Esra Baskin1, Kibriya Fidan2, Bora Gulhan3, Kaan Gulleroglu1, Nur Canpolat4, Alev Yilmaz5, Gonül Parmakiz6, Zeynep Birsin Özçakar7, Fatih Ozaltin3, Oguz Soylemezoglu8. 1. Department of Pediatric Nephrology, Başkent University, Ankara, Turkey. 2. Department of Pediatric Nephrology, School of Medicine, Gazi University, Ankara, Turkey. kibriyafidan@yahoo.co.uk. 3. Department of Pediatric Nephrology, Hacettepe University Faculty of Medicine, Ankara, Turkey. 4. Department of Pediatric Nephrology, Istanbul University-Cerrahpasa, İstanbul, Turkey. 5. Department of Pediatric Nephrology, Istanbul University Istanbul School of Medicine, İstanbul, Turkey. 6. Department of Pediatric Nephrology, Başkent University Adana Dr. Turgut Noyan Training and Research Center, Adana, Turkey. 7. Department of Pediatric Nephrology, Ankara University Medical School, Ankara, Turkey. 8. Department of Pediatric Nephrology, School of Medicine, Gazi University, Ankara, Turkey.
Abstract
INTRODUCTION: Eculizumab is effective treatment of pediatric atypical hemolytic uremic syndrome (aHUS). However, the optimal duration of treatment is not clearly defined. The aim of this study was to retrospectively analyze the outcome of pediatric patients with aHUS, who started eculizumab treatment but discontinued it during the follow-up period. METHODS: The clinical and laboratory findings of the pediatric patients with aHUS were recorded on a web-based, national registry system, known as the Turkish aHUS Registry. The study included 63 patients who had to have received more than four doses of eculizumab during the acute phase of the disease. RESULTS: The median age at diagnosis was 3.62 (IQR: 1.29-6.14) years. During the follow-up period, 39 patients continued to receive standard eculizumab treatment (standard treatment group, treatment every 2 weeks) while 24 received an extended dose of eculizumab at three-four-week intervals (non-standard treatment group). There was no significant difference between both groups in terms of clinical and laboratory parameters. Eculizumab treatment was discontinued in 18 patients (30.7%, F/M:11/7), and the median age of these patients at diagnosis and their median follow-up duration were 4.0 (IQR:2.7-10.2) and 4.2 (IQR:2.2-7) years respectively. The median eGFR at the last visit was 110 (84.7-146.1)ml/min/1.73 m2. Fourteen patients remained in remission without any sign of the disease. Recurrence occurred in four (22.2%) patients, in which eculizumab was immediately started again and complete remission was achieved. CONCLUSION: Eculizumab is a successful treatment option in pediatric patients with aHUS and it can be safely discontinued with close monitoring in a selected group of patients. In case of recurrence, eculizumab should be restarted immediately to achieve complete remission.
INTRODUCTION: Eculizumab is effective treatment of pediatric atypical hemolytic uremic syndrome (aHUS). However, the optimal duration of treatment is not clearly defined. The aim of this study was to retrospectively analyze the outcome of pediatric patients with aHUS, who started eculizumab treatment but discontinued it during the follow-up period. METHODS: The clinical and laboratory findings of the pediatric patients with aHUS were recorded on a web-based, national registry system, known as the Turkish aHUS Registry. The study included 63 patients who had to have received more than four doses of eculizumab during the acute phase of the disease. RESULTS: The median age at diagnosis was 3.62 (IQR: 1.29-6.14) years. During the follow-up period, 39 patients continued to receive standard eculizumab treatment (standard treatment group, treatment every 2 weeks) while 24 received an extended dose of eculizumab at three-four-week intervals (non-standard treatment group). There was no significant difference between both groups in terms of clinical and laboratory parameters. Eculizumab treatment was discontinued in 18 patients (30.7%, F/M:11/7), and the median age of these patients at diagnosis and their median follow-up duration were 4.0 (IQR:2.7-10.2) and 4.2 (IQR:2.2-7) years respectively. The median eGFR at the last visit was 110 (84.7-146.1)ml/min/1.73 m2. Fourteen patients remained in remission without any sign of the disease. Recurrence occurred in four (22.2%) patients, in which eculizumab was immediately started again and complete remission was achieved. CONCLUSION: Eculizumab is a successful treatment option in pediatric patients with aHUS and it can be safely discontinued with close monitoring in a selected group of patients. In case of recurrence, eculizumab should be restarted immediately to achieve complete remission.
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Authors: Fatih Ozaltin; Binghua Li; Alysha Rauhauser; Sung-Wan An; Oguz Soylemezoglu; Ipek Isik Gonul; Ekim Z Taskiran; Tulin Ibsirlioglu; Emine Korkmaz; Yelda Bilginer; Ali Duzova; Seza Ozen; Rezan Topaloglu; Nesrin Besbas; Shazia Ashraf; Yong Du; Chaoying Liang; Phylip Chen; Dongmei Lu; Komal Vadnagara; Susan Arbuckle; Deborah Lewis; Benjamin Wakeland; Richard J Quigg; Richard F Ransom; Edward K Wakeland; Matthew K Topham; Nicolas G Bazan; Chandra Mohan; Friedhelm Hildebrandt; Aysin Bakkaloglu; Chou-Long Huang; Massimo Attanasio Journal: J Am Soc Nephrol Date: 2012-12-28 Impact factor: 14.978