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Relevant bibliographies by topics / Proximal nocturnal hemoglobinuria

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Journal articles

Academic literature on the topic 'Proximal nocturnal hemoglobinuria'

Author: Grafiati

Published: 3 June 2025

Last updated: 31 July 2025

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Journal articles on the topic "Proximal nocturnal hemoglobinuria"

1

Duran, Munevver N., Zehra Tombul, Mutlu Mete, et al. "Predicting Extravascular Hemolysis in Paroxysmal Nocturnal Hemoglobinuria." Blood 144, Supplement 1 (2024): 2695. https://doi.org/10.1182/blood-2024-204213.

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Introduction Paroxysmal Nocturnal Hemoglobinuria (PNH) is a life-threatening blood disorder characterized by the destruction of red blood cells due to complement system dysregulation. The advent of C5 complement inhibitors has markedly improved the outlook for PNH patients by mitigating intravascular hemolytic crises and thrombotic events. However, many patients undergoing C5-inhibitor treatment experience C3-mediated extravascular hemolysis (EVH), which can lead to transfusion dependence, lower quality of life, and poorer health outcomes. The development of proximal complement inhibitors has

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2

Swathi., D. 1* Ramesh Kumar Reddy. P. 1. Dr. R. Siddarama2. "APLASTIC ANEMIA WITH SUB CONJUNCTIVAL HAEMORRHAGE AND PAROXYSMAL NOCTURNAL HAEMOGLOBINURIA: A RARE CASE REPORT." Indo American Journal of Pharmaceutical Sciences 047, no. 07 (2017): 1952–55. https://doi.org/10.5281/zenodo.832437.

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This Case report describes a case of idiopathic acquired aplastic anemia in an 18-year-old male patient with petechial rash, sub conjunctival haemorrhage and paroxysmal nocturnal haemoglobinuria. Patient was treated with elthrombopag olamine- 50mg and cyclosporine- 200mg these two drugs are showing good response in most of the patients with Aplastic anemia in the absence of human leucocytes antigen (HLA) matched sibling donor. In some studies good response was shown for elthrombopag olamine 150mg/day. In this patient only 50mg of elthrombopag olamine is given, patient was died because of the f

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3

Peixoto, Vanda, Ana Carneiro, Fernanda Trigo, Mónica Vieira, and Cristina Prudêncio. "Paroxysmal Nocturnal Hemoglobinuria: A Case Report in a Pandemic Environment." Reports 6, no. 3 (2023): 42. http://dx.doi.org/10.3390/reports6030042.

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Paroxysmal nocturnal hemoglobinuria (PNH) is a clonal, rare, complement-mediated hemolytic anemia. PNH can be associated with marrow failure and thrombophilia. We present a clinical report of splenic vein thrombosis in a patient with classic PNH. A 41-year-old male with classic PNH, naïve to complement inhibitor therapy, developed splenic vein thrombosis as a major adverse effect after vaccination protocol to prevent meningococcal disease. We also report anticoagulant and eculizumab treatment outcomes. In PNH patients, vaccination should be monitored to prevent major outcome events, like vacci

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4

Wong, Raymond S. M. "Safety and efficacy of pegcetacoplan in paroxysmal nocturnal hemoglobinuria." Therapeutic Advances in Hematology 13 (January 2022): 204062072211146. http://dx.doi.org/10.1177/20406207221114673.

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Paroxysmal nocturnal hemoglobinuria (PNH) is a rare, acquired, hematologic disease characterized by complement-mediated hemolysis, thrombosis, and various degrees of bone marrow dysfunction. Until recently, C5 inhibition with eculizumab or ravulizumab represented the only therapies approved for patients with PNH by the United States Food and Drug Administration (US FDA). Although C5-inhibitors reduce PNH-related signs and symptoms, many patients continue to exhibit persistent anemia and require frequent blood transfusions. In May 2021, pegcetacoplan became the third US FDA-approved treatment f

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5

Vallejo, Carlos, Santiago Bonanad Boix, Fernando Carnicero, et al. "Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) Treated with Pegcetacoplan: Real Life Experience." Blood 142, Supplement 1 (2023): 5653. http://dx.doi.org/10.1182/blood-2023-185570.

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Introduction: C5 inhibitors (C5i) treatment has dramatically improved the management of intravascular hemolysis (IVH) in PNH patients. However, a significant proportion of patients remain anemic due to suboptimal control of IVH and the development of extravascular hemolysis (EVH), consequence of C5i treatment. Pegcetacoplan (PEG) is the first proximal inhibitor of the complement system, through the inhibition of C3, allowing to control both IVH and EVH. PEG has recently been marketed in Spain for use in adult PNH patients who remain anemic after treatment with an C5i for at least 3 months. The

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6

Ahmed, Arooj, Mark Orland, Praveena Thiagarajan, et al. "A Real-Life Experience of Iptacopan in Paroxysmal Nocturnal Hemoglobinuria." Blood 144, Supplement 1 (2024): 5695. https://doi.org/10.1182/blood-2024-211788.

