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
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)
Review
Hematology
Austin G. Kulasekararaj, Robert A. Brodsky, Jun-ichi Nishimura, Christopher J. Patriquin, Hubert Schrezenmeier
Summary: Paroxysmal nocturnal hemoglobinuria (PNH) is a rare blood disorder that can cause hemolysis, thrombosis, and organ damage. LDH is used as a biomarker for diagnosis. Eculizumab and ravulizumab, inhibitors of the terminal complement component 5 (C5), have shown effectiveness in treating PNH.
THERAPEUTIC ADVANCES IN HEMATOLOGY
(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)
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
Austin G. Kulasekararaj, Antonio M. Risitano, Jaroslaw P. Maciejewski, Rosario Notaro, Peter Browett, Jong Wook Lee, Mingjun Huang, Michael Geffner, Robert A. Brodsky
Summary: The study showed that adding an oral complement inhibitor danicopan to PNH patients who were dependent on eculizumab led to an increase in Hgb levels, a reduction in blood transfusion requirements, and improvements in fatigue, with good tolerability.
Article
Multidisciplinary Sciences
Patricia Eiko Yamakawa, Ana Rita Fonseca, Ismael Dale Cotrim Guerreiro da Silva, Matheus Vescovi Goncalves, Dirce Maria Marchioni, Antonio Augusto Ferreira Carioca, David Michonneau, Celso Arrais-Rodrigues
Summary: This study aimed to identify the dysfunctional pathways involved in the pathophysiology of PNH by comparing the metabolic profiles of PNH patients to healthy controls, as well as analyzing the metabolomic profiles before and after eculizumab treatment. The results showed significant differences in metabolomes between PNH patients and healthy controls, and eculizumab treatment appeared to improve defects in the Acyl CoA metabolism, reducing oxidative stress and inflammation.
Review
Immunology
Nicole Galli, Loredana Pettine, Mauro Panigada, Laura Daprai, Grazia Suriano, Anna Grancini, Wilma Barcellini, Bruno Fattizzo
Summary: This case report highlights the risk of life-threatening infection by non-groupable Neisseria meningitidis in a young patient with paroxysmal nocturnal haemoglobinuria (PNH) treated with ravulizumab. Prompt diagnosis and treatment are crucial in managing such infections in PNH patients on complement inhibitors.
FRONTIERS IN IMMUNOLOGY
(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)
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
Pharmacology & Pharmacy
Jun-ichi Nishimura, Antoine Soubret, Noriko Arase, Simon Buatois, Masaki Hotta, Jean-Eric Charoin, Yoshikazu Ito, Sasha Sreckovic, Hiroyuki Takamori, Christoph Bucher, Yasutaka Ueda, Jules Hernandez-Sanchez, Keisuke Gotanda, Gregor Jordan, Kenji Shinomiya, Julia Ramos, Jin Seok Kim, Jens Panse, Regis Peffault de Latour, Alexander Roeth, Eiichi Morii, Hubert Schrezenmeier, Yoshitaka Isaka, Simona Sica, Yuzuru Kanakura, Sung-Soo Yoon, Taroh Kinoshita, Ido Paz-Priel, Alexandre Sostelly
Summary: Drug-target-drug complexes (DTDCs) are newly observed phenomena in patients switching from eculizumab to crovalimab. Optimizing crovalimab dosing reduces the proportion of large DTDCs, ensures adequate complement inhibition, and may improve safety.
CLINICAL PHARMACOLOGY & THERAPEUTICS
(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)
Article
Hematology
Deniz Goren Sahin, Olga Meltem Akay, Muzaffer Keklik, Vahap Okan, Abdullah Karakus, Cengiz Demir, Mehmet Ali Erkurt, Kadir Ilkkilic, Rahsan Yildirim, Gulsum Akgun Cagliyan, Salih Aksu, Mehmet Hilmi Dogu, Mehmet Sinan Dal, Volkan Karakus, Ali Ihsan Gemici, Hatice Terzi, Engin Kelkitli, Serdar Sivgin, Ali Unal, Mehmet Yilmaz, Orhan Ayyildiz, Serdal Korkmaz, Bulent Eser, Fevzi Altuntas
Summary: This study aimed to collect PNH patient data from hematology centers across Turkey to identify clinical features and management. After evaluating patients from 19 different institutions, it was found that fatigue and abdominal pain were the most frequent presenting symptoms. This study provided valuable information for understanding the disease and differences between patients in Turkey and existing literature.
