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Artykuły w czasopismach na temat „Proximal nocturnal hemoglobinuria”

Autor: Grafiati

Data publikacji: 3 czerwca 2025

Data aktualizacji: 31 lipca 2025

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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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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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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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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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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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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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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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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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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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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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Anliker, Markus, Daniela Drees, Lorin Loacker, et al. "Upregulation of Checkpoint Ligand Programmed Death-Ligand 1 in Patients with Paroxysmal Nocturnal Hemoglobinuria Explained by Proximal Complement Activation." Journal of Immunology 208, no. 5 (2022): 1248–58. http://dx.doi.org/10.4049/jimmunol.2100031.

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Schrezenmeier, Hubert, Mohammed Mahdi, Christoph Gasteyger, and Cecile De Coster. "Breakthrough Hemolysis Events in Patients with Paroxysmal Nocturnal Hemoglobinuria: Data from Danicopan and Pegcetacoplan Trials." Blood 144, Supplement 1 (2024): 2694. https://doi.org/10.1182/blood-2024-203195.

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Background In the management of paroxysmal nocturnal hemoglobinuria (PNH), breakthrough intravascular hemolysis is indicative of insufficient disease control and may lead to significant morbidity and mortality. The terminal complement component 5 (C5) inhibitor (C5i) ravulizumab is the standard of care treatment for patients with PNH, where available. Danicopan is a first-in-class oral factor D inhibitor that targets the alternate pathway of the complement system. In the phase 3 ALPHA trial (NCT04469465), add-on danicopan therapy to background ravulizumab or eculizumab in patients with PNH and

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Kudlay, D. A., B. A. Bakirov, and V. N. Pavlov. "Biotechnological products for the treatment of complement system disorders including paroxysmal nocturnal hemoglobinuria: currently available and in development." Pediatric Hematology/Oncology and Immunopathology 19, no. 3 (2020): 164–72. http://dx.doi.org/10.24287/1726-1708-2020-19-3-164-172.

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Paroxysmal nocturnal hemoglobinuria (PNH) is a rare acquired clonal blood disorder caused by somatic mutations in phosphatidylinositol glycan, class A gene (PIG-A) in hematopoietic stem cells which manifests as haemolytic anemia, bone marrow failure, thromboses, impaired renal function, and other severe clinical symptoms. The management of PNH is a clinical challenge requiring a comprehensive approach. Over the past decade, target therapy with eculizumab, an antibody inhibitor of terminal complement activation, has played a key role in the treatment of PNH. Eculizumab is the first humanized an

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Villegas, Ana, Ataulfo Gonzalez, Fatima Matute, Jorge Martinez Nieto, and Felix de la Fuente. "Value Of Magnetic Resonance Imaging In The Study and Follow-Up Of Paroxysmal Nocturnal Hemoglobinuria." Blood 122, no. 21 (2013): 4872. http://dx.doi.org/10.1182/blood.v122.21.4872.4872.

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Abstract Introduction Renal damage is relatively frequent in Paroxysmal Nocturnal Hemoglobinuria (PNH). Iron accumulation from hemolysis in the renal tubules has been found to be one of the triggering factors. Hemosiderin accumulates in the epithelial cells of the proximal renal tubules localized in the renal cortex, and they can be measured (and monitoring) using magnetic resonance imaging (MRI). Methods We studied 7 PNH patients with MR using T2-weighted gradient echo and multiecho sequences. We quantified renal, hepatic and myocardial iron with T2* relaxometry model. Four of these patients

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Veeramreddy, Padmaja, and Srinivasa Reddy Sanikommu. "Efficacy and Safety of Single Agent Proximal Complement Inhibitors in Paroxysmal Nocturnal Hemoglobinuria (PNH) Patients with Residual Anemia Despite of Adequate Terminal Complement Inhibitor Use." Blood 142, Supplement 1 (2023): 5650. http://dx.doi.org/10.1182/blood-2023-190291.

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Background: Introduction of C5 inhibitors is a major advancement in the treatment of Paroxysmal Nocturnal Hemoglobinuria (PNH) patients. First Eculizumab and then Ravulizumab significantly improved anemia by controlling intravascular hemolysis (IVH), reduced thrombosis risk, improved quality of life and prolonged survival. However, a significant proportion of PNH patients remain anemic with transfusion dependence despite maximal C5 inhibition due to C3 mediated residual extravascular hemolysis (EVH). Recently two randomized, open label, controlled trials with proximal complement inhibitors wer

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Vara, Míriam, Maite Moreno, Javier Arzuaga, Sara Hormaza, and Juan Carlos García-Ruiz. "A Case Report of a Patient with Paroxysmal Nocturnal Hemoglobinuria Treated with Pegcetacoplan Who Underwent Major Surgery." Blood 142, Supplement 1 (2023): 5656. http://dx.doi.org/10.1182/blood-2023-182242.

