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Paroxysmal Nocturnal Hemoglobinuria pp 271–282Cite as

Clinical Effects of Eculizumab in PNH

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Abstract

Eculizumab is a humanized monoclonal antibody which binds with high affinity to the fifth component of complement preventing its cleavage into C5a and C5b. Given that the clinical manifestations of paroxysmal nocturnal haemoglobinuria (PNH) are based on the activity of C5b-9, (the membrane attack complex) on unprotected PNH erythrocytes, eculizumab was tested as a therapeutic in this disease in four clinical trials beginning in 2002, including a placebo-controlled double-blind study, and was found to have efficacy in reducing markers of intravascular haemolysis, improving haemoglobin levels and reducing transfusion requirements as well as improving renal impairment, decreasing the rate and risk of thromboembolic phenomena and improving many measurable quality of life parameters. Additionally, studies undertaken following the completion of the trials suggest an improvement in overall survival such that patients treated with eculizumab for up to 8 years appear to have survival very similar to an age- and sex-matched population. This well-tolerated therapy has maintained all of the clinical benefits seen in the clinical trials over long-term use.

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Authors and Affiliations

  1. Department of Clinical Haematology & BMT Service, The Royal Melbourne Hospital, 300 Grattan Street, 3050, Parkville, VIC, Australia

    Jeffrey Szer

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  1. Jeffrey Szer
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Correspondence to Jeffrey Szer .

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Editors and Affiliations

  1. Department of Hematology and Ocology, Osaka University Graduate School of Medicine, Suita, Osaka, Japan

    Yuzuru Kanakura

  2. Immunology Frontier Research Center, Osaka University, Suita, Osaka, Japan

    Taroh Kinoshita

  3. Dept of Hematology and Ocology, Osaka University Dept of Hematology and Ocology, Suita, Osaka, Japan

    Jun-ichi Nishimura

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Szer, J. (2017). Clinical Effects of Eculizumab in PNH. In: Kanakura, Y., Kinoshita, T., Nishimura, Ji. (eds) Paroxysmal Nocturnal Hemoglobinuria. Springer, Tokyo. https://doi.org/10.1007/978-4-431-56003-6_16

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  • DOI: https://doi.org/10.1007/978-4-431-56003-6_16

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  • Publisher Name: Springer, Tokyo

  • Print ISBN: 978-4-431-56001-2

  • Online ISBN: 978-4-431-56003-6

  • eBook Packages: MedicineMedicine (R0)

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