ESPN 51th Annual Meeting

ESPN 2018


 
TURKISH ATYPICAL HEMOLYTIC UREMIC SYNDROME REGISTRY: Eculizumab treatment in aHUS patients
ESRA BASKIN 1 NUR CANPOLAT 2 KAAN GULLEROGLU 1 ALEV YILMAZ 2 ENGIN MELEK 3 SELÇUK YÜKSEL 4 BORA GULHAN 5 MUKADDES KALYONCU 6 GONUL PARMAKSIZ 1 BIRSIN OZCAKAR 7 FATIH OZALTIN 8 OGUZ SOYLEMEZOGLU 9

1- BASKENT UNIVERSITY
2- İSTANBUL UNIVERSITY
3- ÇUKUROVA UNIVERSITY
4- PAMUKKALE
5- HACETTEPE UNIVERSITY
6- KTU
7- ANKARA UNIVERSITY
8- HACETTEPE UNIVERSITY
9- GAZI UNIVERSITY
 
Introduction:

 

Eculizumab has recently been introduced as a complement blocking therapy. The standard maintenance treatment requires lifelong eculizumab therapy but, the optimal treatment schedule has not been established. We aimed to analyze characteristics and outcomes of patients with discontinued eculizumab  treatment in our registry group.

Material and methods:

 

Clinical and laboratory features and genetic results of 146 patients  were analyzed from the registry. Sixty-six out of 146 patients had received more than 4 doses eculizumab treatment. In the follow-up period eculizumab treatment  was discontinued in 21 patients  who had been stable clinical findings. Eculizumab doses, duration of treatment and association between outcome and treatment were investigated.

Results:

 

The mean age at diagnosis of 21 patients (F/M:14/7)  was 6.54 ±4.68 years,  the mean follow-up duration was 2.77 ±2.3 years.Nine (42.9%) patients had hypocomplementemia, Renal replacement therapy was performed in 17 patients (77.7%) in acute period. The mean eculizumab dose  was 14.45±9.1  None of the patients required renal replacement treatment in last visit, and mean GFR was 106.04±55.92ml/min/1.73m2. Recurrent disease developed in 3 patients within 6 weeks of discontinuation, but then immediately resumed treatment and completely recovered.  Genetic studies are ongoing  and have been completed only for 10 patients

Conclusions:

 

Eculizumab is an effective and well-tolerated therapy for children with aHUS. However, appropriate indications and optimal duration of the treatment remain unclear. During the acute phase of aHUS, eculizumab treatment should be initiated promptly, genetic mutations must be considered and continuation of the drug should be evaluated carefully for possible risks. Close monitoring of patients is crucial for the decision regarding discontinuation of treatment