位數(shù)生命為10～15年。

Alexion制藥公司(Cheshire, CN, USA)獲得了美國食品藥品管理局 (FDA)批準(zhǔn)使用Soliris (eculizumab)治療PNH患者以降低溶血。PNH患者缺乏一種特異性蛋白，

該蛋白通常保護(hù)紅細(xì)胞免于補(bǔ)體免疫系統(tǒng)的破壞。Soliris是一種補(bǔ)體抑制劑，能夠選擇性阻斷補(bǔ)體。

Safe Effective Therapy Treats Paroxysmal Nocturnal Hemoglobinuria
 
A therapy has been developed for paroxysmal nocturnal hemoglobinuria (PNH), a rare, disabling and life-threatening blood disorder defined by chronic red blood cell

destruction, or hemolysis.

Hemolysis can cause one or more of the following symptoms in patients with PNH: severe anemia, disabling fatigue, recurrent pain, shortness of breath, pulmonary

hypertension, intermittent episodes of dark colored urine (hemoglobinuria), kidney disease, impaired quality of life, and blood clots (thromboses). PNH often strikes people in

the prime of their lives, the estimated median survival for PNH patients is between 10 and 15 years from the time of diagnosis.

Alexion Pharmaceuticals, Inc., (Cheshire, CN, USA) has received marketing approval from the U.S. Food and Drug Administration (FDA) for Soliris (eculizumab) for treatment

of patients with PNH to reduce hemolysis. Patients with PNH are missing a specific protein that normally protects red blood cells from destruction by a component of the

immune system called terminal complement. Soliris, a complement inhibitor prevents hemolysis by selectively blocking terminal complement.