prikaz prve stranice dokumenta Complement Inhibition in Paroxysmal Nocturnal Hemoglobinuria (PNH): A Systematic Review and Expert Opinion from Central Europe on Special Patient Populations
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Scientific paper - Original scientific paper
Complement Inhibition in Paroxysmal Nocturnal Hemoglobinuria (PNH): A Systematic Review and Expert Opinion from Central Europe on Special Patient Populations
Advances in Therapy, 40 (2023), 6; 2752-2772. https://doi.org/10.1007/s12325-023-02510-4
Bodó, Imre; Amine, Ismail; Boban, Ana; Bumbea, Horia; Kulagin, Alexander; Lukina, Elena; Piekarska, Agnieszka; Preložnik Župan, Irena; Sokol, Juraj; Windyga, Jerzy; Cermak, Jaroslav

Institutional repository: Dr Med - University of Zagreb School of Medicine Digital Repository

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Bodó, I., Amine, I., Boban, A., Bumbea, H., Kulagin, A., Lukina, E. ... Cermak, J. (2023). Complement Inhibition in Paroxysmal Nocturnal Hemoglobinuria (PNH): A Systematic Review and Expert Opinion from Central Europe on Special Patient Populations. Advances in Therapy, 40. (6), 2752-2772. doi: 10.1007/s12325-023-02510-4

Bodó, Imre, et al. "Complement Inhibition in Paroxysmal Nocturnal Hemoglobinuria (PNH): A Systematic Review and Expert Opinion from Central Europe on Special Patient Populations." Advances in Therapy, vol. 40, no. 6, 2023, pp. 2752-2772. https://doi.org/10.1007/s12325-023-02510-4

Bodó, Imre, Ismail Amine, Ana Boban, Horia Bumbea, Alexander Kulagin, Elena Lukina, Agnieszka Piekarska, et al. "Complement Inhibition in Paroxysmal Nocturnal Hemoglobinuria (PNH): A Systematic Review and Expert Opinion from Central Europe on Special Patient Populations." Advances in Therapy 40, no. 6 (2023): 2752-2772. https://doi.org/10.1007/s12325-023-02510-4

Bodó, I., et al. (2023) 'Complement Inhibition in Paroxysmal Nocturnal Hemoglobinuria (PNH): A Systematic Review and Expert Opinion from Central Europe on Special Patient Populations', Advances in Therapy, 40(6), pp. 2752-2772. doi: 10.1007/s12325-023-02510-4

Bodó I, Amine I, Boban A, Bumbea H, Kulagin A, Lukina E, and sur.. Complement Inhibition in Paroxysmal Nocturnal Hemoglobinuria (PNH): A Systematic Review and Expert Opinion from Central Europe on Special Patient Populations. Advances in Therapy [Internet]. 2023 April [cited 2025 January 16];40(6):2752-2772. doi: 10.1007/s12325-023-02510-4

I. Bodó, et al., "Complement Inhibition in Paroxysmal Nocturnal Hemoglobinuria (PNH): A Systematic Review and Expert Opinion from Central Europe on Special Patient Populations", Advances in Therapy, vol. 40, no. 6, pp. 2752-2772, April 2023. [Online]. Available at: https://urn.nsk.hr/urn:nbn:hr:105:527109. [Accessed: 16 January 2025]

Metadata
Title (english)Complement Inhibition in Paroxysmal Nocturnal Hemoglobinuria (PNH): A Systematic Review and Expert Opinion from Central Europe on Special Patient Populations
AuthorImre Bodó
AuthorIsmail Amine
AuthorAna Boban
AuthorHoria Bumbea
AuthorAlexander Kulagin
AuthorElena Lukina
AuthorAgnieszka Piekarska
AuthorIrena Preložnik Župan
AuthorJuraj Sokol
AuthorJerzy Windyga
AuthorJaroslav Cermak
Author's institutionUniversity of Zagreb
School of Medicine
(Department of Internal Medicine)
Scientific / art field,
discipline and subdiscipline		BIOMEDICINE AND HEALTHCARE
Clinical Medical Sciences
Internal Medicine
Abstract (english)
Introduction: Hemolysis in paroxysmal nocturnal hemoglobinuria (PNH) is complement-mediated due to the lack of complement inhibitors in the hemopoietic cell membranes, making complement inhibition the best approach to manage PNH. Three complement inhibitors are approved by the European Medicines Agency as targeted therapy for PNH: eculizumab and ravulizumab, two humanized monoclonal antibodies targeting the same complement 5 (C5) epitope, approved in 2007 and 2019, respectively, and the more recently approved cyclic peptide, the complement 3 (C3) inhibitor pegcetacoplan. Although national and international PNH treatment guidelines exist, they do not take into consideration the latest clinical trial evidence. Given the lack of evidence-based data for some clinical situations encountered in real life, we identified specific populations of patients who may benefit from switching to proximal C3 from terminal C5 inhibition. Methods: The expert recommendations presented here were created using a Delphi-like process by a group of expert PNH specialists across Central Europe. Based on an initial advisory board meeting discussion, recommendations were prepared and reviewed as part of a Delphi survey to test agreement. Results: Using a systematic approach, literature databases were searched for relevant studies, and 50 articles were reviewed by the experts and included as supporting evidence. Conclusion: Implementation of these recommendations uniformly across healthcare institutions will promote the best use of complement inhibition in managing PNH, and has the potential to positively impact patient outcomes in Central Europe and worldwide.
Keywords (english)
Languageenglish
Publication typeScientific paper - Original scientific paper
Publication statusPublished
Peer reviewPeer review
Publication versionPublished version
Journal titleAdvances in Therapy
Numberingvol. 40, no. 6, pp. 2752-2772
p-ISSN0741-238X
e-ISSN1865-8652
DOIhttps://doi.org/10.1007/s12325-023-02510-4
URN:NBNurn:nbn:hr:105:527109
Publication2023-04
Document URLhttps://link.springer.com/10.1007/s12325-023-02510-4
Type of resourceText
Access conditionsOpen access
Terms of useLicence Attribution-NonCommercial 4.0 International (CC BY-NC 4.0) icon
Created on2023-11-24 10:43:00