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Expert Review of Hematology Volume 7, 2014 - Issue 5
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Complement in paroxysmal nocturnal hemoglobinuria: exploiting our current knowledge to improve the treatment landscape

Dimitrios C MastellosNCSR ‘Demokritos’ – INRASTES, Division of Biodiagnostic Sciences and Technologies, Aghia Paraskevi Attikis, Greece
,
Daniel RicklinDepartment of Pathology and Laboratory Medicine, University of Pennsylvania, Philadelphia, PA 19104-6100, USA
,
Despina YancopoulouAmyndas Pharmaceuticals, Glyfada, Greece
,
Antonio RisitanoDepartment of Clinical Medicine and Surgery, Division of Hematology, Federico II University of Naples, Naples, Italy
&
John D LambrisDepartment of Pathology and Laboratory Medicine, University of Pennsylvania, Philadelphia, PA 19104-6100, USACorrespondence[email protected]
Pages 583-598 | Published online: 02 Sep 2014

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Sung-Eun Lee & Jong Wook Lee. (2021) Safety of current treatments for paroxysmal nocturnal hemoglobinuria. Expert Opinion on Drug Safety 20:2, pages 171-179.
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Jörg Schubert & Jan Menne. (2017) Eculizumab for the treatment of hemolytic paroxysmal nocturnal hemoglobinuria, atypical hemolytic uremic syndrome and refractory myasthenia gravis. Expert Opinion on Orphan Drugs 5:4, pages 375-379.
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Serena Marotta, Simona Pagliuca & Antonio M Risitano. (2014) Hematopoietic stem cell transplantation for aplastic anemia and paroxysmal nocturnal hemoglobinuria: current evidence and recommendations. Expert Review of Hematology 7:6, pages 775-789.
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Articles from other publishers (37)

Dimitrios C. Mastellos, George Hajishengallis & John D. Lambris. (2023) A guide to complement biology, pathology and therapeutic opportunity. Nature Reviews Immunology.
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Jesse Fishman, Koo Wilson, Aleksandra DrzewieckaMichał PochopieńDavid Dingli. (2023) The cost–effectiveness of pegcetacoplan in complement treatment-naïve adults with paroxysmal nocturnal hemoglobinuria in the USA. Journal of Comparative Effectiveness Research.
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Austin G. Kulasekararaj, David J. Kuter, Morag Griffin, Ilene C. Weitz & Alexander Röth. (2023) Biomarkers and laboratory assessments for monitoring the treatment of patients with paroxysmal nocturnal hemoglobinuria: Differences between terminal and proximal complement inhibition. Blood Reviews 59, pages 101041.
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Christina Lamers, Daniel Ricklin & John D. Lambris. (2023) Complement‐targeted therapeutics: An emerging field enabled by academic drug discovery. American Journal of Hematology 98:S4.
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Anna Duval & Véronique Frémeaux‐Bacchi. (2023) Complement biology for hematologists. American Journal of Hematology 98:S4.
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Christina Lamers, Xiaoguang Xue, Martin Smieško, Henri van Son, Bea Wagner, Nadja Berger, Georgia Sfyroera, Piet Gros, John D. Lambris & Daniel Ricklin. (2022) Insight into mode-of-action and structural determinants of the compstatin family of clinical complement inhibitors. Nature Communications 13:1.
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Raymond S. M. Wong, Humphrey W. H. Pullon, Ismail Amine, Andrija Bogdanovic, Pascal Deschatelets, Cedric G. Francois, Kalina Ignatova, Surapol Issaragrisil, Pimjai Niparuck, Tontanai Numbenjapon, Eloy Roman, Jameela Sathar, Raymond Xu, Mohammed Al-Adhami, Lisa Tan, Eric Tse & Federico V. Grossi. (2022) Inhibition of C3 with pegcetacoplan results in normalization of hemolysis markers in paroxysmal nocturnal hemoglobinuria. Annals of Hematology 101:9, pages 1971-1986.
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Thyago Bispo Leonel, Joel José Megale Gabrili, Carla Cristina Squaiella-Baptistão, Trent M. Woodruff, John D. Lambris & Denise V. Tambourgi. (2022) Bothrops jararaca Snake Venom Inflammation Induced in Human Whole Blood: Role of the Complement System. Frontiers in Immunology 13.