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Paroxysmal Nocturnal Hemoglobinuria (PNH) is characterized by intravascular hemolysis, thrombosis and bone marrow failure. The appearance of the anti-C5 monoclonal antibody eculizumab revolutionized the treatment of PNH, controlling hemolysis and the incidence of thrombosis. However, some patients persist with anemia, due in part to extravascular C3-mediated hemolysis. More recently, the anti-C3 monoclonal antibody pegcetacoplan, which blocks the most proximal part of the complement cascade, has been marketed in Europe, achieving improvements in anemia in these patients. However, intercurrent

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Jang, Jun-Ho, Lily LL Wong, Bor-Sheng Ko, et al. "12-Month Analysis of a Phase 2 Study of Iptacopan (LNP023) Monotherapy for Paroxysmal Nocturnal Hemoglobinuria." Blood 138, Supplement 1 (2021): 2173. http://dx.doi.org/10.1182/blood-2021-152518.

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Abstract Background: Paroxysmal nocturnal hemoglobinuria (PNH) is a rare, acquired hematological disorder caused by somatic mutations in the phosphatidylinositol glycan A (PIGA) gene in hematopoietic stem cells, resulting in complement alternative pathway (AP)-mediated severe hemolysis, life-threatening thrombosis, and impaired bone marrow function. The current standard-of-care for PNH consists of anti-C5 blockade with either eculizumab or ravulizumab. While both monoclonal antibodies effectively control intravascular hemolysis (IVH), reduce thrombosis and improve long-term survival, a signifi

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Peffault De Latour, Regis, Carlos M. de Castro, Jeffrey Szer, et al. "Pegcetacoplan Is Superior to Eculizumab in Patients with Paroxysmal Nocturnal Hemoglobinuria Regardless of Prior Transfusion Requirement." Blood 136, Supplement 1 (2020): 32–33. http://dx.doi.org/10.1182/blood-2020-141061.

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INTRODUCTION Paroxysmal nocturnal hemoglobinuria (PNH) is a rare, acquired hematologic disease characterized by chronic complement-mediated intravascular and extravascular hemolysis. In the phase 3 PEGASUS study (NCT03500549), pegcetacoplan, a C3 inhibitor targeting the proximal complement pathway, was superior to eculizumab (ECU) on the primary endpoint of hemoglobin (Hb) change from baseline at week 16, and improved clinical and hematologic parameters. Additional analyses assessed if any groups of patients might experience further benefit from pegcetacoplan. METHODS Patients ≥18 years old wi

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Gandhi, Shreyans, Regis Peffault De Latour, Katharina Pannagl, et al. "Incremental Effectiveness of Iptacopan Compared with C5 Inhibitors in Adult Patients with Paroxysmal Nocturnal Hemoglobinuria: Results from a Modelling Analysis." Blood 144, Supplement 1 (2024): 5012. https://doi.org/10.1182/blood-2024-201424.

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Objective Intravenous infusion complement-5 inhibitors (C5i; eculizumab, ravulizumab) have been the primary therapies for patients with paroxysmal nocturnal hemoglobinuria (PNH). New treatments with different mechanisms of action have been approved recently, including iptacopan, a Factor B proximal complement inhibitor that is the first oral monotherapy for the treatment of adult patients with PNH. Health technology assessments (HTA) are regularly conducted to inform decision making regarding the funding or reimbursement of new drugs and often include cost-effectiveness analyses, which compare

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Snellman, Josefin, Ranjan Tiwari, Abdulfaheem Khan, Sviatlana Rizk, and Virginia Pilipovic. "Iptacopan Treatment Improves Clinical Parameters in a Real-World Cohort with Paroxysmal Nocturnal Hemoglobinuria: Evidence from a Managed Access Program." Blood 144, Supplement 1 (2024): 5692. https://doi.org/10.1182/blood-2024-209893.