ANNALS OF HEMATOLOGY
(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
Rheumatology
Panagiotis Dolgyras, Antonios Lazaridis, Panagiota Anyfanti, Eleni Gavriilaki, Nikolaos Koletsos, Areti Triantafyllou, Barbara Nikolaidou, Vasiliki Galanapoulou, Stella Douma, Eugenia Gkaliagkousi
Summary: This study examined the microcirculation dynamics in patients with systemic vasculitides (SVs) and found that they exhibited impaired microvascular function and blunted reactivity after occlusion. Microvascular damage was correlated with disease duration. The findings suggest that skin microcirculation may be a useful, non-invasive method for early detection of microvascular dysfunction in SVs patients.
Letter
Biophysics
Raffaella Greco, Jorinde D. Hoogenboom, Edouard F. Bonneville, Achilles Anagnostopoulos, Angela Cuoghi, Jean-Hugues Dalle, Eva M. Weissinger, Peter Lang, Federica Galaverna, Massimo Martino, Alexei Maschan, Christine Mauz-Korholz, Maddalena Noviello, Jakob Passweg, Jacopo Peccatori, Montserrat Rovira, Carlos Solano, Hendrik Veelken, Andrea Velardi, Eva Maria Wagner-Drouet, Xi Zhang, Fabio Ciceri, Chiara Bonini, Luca Vago, Annalisa Ruggeri, Christian Chabannon
BONE MARROW TRANSPLANTATION
(2023)
Review
Oncology
Aikaterini Kosmidou, Athanasios Tragiannidis, Eleni Gavriilaki
Summary: Myeloid Leukemia of Down Syndrome (ML-DS) is a unique subtype of acute myeloid leukemias associated with Down Syndrome, characterized by distinct clinical and molecular features. In ML-DS, acquired mutations in the GATA1 gene on chromosome 21 play a crucial role in selective growth advantage of leukemic cells. Standardized holistic care and risk-adapted treatment protocols are necessary for improved outcomes.
Editorial Material
Medicine, General & Internal
Christos Varelas, Eleni Gavriilaki
JOURNAL OF CLINICAL MEDICINE
(2023)
Article
Oncology
Eleni Gavriilaki, Panagiotis Dolgyras, Sotiria Dimou-Mpesikli, Aikaterini Poulopoulou, Paschalis Evangelidis, Nikolaos Evangelidis, Christos Demosthenous, Evangelia Zachrou, Panagiotis Siasios, Despina Mallouri, Anna Vardi, Zoi Bousiou, Alkistis Panteliadou, Ioannis Batsis, Marianna Masmanidou, Chrysavgi Lalayanni, Evangelia Yannaki, Damianos Sotiropoulos, Achilles Anagnostopoulos, Timoleon-Achilleas Vyzantiadis, Ioanna Sakellari
Summary: This retrospective study investigated the prevalence and outcome of invasive fungal infections (IFD) in patients who received cellular therapies. It was found that CAR T cell and gene therapy recipients did not develop IFD, while autologous HCT recipients with primary refractory/relapsed lymphomas had probable IFD. Among allogeneic HCT recipients, 11 experienced probable IFD, 31 had possible IFD, and 10 had proven IFD. These findings suggest that IFD is associated with poor outcomes in allogeneic HCT recipients.
Article
Oncology
E. Gavriilaki, D. Mallouri, Z. Bousiou, C. Demosthenous, A. Vardi, P. Dolgyras, I. Batsis, E. Stroggyli, P. Karvouni, M. Masmanidou, M. Gavriilaki, A. Bouinta, S. Bitsianis, N. Kapravelos, M. Bitzani, G. Vasileiadou, E. Yannaki, D. Sotiropoulos, S. Papagiannopoulos, D. Kazis, V. Kimiskidis, A. Anagnostopoulos, I. Sakellari
Summary: Our real-world experience confirms that commercial CAR T therapy can be administered with minimal toxicity. Early referral of patients with low tumor burdens and close monitoring and early recognition of side effects are important in preventing major toxicities and potentially expanding the use of CAR T therapy.
