Review
Hematology
Jens Panse
Summary: In the past 20 years, therapy for paroxysmal nocturnal hemoglobinuria (PNH) mainly relied on antibody-based terminal complement inhibition. PNH is a disease characterized by a mutation that causes the absence or deficiency of complement-regulatory proteins on blood cells, leading to intravascular hemolysis and related complications. Recently, there has been a development of new drugs targeting the proximal and terminal complement cascade, with the approval of the first proximal complement inhibitor targeting C3 in 2021. This article aims to provide an overview of the progress made in PNH treatment and discuss the approved therapeutic options, as well as the potential impact and consequences of current and future treatments on patients' lives.
AMERICAN JOURNAL OF HEMATOLOGY
(2023)
Article
Hematology
Gloria F. Gerber, Robert A. Brodsky
Summary: This article discusses the theoretical basis and clinical studies of using C3 inhibitors in the treatment of PNH, as well as provides suggestions for treatment sequencing.
Article
Medicine, General & Internal
Peter Hillmen, Jeff Szer, Ilene Weitz, Alexander Roeth, Britta Hoechsmann, Jens Panse, Kensuke Usuki, Morag Griffin, Jean-Jacques Kiladjian, Carlos de Castro, Hisakazu Nishimori, Lisa Tan, Mohamed Hamdani, Pascal Deschatelets, Cedric Francois, Federico Grossi, Temitayo Ajayi, Antonio Risitano, Regis Peffault de la Tour
Summary: The study demonstrated that Pegcetacoplan was superior to eculizumab in improving hemoglobin and clinical and hematologic outcomes in patients with PNH by providing broad hemolysis control, including control of intravascular and extravascular hemolysis.
NEW ENGLAND JOURNAL OF MEDICINE
(2021)
Review
Hematology
Robert A. Brodsky
Summary: Paroxysmal nocturnal hemoglobinuria (PNH) is a rare complement-mediated hemolytic anemia with diverse manifestations, requiring differentiated treatment approaches; terminal complement inhibition is effective for intravascular hemolysis treatment but not bone marrow failure; novel complement inhibitors under clinical development show promising prospects for future applications.
Review
Immunology
Melissa A. Colden, Sushant Kumar, Bolormaa Munkhbileg, Daria V. Babushok
Summary: Paroxysmal Nocturnal Hemoglobinuria (PNH) is a disease that involves mutations in a specific gene, leading to hemolysis and abnormal clonal expansion of blood cells. The mechanisms behind this expansion are still debated, but recent advancements in research and technology offer new opportunities for understanding the disease.
FRONTIERS IN IMMUNOLOGY
(2022)
Review
Hematology
Austin G. Kulasekararaj, Ioanna Lazana
Summary: Paroxysmal nocturnal hemoglobinuria (PNH) is a rare hematological disorder characterized by deficiency of GPI-linked complement regulators. Despite the introduction of C5 inhibitors, residual hemolysis still occurs, leading to anemia and transfusion dependency in some patients. The development of longer-acting and subcutaneous formulations of C5 inhibitors, as well as proximal complement inhibitors, have shown promising results in improving hemoglobin levels and reducing hemolysis. Combination treatments have also been explored. This review discusses the current therapeutic options and emerging approaches for PNH.
AMERICAN JOURNAL OF HEMATOLOGY
(2023)
Article
Oncology
Eleni Gavriilaki, Athanasios Tragiannidis, Maria Papathanasiou, Sotiria Besikli, Paraskevi Karvouni, Vassiliki Douka, Eleni Paphianou, Emmanuel Hatzipantelis, Giorgos Papaioannou, Anastasia Athanasiadou, Anastasia Marvaki, Alkistis-Kira Panteliadou, Anna Vardi, Ioannis Batsis, Antonia Syrigou, Despina Mallouri, Chrysavgi Lalayanni, Ioanna Sakellari
Summary: This study aims to investigate the efficiency and safety of therapeutic methods for aplastic anemia and paroxysmal nocturnal hemoglobinuria. The study found that novel treatments are changing the field of BMF syndromes, but further research is needed to personalize algorithms.