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Background: Paroxysmal nocturnal hemoglobinuria (PNH), a rare acquired hematopoietic stem cell disorder, is life-threatening and associated with complement-mediated hemolysis, thrombosis, and bone marrow failure. While several treatment options for PNH are available, remaining unmet clinical need and access to effective therapies continue to be a challenge for some patients. Iptacopan is the first oral, proximal complement inhibitor that targets Factor B to inhibit the alternative pathway. In 2023, prior to launch, a managed access program (MAP) was initiated to provide iptacopan (200 mg, twic

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Risitano, Antonio, Ilene C. Weitz, Carlos M. de Castro, et al. "Categorized Hematologic Response to Pegcetacoplan Versus Eculizumab in Patients with Paroxysmal Nocturnal Hemoglobinuria: Post Hoc Analysis of Data from a Phase 3 Randomized Trial (PEGASUS)." Blood 136, Supplement 1 (2020): 44–45. http://dx.doi.org/10.1182/blood-2020-142166.

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INTRODUCTION Paroxysmal nocturnal hemoglobinuria (PNH) is a rare, acquired, clonal, nonmalignant hematologic disease characterized by complement-mediated red blood cell hemolysis. The current standard of care for patients with PNH is C5 inhibition. Anemia persists in up to ~70% of patients receiving eculizumab and is attributed to persistent intravascular hemolysis (IVH) and mostly to C3-mediated extravascular hemolysis (EVH). Pegcetacoplan is a pegylated pentadecapeptide C3 inhibitor targeting proximal complement to control both IVH and EVH. PEGASUS is a phase 3, open-label, active-comparator

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Brodsky, Robert A., Regis Peffault De Latour, Scott T. Rottinghaus, et al. "A Prospective Analysis of Breakthrough Hemolysis in 2 Phase 3 Randomized Studies of Ravulizumab (ALXN1210) Versus Eculizumab in Adults with Paroxysmal Nocturnal Hemoglobinuria." Blood 132, Supplement 1 (2018): 2330. http://dx.doi.org/10.1182/blood-2018-99-110874.

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Abstract Background/Objective: Breakthrough hemolysis (BTH) is the return of hemolytic disease activity during treatment with complement C5 inhibitors for paroxysmal nocturnal hemoglobinuria (PNH). BTH may be associated with inadequate C5 inhibition or complement activating conditions (eg, infection). Despite reports that up to 19% of patients (pts) receiving eculizumab may experience BTH, there is no commonly accepted definition of BTH. The definition of BTH was derived from literature review and expert consensus. BTH was defined as ≥1 new or worsening symptom/sign of intravascular hemolysis

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Hill, Anita, Caroline I. Piatek, Régis Peffault de Latour, et al. "Breakthrough Hemolysis in Adult Patients with Paroxysmal Nocturnal Hemoglobinuria Treated with Ravulizumab: Results of a 52-Week Extension from Two Phase 3 Studies." Blood 134, Supplement_1 (2019): 952. http://dx.doi.org/10.1182/blood-2019-128929.

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INTRODUCTION In patients (pts) with paroxysmal nocturnal hemoglobinuria (PNH) receiving eculizumab, approximately 11%-27% may experience breakthrough hemolysis (BTH)-the return of hemolytic disease activity. BTH may be associated with inadequate C5 inhibition or complement-activating conditions (CACs; eg, infection). Although there is no broad consensus regarding the definition of BTH, this study defined BTH based on literature review and expert consensus: ≥1 new or worsening sign or symptom of intravascular hemolysis (fatigue, hemoglobinuria, abdominal pain, dyspnea, anemia [hemoglobin &l

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Risitano, Antonio M., Regis Peffault De Latour, Jun Ho Jang, et al. "Exposure-Response Relationships between the Complement Factor B Inhibitor Iptacopan and Lactate Dehydrogenase (LDH) and Hemoglobin (Hb) in Patients (Pts) with Paroxysmal Nocturnal Hemoglobinuria (PNH)." Blood 142, Supplement 1 (2023): 5643. http://dx.doi.org/10.1182/blood-2023-179960.

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Background: PNH is an ultrarare disease characterized by complement-mediated hemolysis and subsequent anemia, bone marrow failure and thrombosis. In pts not treated with complement inhibitors, intravascular hemolysis (IVH) results from unregulated progression of the complement system and membrane attack complex formation. IVH is controlled in pts treated with anti-C5 therapy, but extravascular hemolysis (EVH) may emerge and cause persistent anemia. High LDH levels are a marker of ongoing IVH, while anemia can result from both IVH and EVH. Iptacopan is the first oral proximal complement inhibit

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Baltcheva, Irina, Christian Bartels, Marie-Anne Valentin, Robert Schmouder, Guido Junge, and Jing Yu. "Exposure-Response Relationships between the Factor B Inhibitor Iptacopan and Complement Biomarkers in Healthy Volunteers and Patients (Pts) with Paroxysmal Nocturnal Hemoglobinuria (PNH), C3 Glomerulopathy (C3G) or IgA Nephropathy (IgAN)." Blood 142, Supplement 1 (2023): 5640. http://dx.doi.org/10.1182/blood-2023-180004.

