Long-term follow-up of patients with paroxysmal nocturnal hemoglobinuria treated with eculizumab: post-marketing surveillance in Japan

Autor: Takayuki Ikezoe, Hideyoshi Noji, Yasutaka Ueda, Yoshinobu Kanda, Shinichiro Okamoto, Kensuke Usuki, Takahisa Matsuda, Hirozumi Akiyama, Akihiko Shimono, Yuji Yonemura, Tatsuya Kawaguchi, Shigeru Chiba, Yuzuru Kanakura, Jun‑ichi Nishimura, Haruhiko Ninomiya, Naoshi Obara
Rok vydání: 2022
Předmět:
Zdroj: International Journal of Hematology. 115:470-480
ISSN: 1865-3774
0925-5710
DOI: 10.1007/s12185-022-03287-y
Popis: All Japanese patients with paroxysmal nocturnal hemoglobinuria (PNH) treated with eculizumab were enrolled in post-marketing surveillance (PMS) between June 2010 and August 2019 to assess the long-term effectiveness and safety of eculizumab. The reduction in intravascular hemolysis, the change in hemoglobin (Hb) level, and the change in renal function were assessed to determine the effectiveness of eculizumab. The types and frequencies of adverse events (AEs) were assessed to determine its safety. A total of 632 patients were enrolled and the median treatment duration was 3.6 years. Treatment with eculizumab significantly reduced lactate dehydrogenase (LDH) levels and significantly increased Hb levels. These changes were maintained for up to 5 years of treatment. An estimated glomerular filtration rate ≥ 60 ml/min/1.73 m
Databáze: OpenAIRE
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