目标:最近依库珠单抗,C5单克隆抗体可改善非典型溶血性尿毒综合征(aHUS)的病程,并被国际共识指南推荐为一线治疗.然而,在aHUS急性期使用依库珠单抗的几个实际问题尚未解决.
方法:纳入2010年3月至2015年12月在东京都儿童医学中心接受依库珠单抗诊断为aHUS的儿童。在排除志贺毒素诱导大肠杆菌(STEC)相关HUS和血小板减少性紫癜后,根据溶血性尿毒症综合征(HUS)标准诊断aHUS。我们从我们机构的电子病历中检索并分析了数据。
结果:我们回顾了4例疑似aHUS患者。Eculizumab在一名后来诊断为STEC-HUS的患者中停用。其余3例患者继续治疗,无复发。实际问题包括难以诊断aHUS,特别是在急性期,被包裹的生物体感染的风险,尤其是脑膜炎奈瑟菌,和输液反应。除了与急性期有关的问题,必须考虑在慢性期稳定患者中停用依库珠单抗.
结论:Eculizumab,aHUS患儿的一线治疗,通常是有效的。然而,与使用相关的某些问题需要谨慎行事。由于关于依库珠单抗的临床信息仍然非常有限,其长期使用的理由尚未确定,在使用依库珠单抗管理aHUS时,建议医生进行护理.
OBJECTIVE: Recently eculizumab, a monoclonal antibody to C5, was found to improve the disease course of atypical haemolytic uraemic syndrome (aHUS) and has been recommended as the first line treatment by an international consensus guideline. However, several practical issues in the use of eculizumab for the acute phase of aHUS have yet to be resolved.
METHODS: Children who received eculizumab with diagnosis of aHUS between March 2010 and December 2015 at
Tokyo Metropolitan Children\'s Medical Center were enrolled. aHUS was diagnosed according to the haemolytic uraemic syndrome (HUS) criteria after excluding Shiga toxin-inducing Escherichia coli (STEC) -associated HUS and thrombocytopaenic purpura. We retrieved and analyzed data from the electronic medical records at our institution.
RESULTS: We reviewed four patients with suspected aHUS. Eculizumab was discontinued in one patient in whom STEC-HUS was later diagnosed. Treatment was continued in the remaining three patients without recurrence. Practical issues included difficulty in diagnosing aHUS, particularly in the acute phase, risk of infection by encapsulated organisms, especially Neisseria meningitis, and infusion reaction. In addition to issues relating to the acute phase, discontinuing eculizumab in stable patients in the chronic phase must be considered.
CONCLUSIONS: Eculizumab, the first line treatment for children with aHUS, is usually effective. However, certain problems associated with its use require caution to be exercised. As clinical information on eculizumab are still very limited, and the rationale for its long-term use has yet to be established, physicians are advised to exercise care when using eculizumab to manage aHUS.