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Background: Iptacopan is the first oral proximal complement inhibitor that targets factor B to selectively inhibit the alternative pathway (AP) of the complement system. Iptacopan is currently being investigated in Phase III trials in PNH, C3G and IgAN. AP biomarkers can be used to evaluate the inhibitory effect of iptacopan. Wieslab ® ex vivo assay measures the amount of soluble C5b-9 (sC5b-9) formation in serum after AP activation. Plasma sC5b-9 is the final product of complement activation and is also known as the membrane attack complex. Plasma Bb levels increase following AP activation. A

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Peffault De Latour, Regis, Austin Kulasekararaj, Phillip Scheinberg, et al. "Clinical Breakthrough Hemolysis (BTH) during Monotherapy with the Oral Factor B Inhibitor Iptacopan Is Generally Not Severe and Managed without Treatment Discontinuation: 48-Week Data from the Phase III Apply-PNH and Appoint-PNH Trials in Paroxysmal Nocturnal Hemoglobinuria (PNH)." Blood 142, Supplement 1 (2023): 1338. http://dx.doi.org/10.1182/blood-2023-179377.

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Background: In PNH, BTH is characterized by the re-emergence of intravascular hemolysis (IVH) despite ongoing complement inhibition and is recognized by the reappearance of IVH/PNH signs/symptoms, coupled with a marked increase in lactate dehydrogenase (LDH) and sharp decrease in hemoglobin (Hb). Iptacopan is the first oral proximal complement inhibitor targeting factor B in the alternative pathway and has shown efficacy and safety in complement inhibitor-naïve PNH patients (pts; APPOINT-PNH; NCT04820530) and pts with persistent anemia despite anti-C5 treatment (APPLY-PNH; NCT04558918). In the

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Ito, Satoko, Karthik Chetlapalli, Kunal C. Potnis, et al. "Setting Cost-Effective Price Thresholds before FDA Approval: Cost-Effectiveness of Iptacopan Monotherapy Versus Standard-of-Care Anti-C5 Therapy in Transfusion-Dependent, Treatment-Experienced Adult Patients with Paroxysmal Nocturnal Hemoglobinuria in the United States." Blood 142, Supplement 1 (2023): 5042. http://dx.doi.org/10.1182/blood-2023-188063.

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Introduction: Oral complement inhibition therapy has the potential to transform the care of patients with paroxysmal nocturnal hemoglobinuria (PNH), a life-threatening hematological disorder characterized by intravascular hemolysis, thrombosis, and bone marrow failure. Current standard-of-care (SOC) for PNH are monoclonal antibodies targeting the distal complement component 5 (C5) and include the intravenous agents eculizumab and ravulizumab. However, this distal mechanism of action allows extravascular hemolysis to persist. Intravenous therapy also requires patient and nursing time spent for

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Schmouder, Robert L., Melissa Hackling, Bharti Shah, S. Eralp Bellibas, and Kenneth Kulmatycki. "Multiple Supratherapeutic Doses of Iptacopan 400 Mg Twice Daily Are Well Tolerated in Healthy Participants: Safety Findings from a Randomized, Participant-Blinded, Placebo-Controlled, Phase I Study." Blood 144, Supplement 1 (2024): 5670. https://doi.org/10.1182/blood-2024-198628.

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Background: Iptacopan, the first oral proximal complement inhibitor that targets factor B to selectively inhibit the alternative pathway of the complement system, is approved as a monotherapy for adults with paroxysmal nocturnal hemoglobinuria and is being investigated in diseases such as immunoglobulin A nephropathy, C3 glomerulopathy and atypical hemolytic uremic syndrome. Iptacopan is administered at a therapeutic dose of 200 mg twice daily (bid). A Phase I, randomized, participant-blinded, placebo-controlled study (A2109) is being conducted to assess the safety, pharmacokinetics and pharma

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Zhang, Fengkui, Li Zhang, Chen Yang, et al. "KP104, a Bifunctional C5 Mab-Factor H Fusion Protein, Demonstrates Sustained Long-Term Efficacy and Safety in Complement Inhibitor-Naïve PNH Patients: Updated Results from a Phase 2 Study at 36/38 Weeks of Obd Treatment." Blood 144, Supplement 1 (2024): 2699. https://doi.org/10.1182/blood-2024-210086.

