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.
Review
Hematology
Raymond S. M. Wong
Summary: This article reviews multiple clinical studies on the efficacy and safety of pegcetacoplan treatment in adults with PNH. The studies found that pegcetacoplan showed superior improvements in hemoglobin levels and other hematologic parameters, and effectively managed anemia and anemia-related complications.
THERAPEUTIC ADVANCES IN HEMATOLOGY
(2022)
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.
Article
Medicine, General & Internal
Jin Seok Kim, Jun Ho Jang, Deog-Yeon Jo, Seo-Yeon Ahn, Sung-Soo Yoon, Je-Hwan Lee, Sung-Hyun Kim, Chul Won Choi, Ho-Jin Shin, Min-Kyoung Kim, Jae Hoon Lee, Yeung-Chul Mun, Jee Hyun Kong, BokJin Hyun, HyunSun Nam, Eunhye Kim, Min Joo Kwak, Yong Kyun Won, Jong Wook Lee
Summary: This study assessed the long-term efficacy and safety of eculizumab in Korean patients with paroxysmal nocturnal hemoglobinuria (PNH) using real-world data. The results showed that eculizumab had long-term efficacy and safety in Korean PNH patients with high disease burdens.
JOURNAL OF KOREAN MEDICAL SCIENCE
(2023)
Article
Medicine, Research & Experimental
Vivek Sharma, Jamie Koprivnikar, Kristen Drago, Jessica Savage, Allison Bachelor
Summary: This study evaluated the incidence and severity of injection site reactions (ISRs) in patients receiving long-term treatment with the targeted complement C3 therapy Pegcetacoplan. The results showed that most ISRs were mild events, patient compliance remained high, and no treatment discontinuations occurred due to ISRs, suggesting that ISRs do not pose a barrier to long-term Pegcetacoplan treatment.
ADVANCES IN THERAPY
(2023)
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
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
Biochemistry & Molecular Biology
Ioanna Lazana, Sean Apap Mangion, Selma Babiker, Joanna Large, Roochi Trikha, Mark Zuckerman, Shreyans Gandhi, Austin G. Kulasekararaj
Summary: Paroxysmal nocturnal hemoglobinuria (PNH) is a disease characterized by hemolysis and thrombosis, with a significant impact on morbidity and mortality. This study investigated the association between respiratory virus infections and breakthrough hemolysis (BTH) in PNH patients on eculizumab treatment. The results indicate that respiratory virus infections pose a significant risk for BTH in PNH patients and highlight the need for regular screening and close monitoring of patients with respiratory symptoms.
INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES
(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)
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
Hematology
Austin G. Kulasekararaj, Morag Griffin, Saskia Langemeijer, Kensuke Usuki, Alexander Kulagin, Masayo Ogawa, Ji Yu, Arshad Mujeebuddin, Jun-ichi Nishimura, Jong Wook Lee, Regis Peffault de Latour
Summary: This study describes the long-term follow-up results of over 400 patients with PNH treated with ravulizumab for 27 weeks to 2 years, showing durable efficacy and good tolerability, highlighting the importance of C5 inhibitors as the mainstay of PNH treatment.
EUROPEAN JOURNAL OF HAEMATOLOGY
(2022)
Article
Hematology
Raymond Siu Ming Wong, Juan Ramon Navarro-Cabrera, Narcisa Sonia Comia, Yeow Tee Goh, Henry Idrobo, Daolada Kongkabpan, David Gomez-Almaguer, Mohammed Al-Adhami, Temitayo Ajayi, Paulo Alvarenga, Jessica Savage, Pascal Deschatelets, Cedric Francois, Federico Grossi, Teresita Dumagay
Summary: PNH is a rare disease characterized by complement-mediated hemolysis. Pegcetacoplan, the first C3-targeted therapy, has shown superior efficacy and safety compared to supportive care in complement inhibitor-naive patients with PNH, leading to significant stabilization of hemoglobin levels and reduction in lactate dehydrogenase levels.
Article
Hematology
Honglei Wang, Hui Liu, Liyan Li, Yingying Chen, Zhaoyun Liu, Lijuan Li, Shaoxue Ding, Kai Ding, Rong Fu
Summary: By analyzing the samples of 35 PNH patients, it was found that lncRNA FAM157C may play a role in promoting clone proliferation in PNH.
ANNALS OF HEMATOLOGY
(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)