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Dimitrios C. Mastellos, Daniel Ricklin, Georgia Sfyroera & Arvind Sahu. (2022) From discovery to approval: A brief history of the compstatin family of complement C3 inhibitors. Clinical Immunology 235, pages 108785.
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Raymond S.M. Wong. (2022) Safety and efficacy of pegcetacoplan in paroxysmal nocturnal hemoglobinuria. Therapeutic Advances in Hematology 13, pages 204062072211146.
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Antonio M. Risitano, Regis Peffault de Latour, Luana Marano & Camilla Frieri. (2022) Discovering C3 targeting therapies for paroxysmal nocturnal hemoglobinuria: Achievements and pitfalls. Seminars in Immunology 59, pages 101618.
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M. Jalink, E. C. W. de Boer, D. Evers, M. Q. Havinga, J. M. I. Vos, S. Zeerleder, M. de Haas & I. Jongerius. (2021) Halting targeted and collateral damage to red blood cells by the complement system. Seminars in Immunopathology 43:6, pages 799-816.
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Bruno Fattizzo & Austin G. Kulasekararaj. (2020) Second-Generation C5 Inhibitors for Paroxysmal Nocturnal Hemoglobinuria. BioDrugs 34:2, pages 149-158.
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Christopher J Patriquin & Kevin H.M. Kuo. (2019) Eculizumab and Beyond: The Past, Present, and Future of Complement Therapeutics. Transfusion Medicine Reviews 33:4, pages 256-265.
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Dimitrios C. Mastellos, Daniel Ricklin & John D. Lambris. (2019) Clinical promise of next-generation complement therapeutics. Nature Reviews Drug Discovery 18:9, pages 707-729.
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Hongmei Cao, Guodong Zhang, Kristina Ciociola, Lakshmi Amaravadi, Yongchang Qiu & Jiang Wu. (2019) An LC–MS/MS approach to assess total and free protein target in the serum of cynomolgus monkey. Bioanalysis 11:5, pages 393-406.
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Han-Mou Tsai. (2019) Atypical Hemolytic Uremic Syndrome: Beyond Hemolysis and Uremia. The American Journal of Medicine 132:2, pages 161-167.
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Dimitrios C. Mastellos, Edimara S. Reis, Despina Yancopoulou, Antonio M. Risitano & John D. Lambris. (2018) Expanding Complement Therapeutics for the Treatment of Paroxysmal Nocturnal Hemoglobinuria. Seminars in Hematology 55:3, pages 167-175.
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Astrid J.F. Thielen, Sacha Zeerleder & Diana Wouters. (2018) Consequences of dysregulated complement regulators on red blood cells. Blood Reviews 32:4, pages 280-288.
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Amanda Mener, Connie M. Arthur, Seema R. Patel, Jingchun Liu, Jeanne E. Hendrickson & Sean R. Stowell. (2018) Complement Component 3 Negatively Regulates Antibody Response by Modulation of Red Blood Cell Antigen. Frontiers in Immunology 9.
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Astrid J.F. Thielen, Iris M. van Baarsen, Marlieke L. Jongsma, Sacha Zeerleder, Robbert M. Spaapen & Diana Wouters. (2018) CRISPR/Cas9 generated human CD46, CD55 and CD59 knockout cell lines as a tool for complement research. Journal of Immunological Methods 456, pages 15-22.
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Stefan MichelfelderFriedericke FischerAstrid WäldinKim V. HörleMartin PohlJuliana Parsons, Ralf Reski, Eva L. Decker, Peter F. Zipfel, Christine Skerka & Karsten Häffner. (2018) The MFHR1 Fusion Protein Is a Novel Synthetic Multitarget Complement Inhibitor with Therapeutic Potential. Journal of the American Society of Nephrology 29:4, pages 1141-1153.
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Elizabeth Elliott, Salman Waheed, Sahr Syed, Sheila Eswaran, Stephanie Gregory & Jamile Shammo. (2018) Hereditary Hemochromatosis Manifesting After Treatment of Paroxysmal Nocturnal Hemoglobinuria With Eculizumab. Clinical Lymphoma Myeloma and Leukemia 18:1, pages e9-e12.