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Background: Paroxysmal nocturnal hemoglobinuria (PNH) is a rare and life-threatening hematologic condition marked by intravascular and extravascular hemolysis (IVH and EVH), driven by the complement terminal and proximal effectors of the alternative pathway, respectively. KP104, a novel bifunctional recombinant fusion protein comprised of a humanized anti-C5 mAb and the functional domain of complement regulator factor H, can simultaneously inhibit both terminal and proximal complement activations. We previously reported data from an ongoing Phase II study (NCT05476887) involving 18 complement

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Peffault De Latour, Regis, Austin G. Kulasekararaj, Phillip Scheinberg, et al. "The Effect of Oral Iptacopan Monotherapy on Hematological Parameters in Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) Is Consistent Regardless of the Type of Prior Anti-C5 Treatment Received: A Post Hoc Analysis of 24-Week Data from the Randomized Phase III APPLY-PNH Trial." Blood 144, Supplement 1 (2024): 4087. https://doi.org/10.1182/blood-2024-200763.

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Background: Iptacopan, an oral proximal complement inhibitor that targets factor B, is approved as a monotherapy for the treatment of adults with PNH based on the results of 2 Phase III trials (APPLY-PNH [NCT04558918] and APPOINT-PNH [NCT04820530]). APPLY-PNH enrolled patients with PNH that were receiving either intravenous eculizumab or ravulizumab. Both treatments inhibit C5; however, ravulizumab has an extended half-life, allowing dosing every 8 weeks compared with eculizumab, which is dosed every 2 weeks. In APPLY-PNH, 24 weeks of iptacopan monotherapy was superior to anti-C5 treatment acr

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Peffault de Latour, Regis, Alexander Roeth, Austin Kulasekararaj, et al. "Oral Monotherapy with Iptacopan, a Proximal Complement Inhibitor of Factor B, Has Superior Efficacy to Intravenous Terminal Complement Inhibition with Standard of Care Eculizumab or Ravulizumab and Favorable Safety in Patients with Paroxysmal Nocturnal Hemoglobinuria and Residual Anemia: Results from the Randomized, Active-Comparator-Controlled, Open-Label, Multicenter, Phase III Apply-PNH Study." Blood 140, Supplement 2 (2022): LBA—2—LBA—2. http://dx.doi.org/10.1182/blood-2022-171469.

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Morado-Arias, Marta, Monica Ballesteros, Miguel Gómez-Álvarez, Federico Herrera, and Ana Villegas. "Response to Pegcetacoplan in Patients with PNH Assessed By Erythroid Clonal Size and Laboratory Markers." Blood 144, Supplement 1 (2024): 5679. https://doi.org/10.1182/blood-2024-203498.

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Introduction: Pegcetacoplan (PEG) is a proximal complement inhibitor used to treat patients with paroxysmal nocturnal hemoglobinuria (PNH). By blocking the complement at C3 level, it inhibits intravascular hemolysis without inducing extravascular hemolysis due to C3 deposition, a typical side effect of C5 inhibitors (C5i). This mechanism of action explains its greater effectiveness in controlling anemia compared to terminal inhibitors. Response criteria to PEG can be assessed using laboratory markers of hemolysis as well as erythroid clonal size, which increases to achieve the same size as in

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Karnabeda, Oksana, Valentyn Moskalenko, Zoreslava Lysak, et al. "OMS906, a Novel Alternative Pathway MASP-3 Inhibitor, Normalizes Hemoglobin Levels and Increases Clone Size in Treatment-Naïve PNH Patients." Blood 142, Supplement 1 (2023): 573. http://dx.doi.org/10.1182/blood-2023-177921.

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Background: Paroxysmal nocturnal hemoglobinuria (PNH) is a life-threatening disorder characterized by hemolytic anemia in combination with various degrees of marrow failure. PNH is driven by dysregulation of the complement system where the absence of CD55 and CD59 cell surface proteins leads to red blood cell (RBC) lysis. Untreated PNH can lead to debilitating anemia, thrombosis, fatigue, and increased mortality. Terminal complement inhibition attenuates intravascular hemolysis (IVH) but inevitably leads to extravascular hemolysis (EVH). Proximal and alternative pathway (AP) inhibition block b

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Risitano, Antonio M., Austin Kulasekararaj, Alexander Roeth, et al. "Factor B Inhibition with Oral Iptacopan Monotherapy Demonstrates Sustained Long-Term Efficacy and Safety in Anti-C5-Treated Patients (pts) with Paroxysmal Nocturnal Hemoglobinuria (PNH) and Persistent Anemia: Final 48-Week Results from the Multicenter, Phase III APPLY-PNH Trial." Blood 142, Supplement 1 (2023): 571. http://dx.doi.org/10.1182/blood-2023-180780.