Editorial Material
Oncology
Eleni Gavriilaki
FRONTIERS IN ONCOLOGY
(2023)
Meeting Abstract
Peripheral Vascular Disease
Panagiota Anyfanti, Antonia Dimitriadou, Eleni Gavriilaki, Areti Triantafyllou, Barbara Nikolaidou, Elena Angeloudi, Evdoxia Evaggeli Koravou, Theodoros Dimitroulas, Eugenia Gkaliagkousi
JOURNAL OF HYPERTENSION
(2023)
Meeting Abstract
Pediatrics
Antonia Kondou, Vasiliki Karava, John Dotis, Eleni Gavriilaki, Dimitrios Zafeiriou, Nikoleta Printza
PEDIATRIC NEPHROLOGY
(2023)
Review
Geriatrics & Gerontology
Maria Gavriilaki, Panagiota Anyfanti, Konstantinos Mastrogiannis, Eleni Gavriilaki, Antonios Lazaridis, Vasilios Kimiskidis, Eugenia Gkaliagkousi
Summary: The objective of this systematic review and meta-analysis is to investigate the association between nocturnal blood pressure fall and abnormal cognitive function. The analysis of 28 studies showed that individuals with non-dipping and reverse dipping blood pressure patterns have a higher risk of abnormal cognitive function and dementia. On the other hand, those with dipping blood pressure patterns have a lower risk of abnormal cognitive function and dementia.
AGING CLINICAL AND EXPERIMENTAL RESEARCH
(2023)
Article
Rheumatology
N. Koletsos, K. Dipla, A. Triantafyllou, A. Lazaridis, N. G. Papadopoulos, P. Dolgyras, E. Gavriilaki, P. Anyfanti, A. Zafeiridis, E. -E. Koravou, S. Aslanidis, S. Douma, E. Gkaliagkousi
Summary: This study used near-infrared spectroscopy (NIRS) to evaluate differences in cerebral oxygenation during exercise in systemic lupus erythematosus (SLE) patients compared to controls, and investigated possible underlying mechanisms through brain derived neurotrophic factor (BDNF) levels. The results showed that SLE patients, even without obvious neuropsychiatric manifestations, exhibited a blunted increase in cerebral oxygenation during submaximal exercise. Examining brain oxygenation during a simple exercise task may help identify patients with early cerebral function alterations.
CLINICAL AND EXPERIMENTAL RHEUMATOLOGY
(2023)
Review
Biochemistry & Molecular Biology
Paschalis Evangelidis, Theodora-Maria Venou, Barmpageorgopoulou Fani, Efthymia Vlachaki, Eleni Gavriilaki
Summary: Hemoglobinopathies, such as beta-thalassemia and sickle cell disease, are common genetic blood disorders that can cause endocrine disorders. Iron overload, oxidative stress, chronic anemia, and HCV infection contribute to endocrinopathies in beta-thalassemia, while vaso-occlusive events and microcirculation defects play a crucial role in endocrine dysfunction in SCD patients. These disorders have various manifestations, including diabetes mellitus, hypothyroidism, and adrenal insufficiency, impacting the quality of life of patients.
INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES
(2023)
Article
Biochemistry & Molecular Biology
Apostolia Papalexandri, Eleni Gavriilaki, Anna Vardi, Nikolaos Kotsiou, Christos Demosthenous, Natassa Constantinou, Tasoula Touloumenidou, Panagiota Zerva, Fotini Kika, Michalis Iskas, Ioannis Batsis, Despina Mallouri, Evangelia Yannaki, Achilles Anagnostopoulos, Ioanna Sakellari
Summary: This study investigated the occurrence of EBV-PTLD in HCT recipients and identified a cutoff point for predicting PTLD. The study also revealed the independent association of ATG and GVHD with lower survival rates and higher treatment-related mortality. The study emphasizes the importance of frequent and careful follow-ups and early intervention.
INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES
(2023)
Review
Peripheral Vascular Disease
Eleni Gavriilaki, Antonios Lazaridis, Panagiota Anyfanti, Efthalia Yiannaki, Panagiotis Dolgyras, Barbara Nikolaidou, Ioannis Vasileiadis, Maria Eleni Alexandrou, Anastasia Margouta, Dimitra Markala, Ioannis Zarifis, Panteleimon Sarafidis, Michail Doumas, Eugenia Gkaliagkousi
Summary: This study measured three types of microvesicles in patients with cardiovascular risk and compared them to patients with coronary artery disease and healthy individuals. The results showed that microvesicles are associated with increased cardiovascular risk and have high predictive value.
JOURNAL OF HUMAN HYPERTENSION
(2023)