イトウ　ナオコ   井藤　奈央子    所属   医学部 医学科（東京女子医科大学病院）    職種   助教
論文種別	原著
言語種別	英語
査読の有無	査読あり
表題	Efficacy and safety of eculizumab in childhood atypical hemolytic uremic syndrome in Japan.
掲載誌名	正式名：Clinical and experimental nephrology 略　 称：Clin Exp Nephrol ISSNコード：14377799/13421751
掲載区分	国外
巻・号・頁	20(2),pp.265-72
著者・共著者	Ito Naoko, Hataya Hiroshi, Saida Ken, Amano Yoshiro, Hidaka Yoshihiko, Motoyoshi Yaeko, Ohta Toshiyuki, Yoshida Yasuhiro, Terano Chikako, Iwasa Tadashi, Kubota Wataru, Takada Hidetoshi, Hara Toshiro, Fujimura Yoshihiro, Ito Shuichi
発行年月	2016/04
概要	BACKGROUND:Atypical hemolytic uremic syndrome (aHUS) is a severe life-threatening disease with frequent progression to end-stage renal disease (ESRD). Eculizumab, a humanized anti-C5 monoclonal antibody targeting the activated complement pathway, has recently been introduced as a novel therapy against aHUS. We, therefore, investigated the efficacy and safety of eculizumab in Japanese pediatric patients.METHODS:We retrospectively analyzed clinical course and laboratory data of the first ten children with aHUS treated with eculizumab nationwide.RESULTS:Seven patients were resistant to plasma therapy and three were dependent on it. Causative gene mutations were found in five patients. Two patients had anti-complement factor H autoantibody. Three patients had a family history of thrombotic microangiopathy (TMA). After initiation of eculizumab, all patients immediately achieved hematological remission and could successfully discontinue plasma therapy. The median periods to normalization of platelet count, lactate dehydrogenase levels and disappearance of schistocytes were 5.5, 17 and 12 days, respectively. Nine patients recovered their renal function and the median period to terminate renal replacement therapy (RRT) was 3 days. However, two patients progressed to ESRD and required chronic RRT at the last observation. No patients had a relapse of TMA under regular eculizumab therapy. No serious adverse events occurred during the follow-up period.CONCLUSIONS:Eculizumab is efficacious and well-tolerated therapy for children with aHUS. Although pathogenic mutations could not be detected in five patients, all patients showed immediate normalization of hematological abnormalities, strongly suggesting complement-related aHUS. This prompt hematological amelioration can become an indicator for therapeutic efficacy of eculizumab. However, appropriate indications and optimal duration of the treatment remain unclear.
DOI	10.1007/s10157-015-1142-y
PMID	26156042