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Drs Roeth and Kulasekararaj contributed equally as authors. Background: Iptacopan is the first oral complement inhibitor that acts proximally in the complement system to target factor B in the alternative pathway. Iptacopan has shown efficacy/safety in PNH pts with persistent anemia despite anti-C5 therapy and complement inhibitor-naive pts. In the Phase III APPLY-PNH trial (NCT04558918), iptacopan monotherapy led to clinically meaningful hemoglobin (Hb) increases and normal/near-normal Hb levels in a majority of pts, transfusion avoidance and improved pt-reported fatigue, showing superiority

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Risitano, Antonio M., Bing Han, Austin Kulasekararaj, et al. "Categorization of Hematological Responses to Oral Iptacopan Monotherapy in Anti-C5-Treated Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) and Persistent Anemia in the Phase III APPLY-PNH Trial and Complement Inhibitor-Naïve Patients in the Phase III APPOINT-PNH Trial." Blood 142, Supplement 1 (2023): 4084. http://dx.doi.org/10.1182/blood-2023-180866.

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Background: PNH is an ultrarare disease characterized by complement-mediated hemolysis and consequent anemia. Iptacopan is the first oral, selective complement inhibitor that targets factor B to inhibit the alternative pathway proximally in the complement system. Iptacopan monotherapy led to normal/near normal hemoglobin (Hb) values and transfusion avoidance in the majority of complement inhibitor-naïve patients and anti-C5-treated patients with persistent anemia in the Phase III APPOINT-PNH (NCT04820530) and APPLY-PNH (NCT04558918) trials, respectively, with iptacopan achieving its primary en

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Panse, Jens Peter, Britta Höchsmann, and Jörg Schubert. "Paroxysmal Nocturnal Hemoglobinuria, Pathophysiology, Diagnostics, and Treatment." Transfusion Medicine and Hemotherapy, August 21, 2024, 310–20. http://dx.doi.org/10.1159/000540474.

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Background: Paroxysmal nocturnal hemoglobinuria (PNH) is characterized by intravascular hemolysis (IVH) due to diminished or absent inhibition of the complement system because of deficient expression of cell-anchored complement regulating surface proteins. IVH leads to heterogeneous symptoms such as anemia, abdominal pain, dyspnea, fatigue and increased rates of thrombophilia. Inhibitors of the terminal Complement cascade can reverse IVH leading to a significant reduction of disease burden such as thrombembolic events and also mortality. Summary: Therapeutic inhibitors of the terminal compleme

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Schmidt, Christoph Q., Britta Höchsmann, and Hubert Schrezenmeier. "The complement model disease paroxysmal nocturnal hemoglobinuria." European Journal of Immunology, August 5, 2024. http://dx.doi.org/10.1002/eji.202350817.

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AbstractWe describe initial, current, and future aspects of complement activation and inhibition in the rare hematological disease paroxysmal nocturnal hemoglobinuria (PNH). PNH is a rare but severe hematological disorder characterized by complement‐mediated intravascular hemolysis resulting in anemia and severe thrombosis. Insights into the complement‐mediated pathophysiology ultimately led to regulatory approval of the first‐in‐class complement inhibitor, eculizumab, in 2007. This anti‐complement C5 therapy resulted in the stabilization of many hematologic parameters and dramatically reduced

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Graf, Michael, and Vincent S. Gallicchio. "History, Etiology, and Treatment of Paroxysmal Nocturnal Hemoglobinuria." Trends in Internal Medicine 2, no. 1 (2022). http://dx.doi.org/10.33425/2771-5906.1015.

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Paroxysmal nocturnal hemoglobinuria is an acquired, rare, nonmalignant hematological disease that is characterized by uncontrolled complement activation and thus intravascular hemolysis. It is caused by somatic mutations in the PIG-A gene that leads to a deficiency in necessary GPI-anchored proteins, which leads to uncontrolled complement activation and thus the clinical manifestations that PNH is often associated with. It is generally accepted that Dr. Paul Strübing first described the disease in 1882, albeit it was Thomas Hale Ham who designed the first diagnostic test (Ham test or acidified

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Risitano, Antonio M., Austin G. Kulasekararaj, Jong Wook Lee, et al. "Danicopan: an oral complement factor D inhibitor for paroxysmal nocturnal hemoglobinuria." Haematologica, October 29, 2020, 0. http://dx.doi.org/10.3324/haematol.2020.261826.

