From the Institute of Cellular Medicine, the Newcastle Upon Tyne Hospitals NHS Foundation Trust, UK
the Institute of Genetic Medicine Newcastle University and the Newcastle Upon Tyne Hospitals NHS Foundation Trust, UK
QJM. 2016 Jan;109(1):27-33. doi: 10.1093/qjmed/hcv082. Epub 2015 Apr 21.
In 2013 NHS England commissioned the use of eculizumab for both new patients with atypical haemolytic uraemic syndrome (aHUS) and those undergoing transplantation. This national service is delivered locally but coordinated by an expert centre at the Newcastle upon Tyne Hospitals NHS Foundation Trust.
In the first year of service, 43 aHUS patients received eculizumab, 15 children and 28 adults. Twenty-three were new patients and 20 prevalent. Fifteen of the 23 new patients required dialysis before eculizumab was started, 8 of these recovered renal function. Twelve of the 20 prevalent patients who received eculizumab were transplant patients, 8 with prophylactic use and 4 for recurrent disease; the outcome in all was good. Eculizumab was withdrawn in 14 patients, 5 were patients who had not recovered renal function. In 3 of the 14 patients, it was necessary to reintroduce eculizumab because of recurrent disease (2 extra-renal and 1 renal). There were 2 deaths in the 43 patients, and neither was associated with use of eculizumab. There were no episodes of meningococcal disease.
The establishment of this national service has enabled aHUS patients in England to receive eculizumab when they need it for as long as they need it.
2013 年，英国国民保健署（NHS England）委托使用依库珠单抗治疗新的非典型溶血性尿毒症综合征（aHUS）患者和接受移植的患者。这项国家服务在当地提供，但由泰恩河畔纽卡斯尔医院 NHS 基金会信托的一个专家中心协调。
在服务的第一年，有 43 名 aHUS 患者接受了依库珠单抗治疗，其中 15 名儿童和 28 名成人。23 名是新患者，20 名是现患者。在开始使用依库珠单抗之前，23 名新患者中有 15 名需要透析，其中 8 名恢复了肾功能。在接受依库珠单抗治疗的 20 名现患者中，有 12 名是移植患者，其中 8 名预防性使用，4 名用于复发性疾病；所有患者的结局都很好。14 名患者停用了依库珠单抗，其中 5 名患者未恢复肾功能。在这 14 名患者中，有 3 名因复发性疾病（2 例肾外和 1 例肾内）需要重新使用依库珠单抗。在这 43 名患者中，有 2 人死亡，均与依库珠单抗的使用无关。没有发生脑膜炎球菌病的病例。
这项国家服务的建立使英格兰的 aHUS 患者能够在需要时长期接受依库珠单抗治疗。
