PDF(Pubmed)
Abstract：
Paroxysmal nocturnal haemoglobinuria (PNH) is a rare acquired haematopoietic stem cell disease characterized by complement-mediated intravascular hemolysis, thrombosis, and bone marrow failure. Eculizumab and ravulizumab are anti-C5 monoclonal antibodies that reduce hemolysis, anaemia and thrombotic risk, but are associated with increased risk of infection with encapsulated bacteria, including Neisseria meningitidis. We report a case of life-threatening infection by non-groupable Neisseria meningitidis in a young PNH patient treated with ravulizumab. Despite prompt admission to the intensive care unit, microbe isolation was delayed due to the negativity of capsular antigens, and the patient required intubation, dialysis, and transfusion support for pancytopenia. Notably, PNH disease activity remained controlled and no additional anti-C5 doses were administered. Increasing awareness regarding septic risk in PNH patients on complement inhibitors despite vaccinations is pivotal. A warning about serotypes generally not pathogenetic and not covered by vaccination, such as non-capsulated forms, is emerging.
摘要：
阵发性睡眠性血红蛋白尿症（PNH）是一种罕见的获得性造血干细胞疾病，以补体介导的血管内溶血为特征，血栓形成,骨髓衰竭.Eculizumab和ravulizumab是抗C5单克隆抗体，可减少溶血，贫血和血栓形成的风险，但与被包裹的细菌感染的风险增加有关，包括脑膜炎奈瑟菌.我们报告了一名接受ravulizumab治疗的年轻PNH患者中不可分组的脑膜炎奈瑟菌感染危及生命的病例。尽管迅速进入重症监护室，由于荚膜抗原的阴性，微生物分离被推迟，病人需要插管,透析,和全血细胞减少症的输血支持。值得注意的是,PNH疾病活动保持受控,并且不施用额外的抗C5剂量。尽管接种了疫苗，但提高对补体抑制剂的PNH患者败血症风险的认识至关重要。关于血清型通常不具有致病性且不在疫苗接种范围内的警告，如非胶囊形式，正在出现。