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Paroxysmal nocturnal hemoglobinuria (PNH) is characterised by complement-mediated intravascular hemolysis (IVH) due to absence of complement regulators CD55 and CD59 on affected erythrocytes. Danicopan is a first-in-class oral proximal, complement alternative pathway factor D (FD) inhibitor. Therapeutic FD inhibition was designed to control IVH and prevent C3-mediated extravascular hemolysis (EVH). In this open-label, phase 2, dose-finding trial, 10 untreated hemolytic PNH patients received danicopan monotherapy (100-200 mg thrice daily). Endpoints included change in lactate dehydrogenase (LDH

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Kawahara, Hiroshi, Nobuhide Watanabe, Akihiro Endo, Hiroyuki Yoshitomi, and Kazuaki Tanabe. "Subacute stent thrombosis with spontaneously resolved secondary thrombi in paroxysmal nocturnal hemoglobinuria: a case report." BMC Cardiovascular Disorders 22, no. 1 (2022). http://dx.doi.org/10.1186/s12872-022-02850-z.

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Abstract Background Stent thrombosis (ST) is a serious complication; however, a method to prevent ST in patients with thrombophilic diseases has not been established. Case presentation We report a case of subacute ST in a patient with paroxysmal nocturnal hemoglobinuria (PNH) who was receiving continuous heparin treatment in addition to the usual dual antiplatelet therapy for contrast defects at the proximal site of the occluded right coronary artery and the proximal site of the left circumflex artery. Despite the resolution of thrombi in secondary lesions, subacute ST occurred. After percutan

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White, Taylor S., Justin R. Arnall, Paul Christopher Parish, Jamie Tolerico, Thuy Tran, and Donald C. Moore. "Proximal complement inhibitors in paroxysmal nocturnal hemoglobinuria: an abundance of options in a rare disease." Expert Review of Hematology, January 6, 2025, 1–5. https://doi.org/10.1080/17474086.2025.2449864.

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Ussowicz, Marek, Dawid Przystupski, Patrycja Mensah-Glanowska, and Agnieszka Piekarska. "Current status and perspectives of hematopoietic cell transplantation in patients with paroxysmal nocturnal hemoglobinuria." Frontiers in Immunology 15 (January 7, 2025). https://doi.org/10.3389/fimmu.2024.1521484.

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BackgroundParoxysmal nocturnal hemoglobinuria (PNH) is a rare complement-driven acquired hemolytic anemia with specific presentations of hemoglobinuria, abdominal pain, fatigue, and thrombosis.ObjectiveTo review the current therapeutic strategies for PNH, including anti-complement therapy and allogeneic hematopoietic cell transplantation (alloHCT), focusing on the tailoring of the approach to the disease subtype.ResultsThe outcome of alloHCT varies depending on disease severity, thrombotic history, and response to prior therapies. Non-transplant PNH therapies include anti-C5 monoclonal antibod

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Hakimi, Zalmai, Koo Wilson, Eoin McAughey, et al. "The cost–effectiveness, of pegcetacoplan compared with ravulizumab for the treatment of paroxysmal nocturnal hemoglobinuria, in a UK setting." Journal of Comparative Effectiveness Research, July 7, 2022, 00. http://dx.doi.org/10.2217/cer-2022-0076.

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Aim: Paroxysmal nocturnal hemoglobinuria (PNH) is a rare blood disorder characterized by hemolytic anemia, bone marrow failure and thrombosis. We evaluated, the cost–effectiveness of pegcetacoplan, a novel proximal C3 inhibitor, versus ravulizumab in patients with PNH and hemoglobin levels <10.5 g/dl despite eculizumab treatment in the UK healthcare and social services setting. Materials & methods: A Markov cohort framework model, based on the data from the pivotal trial of pegcetacoplan (PEGASUS/NCT03500549), evaluated lifetime costs and outcomes. Patients transitioned through 3 PNH he

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Fattizzo, Bruno, Francesco Versino, and Wilma Barcellini. "Breakthrough hemolysis in paroxysmal nocturnal hemoglobinuria throughout clinical trials: from definition to clinical practice." Blood Journal, April 15, 2025. https://doi.org/10.1182/blood.2024027574.

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Breakthrough hemolysis (BTH) is the hemolytic exacerbation occurring in a patient with paroxysmal nocturnal hemoglobinuria (PNH) on treatment with anti-complement therapies. In this review article we analysed the definition, frequency and severity of BTH events across phase 3 clinical trials with terminal (anti-C5 ravulizumab and crovalimab) and complement inhibitors upstream C5 (anti-C3 pegcetacoplan, alternative-pathway inhibitors iptacopan and danicopan), as well as from real-world reports. Furthermore, we reviewed the impact of the various compounds on quality of life and patients reported

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Kulesekararaj, Austin, Antonio Maria Risitano, Jaroslaw P. Maciejewski, et al. "Phase 2 Study of Danicopan in Paroxysmal Nocturnal Hemoglobinuria Patients with an Inadequate Response to Eculizumab." Blood, July 27, 2021. http://dx.doi.org/10.1182/blood.2021011388.

