Eculizumab in atypical hemolytic uremic syndrome: strategies toward restrictive use

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 treat...

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Bibliographic Details
Published in:Pediatric nephrology (Berlin, West) West), 2019-11, Vol.34 (11), p.2261-2277
Main Authors: Wijnsma, Kioa L., Duineveld, Caroline, Wetzels, Jack F. M., van de Kar, Nicole C. A. J.
Format: Article
Language:English
Subjects:
Case reports
Children
Complement component C5
Diagnosis
Diseases
Drug therapy
Evaluation
Hemolytic uremic syndrome
Kidney transplantation
Medicine
Medicine & Public Health
Monoclonal antibodies
Morbidity
Nephrology
Patients
Pediatrics
Practice guidelines (Medicine)
Precision medicine
Review
Thrombotic microangiopathy
Urology
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Description
Summary: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.
ISSN:0931-041X
1432-198X
DOI:10.1007/s00467-018-4091-3