FRONTIERS IN ONCOLOGY
(2022)
Article
Medicine, General & Internal
Laurence Schenone, Anne-Beatrice Notarantonio, Veronique Latger-Cannard, Veronique Fremeaux-Bacchi, Marcelo De Carvalho-Bittencourt, Marie-Therese Rubio, Marc Muller, Maud D'Aveni
Summary: Patients with paroxysmal nocturnal hemoglobinuria (PNH) may have a clonal population of blood cells deficient in GPI-anchored proteins due to mutations, such as in the PIGA or, as seen in this case, in the rare biallelic PIGT gene. This case report suggests that allo-SCT may be a potential curative treatment for PIGT-PNH patients, as seen in the successful outcome of the patient described.
WORLD JOURNAL OF CLINICAL CASES
(2022)
Review
Medicine, Research & Experimental
Bo Xu
Summary: Paroxysmal nocturnal hemoglobinuria (PNH) is a rare and life-threatening blood disease. The new targeted C3 therapy Empaveli (pegcetacoplan) has been approved by the US FDA for adult PNH patients. Pegcetacoplan targets C3 and effectively controls the complement cascade, resulting in improved hemoglobin levels and reduced need for blood transfusions.
CLINICAL AND EXPERIMENTAL MEDICINE
(2023)
Review
Medicine, General & Internal
Bruno Fattizzo, Fabio Serpenti, Juri Alessandro Giannotta, Wilma Barcellini
Summary: Paroxysmal nocturnal hemoglobinuria (PNH) is an intriguing disease with ongoing research on its pathophysiology, diagnostics, and treatment. Advanced flow cytometry techniques have enabled detection of small PNH clones, but data interpretation remains challenging. New complement inhibitors may improve patients' quality of life and response rates, but questions regarding their use and long-term safety need further investigation.
JOURNAL OF CLINICAL MEDICINE
(2021)
Article
Chemistry, Analytical
Kaan Batu Soysal, Seyma Parlatan, Metban Mastanzade, Murat Ozbalak, Mustafa Nuri Yenerel, Mehmet Burcin Unlu, Gunay Basar, Ugur Parlatan
Summary: The study proposed a combined approach using Raman spectroscopy and machine learning to analyze blood samples from volunteers with and without PNH conditions. Results showed a significant difference between the two groups, with a specificity of 81.8% and a sensitivity of 78.3% in the training performed by support vector machine (SVM) analysis. This method demonstrates the potential for immediate and high accuracy testing for PNH disease.
ANALYTICAL METHODS
(2021)
Review
Hematology
Yali Du, Bing Han
Summary: In countries where eculizumab is unavailable, patients with refractory AA/PNH and severe classical PNH are candidates for HSCT, with major causes of death from transplantation being GVHD, infection, and thrombotic microangiopathy. A haploidentical donor is a potential choice for patients without an HLA-matched donor. The combination of eculizumab and HSCT may help prevent GVHD.
TRANSPLANTATION AND CELLULAR THERAPY
(2021)
Review
Immunology
Chenyuan Li, Xifeng Dong, Huaquan Wang, Zonghong Shao
Summary: Paroxysmal nocturnal hemoglobinuria (PNH) is an acquired genetic mutation disease caused by defects in the PIG-A gene, leading to hemolysis of erythrocyte membranes.
FRONTIERS IN IMMUNOLOGY
(2021)
Article
Medicine, General & Internal
Runsen Du, Lihua Zheng, Peng Liu, Yaheng Zhao, Yan Yang, Lei Zhang, Zengren Zhao
Summary: We report a case of a female who was cured of hemorrhagic esophageal varices caused by paroxysmal nocturnal hemoglobinuria (PNH) through transjugular intrahepatic portosystemic shunt (TIPS) treatment. PNH complicated by portal vein and visceral veins thrombosis without hepatic veins is extremely rare, and it can be easily misdiagnosed due to lack of awareness. Hemorrhagic esophageal varices due to PNH with portal vein thrombosis have only been reported in one previous case in 1974, and this report presents the second case.
FRONTIERS IN MEDICINE
(2023)
Article
Pharmacology & Pharmacy
Sung-Eun Lee, Jong Wook Lee
Summary: This article reviews the safety data of eculizumab and ravulizumab in the treatment of PNH, especially in special settings. Expert opinion suggests that both drugs have been found to be safe and well tolerated in clinical trials, but vigilance towards meningococcal infections is still necessary.
EXPERT OPINION ON DRUG SAFETY
(2021)