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Paroxysmal nocturnal hemoglobinuria (PNH) is characterised by uncontrolled terminal complement activation and subsequent intravascular hemolysis (IVH). C5 inhibitors prevent membrane attack complex formation, but patients may experience extravascular hemolysis (EVH) and continue to require blood transfusions. Danicopan, an oral, proximal complement inhibitor of alternative pathway factor D (FD), is designed to control IVH and EVH. In a Phase 2, dose-finding trial, eculizumab-treated, transfusion-dependent PNH patients (n=12) received danicopan 100-200 mg thrice daily in addition to their eculi

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Kato, Yuta, Mitsuyoshi Hadase, and Takashi Nakamura. "Recurrent acute myocardial and renal infarction with aplastic anemia/paroxysmal nocturnal hemoglobinuria syndrome: A case report." European Heart Journal - Case Reports, September 23, 2024. http://dx.doi.org/10.1093/ehjcr/ytae526.

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Abstract Background Aplastic anemia (AA) is known to progress to paroxysmal nocturnal hemoglobinuria (PNH) during treatment, and thrombosis is a characteristic symptom of PNH. This case report investigates a case of repeated and rapidly progressive multiple arterial thrombosis due to PNH. Case Summary This case is a 24-year-old woman undergoing treatment for AA. She presented with chest pain and underwent emergency coronary angiography. Thrombus occlusion was found in the distal portion of the right coronary artery, acute myocardial infarction was diagnosed and percutaneous coronary interventi

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Fishman, Jesse, Koo Wilson, Aleksandra Drzewiecka, Michał Pochopień, and David Dingli. "The cost–effectiveness of pegcetacoplan in complement treatment-naïve adults with paroxysmal nocturnal hemoglobinuria in the USA." Journal of Comparative Effectiveness Research, August 31, 2023. http://dx.doi.org/10.57264/cer-2023-0055.

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Aim: Paroxysmal nocturnal hemoglobinuria (PNH) is a rare blood disorder characterized by hemolytic anemia, bone marrow failure and thrombosis, and is associated with high healthcare burden. We evaluated the cost–effectiveness of pegcetacoplan, a proximal complement-3 inhibitor (C3i), compared with the C5i, eculizumab and ravulizumab, in complement treatment-naive adults with PNH, from the US healthcare payer perspective. Materials & methods: A de novo cost–effectiveness model based on a Markov cohort structure evaluated lifetime (55-year) PNH costs and outcomes. The 6-month cycles of the m

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Füreder, Wolfgang, Renate Thalhammer, and Peter Valent. "Resolution of extravascular hemolysis with oral iptacopan monotherapy in a patient with treatment experienced paroxysmal nocturnal hemoglobinuria (PNH)." Wiener klinische Wochenschrift, July 1, 2024. http://dx.doi.org/10.1007/s00508-024-02390-w.

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SummaryParoxysmal nocturnal hemoglobinuria (PNH) is an acquired hematologic disorder characterized by a loss of glycosyl-phosphatidyl-inositol-linked (GPI) proteins on various hematopoietic cells. Some GPI proteins are involved in the regulation of the complement system, and their absence renders erythrocytes susceptible to complement-mediated lysis. Current standard of care in PNH is to block the complement system at the level of C5 using ravulizumab or eculizumab; however, some patients with PNH may develop extravascular hemolysis (EVH) during treatment with C5 inhibitors. The proximal compl

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Risitano, Antonio M., Serena Marotta, Patrizia Ricci, et al. "Anti-complement Treatment for Paroxysmal Nocturnal Hemoglobinuria: Time for Proximal Complement Inhibition? A Position Paper From the SAAWP of the EBMT." Frontiers in Immunology 10 (June 14, 2019). http://dx.doi.org/10.3389/fimmu.2019.01157.

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Kulasekararaj, Austin G., David J. Kuter, Morag Griffin, Ilene C. Weitz, and Alexander Röth. "Biomarkers and laboratory assessments for monitoring the treatment of patients with paroxysmal nocturnal hemoglobinuria: Differences between terminal and proximal complement inhibition." Blood Reviews, January 2023, 101041. http://dx.doi.org/10.1016/j.blre.2023.101041.

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