期刊
PEDIATRIC NEPHROLOGY
卷 34, 期 11, 页码 2261-2277出版社
SPRINGER
DOI: 10.1007/s00467-018-4091-3
关键词
Atypical hemolytic uremic syndrome; Eculizumab; Restrictive treatment regimen; Personalized medicine; Thrombotic microangiopathy
资金
- ZonMw [836031008]
- Zorgverzekeraars Nederland
With the introduction of the complement C5-inhibitor eculizumab, a new era was entered for patients with atypical hemolytic uremic syndrome (aHUS). Eculizumab therapy very effectively reversed thrombotic microangiopathy and reduced mortality and morbidity. Initial guidelines suggested lifelong treatment and recommended prophylactic use of eculizumab in aHUS patients receiving a kidney transplant. However, there is little evidence to support lifelong therapy or prophylactic treatment in kidney transplant recipients. Worldwide, there is an ongoing debate regarding the optimal dose and duration of treatment, particularly in view of the high costs and potential side effects of eculizumab. An increasing but still limited number of case reports and small cohort studies suggest that a restrictive treatment regimen is feasible. We review the current literature and focus on the safety and efficacy of restrictive use of eculizumab. Our current treatment protocol is based on restrictive use of eculizumab. Prospective monitoring will provide more definite proof of the feasibility of such restrictive treatment.
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