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The availability of complement inhibitors has dramatically changed the management of PNH. The longest experience exists with C5 inhibitors. Introduction of C3 blockade allowed also to overcome extravascular hemolysis in patients with break through hemolysis. Parenteral treatment options like complement protein C5 and C3 inhibitors, thus, became the mainstay therapeutic options in PNH. Recently, the PNH therapeutic armamentarium has been expanded by the FDA approval of an oral factor B blocker, iptacopan administered as single agent to effectively control hemolysis in PNH. Obviously, there are

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7

Peffault de Latour, Régis, Jeffrey Szer, Koo Wilson, et al. "Anchored Indirect Treatment Comparison Finds Comparable Effects of Pegcetacoplan and Iptacopan in Paroxysmal Nocturnal Hemoglobinuria." Blood 144, Supplement 1 (2024): 7706. https://doi.org/10.1182/blood-2024-209712.

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Background Paroxysmal nocturnal hemoglobinuria (PNH) is an ultra-rare, acquired non-malignant hematological disorder characterized by thrombosis risk, high morbidity, and high symptom burden. This anchored indirect treatment comparison (ITC) evaluated the proximal complement inhibitors (Ci), pegcetacoplan (C3i) and iptacopan (factor B inhibitor) in C5i treatment-experienced patients with PNH. Methods Data obtained retrospectively from 2 pivotal clinical studies informed the ITC: patient-level data for pegcetacoplan from PEGASUS (NCT03500549) at 16 weeks, and digitalised published trial data fo

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8

De Castro, Carlos, Phillip Scheinberg, Bing Han, et al. "Patient Experience Interviews with a Novel Treatment Iptacopan for Paroxysmal Nocturnal Hemoglobinuria." Blood 142, Supplement 1 (2023): 7339. http://dx.doi.org/10.1182/blood-2023-186840.

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Introduction: Paroxysmal nocturnal hemoglobinuria (PNH) is an acquired clonal and complex disorder characterized by hemolytic anemia, thrombosis, smooth muscle dystonia, hemoglobinuria, and chronic kidney disease. Individuals with PNH experience severe and chronic fatigue, which can significantly impact their quality of life. While two anti-C5 antibody therapies have been approved for treating PNH (eculizumab and ravulizumab), the majority of patients who use these therapies still do not achieve normal hemoglobin levels. Iptacopan, the first oral proximal complement inhibitor developed to fill

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9

Ninomiya, Haruhiko, Naoshi Obara, Akiko Niiori-Onishi, et al. "Improvement of Renal Function by Long-Term Sustained Eculizumab Treatment in a Patient with Paroxysmal Nocturnal Hemoglobinuria." Case Reports in Hematology 2015 (2015): 1–4. http://dx.doi.org/10.1155/2015/673195.

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Chronic kidney disease (CKD) is one of the major manifestations of paroxysmal nocturnal hemoglobinuria (PNH). CKD in PNH is induced mainly by intravascular hemolysis of PNH-affected red blood cells (RBC) missing the glycosylphosphatidylinositol-anchored proteins with complement-regulatory activities, CD55 and CD59. CKD develops by heme absorption in the proximal tubules resulting in the interstitial deposition of iron in the kidneys. We administered eculizumab to a patient with PNH, who was one of 29 patients enrolled in the AEGIS clinical trial, an open-label study of eculizumab in Japan. The

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10

Horneff, Regina, Barbara Czech, Michael Yeh, and Elena Surova. "Three Years On: The Role of Pegcetacoplan in Paroxysmal Nocturnal Hemoglobinuria (PNH) since Its Initial Approval." International Journal of Molecular Sciences 25, no. 16 (2024): 8698. http://dx.doi.org/10.3390/ijms25168698.

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Paroxysmal nocturnal hemoglobinuria (PNH) is a rare disease characterized by complement-mediated hemolysis and potentially life-threatening complications. Pegcetacoplan, an inhibitor of complement components C3 and C3b, was approved by the US Food and Drug Administration (FDA) and European Medicines Agency (EMA) in 2021. A recent expansion to its indication by the EMA has made pegcetacoplan available for the treatment of both complement inhibitor-naïve and -experienced patients with PNH who have hemolytic anemia, a similarly broad patient population as in the US. This approval was based on res

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