Ontology type: schema:ScholarlyArticle Open Access: True
2018-07-23
AUTHORSShuichi Ito, Yoshihiko Hidaka, Norimitsu Inoue, Shinya Kaname, Hideki Kato, Masanori Matsumoto, Yoshitaka Miyakawa, Masashi Mizuno, Hirokazu Okada, Akihiko Shimono, Takahisa Matsuda, Shoichi Maruyama, Yoshihiro Fujimura, Masaomi Nangaku, Shoji Kagami
ABSTRACTBackgroundIn 2013, eculizumab was approved for treatment of the atypical hemolytic–uremic syndrome (aHUS) in Japan, which was defined as a thrombotic microangiopathy (TMA) excluding Shiga toxin-producing Escherichia coli-HUS and thrombotic thrombocytopenic purpura. Simultaneously, post-marketing surveillance was started to assess its safety and effectiveness. In 2016, Japanese clinical guide redefined terms to limit the use of “aHUS” to complement-mediated HUS only. Accordingly, TMA with other causes was defined as secondary TMA. Here we report the interim analysis of post-marketing surveillance of pediatric patients with aHUS and secondary TMA.MethodsPediatric patients treated with eculizumab from approval to 15 March 2017 were included in this observational real-world study. Clinical endpoints of effectiveness were TMA event–free status, complete TMA response, platelet count normalization, and improvement of estimated glomerular filtration rate (eGFR). Adverse reactions to eculizumab were also analyzed.ResultsIn 27 pediatric patients with aHUS, median age at diagnosis was 4 years. Complement genes’ variants were detected in 14 of 21 patients (66.7%). Median time from diagnosis to eculizumab initiation was 2.0 days. TMA event–free status, complete TMA response, platelet normalization, and improvement in eGFR were achieved in 85.2, 36.4, 78.3, and 75.0% of patients, respectively. Three patients with aHUS died. Twenty-four and 10 adverse reactions were reported in 31 aHUS patients and 17 secondary TMA patients, respectively; however, no eculizumab-related death or meningococcal infection was reported.ConclusionsThis interim analysis confirmed that eculizumab is well-tolerated and effective for Japanese pediatric patients with aHUS in a real-world setting. More... »
PAGES112-121
http://scigraph.springernature.com/pub.10.1007/s10157-018-1610-2
DOIhttp://dx.doi.org/10.1007/s10157-018-1610-2
DIMENSIONShttps://app.dimensions.ai/details/publication/pub.1105776110
PUBMEDhttps://www.ncbi.nlm.nih.gov/pubmed/30039480
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128 URIs
113 LITERALS
28 BLANK NODES