Risitano Antonio M
Bone Marrow Transplantation Clinical Unit, Hematology, Department of Clinical Medicine and Surgery, Federico II University of Naples, Via Pansini 5, Naples 80131, Italy.
Hematol Oncol Clin North Am. 2015 Jun;29(3):561-82. doi: 10.1016/j.hoc.2015.01.009. Epub 2015 Apr 4.
The availability of anticomplement therapies has been a major achievement for medicine in the last decade. Indeed, eculizumab has changed the treatment paradigm of paroxysmal nocturnal hemoglobinuria and atypical hemolytic uremic syndrome and promises to do the same in several other human complement-mediated diseases. Nowadays, a 10-year experience has also taught us that there are some pitfalls that represent a challenge to improve the current anticomplement treatment. Most of these observations come from paroxysmal nocturnal hemoglobinuria, where unmet clinical needs are emerging, triggering the attention of several investigators and pharmaceutical companies.
在过去十年中，抗补体疗法的出现是医学领域的一项重大成就。事实上，依库珠单抗已经改变了阵发性夜间血红蛋白尿和非典型溶血性尿毒症综合征的治疗模式，并有望在其他几种人类补体介导的疾病中发挥同样的作用。如今，十年的经验也让我们认识到，存在一些陷阱，对改进当前的抗补体治疗构成挑战。这些观察结果大多来自阵发性夜间血红蛋白尿，该疾病中尚未满足的临床需求不断出现，引发了众多研究人员和制药公司的关注。