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Dimitrios C. Mastellos, Edimara S. Reis, Daniel Ricklin, Richard J. Smith & John D. Lambris. (2017) Complement C3-Targeted Therapy: Replacing Long-Held Assertions with Evidence-Based Discovery. Trends in Immunology 38:6, pages 383-394.
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Benjamin P. Davis & Zuhair K. Ballas. (2017) Biologic response modifiers: Indications, implications, and insights. Journal of Allergy and Clinical Immunology 139:5, pages 1445-1456.
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Alexandra Primikyri, Malvina Papanastasiou, Yiannis Sarigiannis, Sophia Koutsogiannaki, Edimara S. Reis, Joel V. Tuplano, Ranillo R.G. Resuello, Bo Nilsson, Daniel Ricklin & John D. Lambris. (2017) Method development and validation for the quantitation of the complement inhibitor Cp40 in human and cynomolgus monkey plasma by UPLC-ESI-MS. Journal of Chromatography B 1041-1042, pages 19-26.
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Chao-Yie Yang, James G. Phillips, Jeanne A. Stuckey, Longchuan Bai, Haiying Sun, James Delproposto, William Clay Brown & Krishnapriya Chinnaswamy. (2016) Buried Hydrogen Bond Interactions Contribute to the High Potency of Complement Factor D Inhibitors. ACS Medicinal Chemistry Letters 7:12, pages 1092-1096.
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Charles J. Parker. (2016) Update on the diagnosis and management of paroxysmal nocturnal hemoglobinuria. Hematology 2016:1, pages 208-216.
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Dimitrios C. Mastellos, Edimara S. Reis, Despina Yancopoulou, George Hajishengallis, Daniel Ricklin & John D. Lambris. (2016) From orphan drugs to adopted therapies: Advancing C3-targeted intervention to the clinical stage. Immunobiology 221:10, pages 1046-1057.
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Daniel Ricklin & John D. Lambris. (2016) New milestones ahead in complement-targeted therapy. Seminars in Immunology 28:3, pages 208-222.
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Daniel Ricklin & John D. Lambris. (2016) Therapeutic control of complement activation at the level of the central component C3. Immunobiology 221:6, pages 740-746.
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Sarah E. Henrickson, Melanie A. Ruffner & Mildred Kwan. (2016) Unintended Immunological Consequences of Biologic Therapy. Current Allergy and Asthma Reports 16:6.
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D.C. Mastellos, D. Ricklin, E. Hajishengallis, G. Hajishengallis & J.D. Lambris. (2016) Complement therapeutics in inflammatory diseases: promising drug candidates for C3-targeted intervention. Molecular Oral Microbiology 31:1, pages 3-17.
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Mie Anemone Nordmaj, Lea Munthe‐Fog, Estrid Hein, Mikkel‐Ole Skjoedt & Peter Garred. (2015) Genetically engineered fusion of MAP‐1 and factor H domains 1–5 generates a potent dual upstream inhibitor of both the lectin and alternative complement pathways. The FASEB Journal 29:12, pages 4945-4955.
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Edimara S. Reis, Dimitrios C. Mastellos, Despina Yancopoulou, Antonio M. Risitano, Daniel Ricklin & John D. Lambris. (2015) Applying complement therapeutics to rare diseases. Clinical Immunology 161:2, pages 225-240.
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Zhuoer LinChristoph Q. Schmidt, Sophia KoutsogiannakiPatrizia Ricci, Antonio M. Risitano, John D. Lambris & Daniel Ricklin. (2015) Complement C3dg-mediated erythrophagocytosis: implications for paroxysmal nocturnal hemoglobinuria. Blood 126:7, pages 891-894.
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Dimitrios C. Mastellos, Despina Yancopoulou, Petros Kokkinos, Markus Huber-Lang, George Hajishengallis, Ali R. Biglarnia, Florea Lupu, Bo Nilsson, Antonio M. Risitano, Daniel Ricklin & John D. Lambris. (2015) Compstatin: a C3-targeted complement inhibitor reaching its prime for bedside intervention. European Journal of Clinical Investigation 45:4, pages 423-440.
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