Review
Biology
Ge Tan, Katelyn M. Spillane, John Maher
Summary: The human NKG2D ligand family consists of eight stress-induced molecules. These ligands are expressed by over 80% of human cancers, but can also have immunosuppressive effects due to shedding, release via exosomes, and intracellular trapping. NKG2D deficiency in mice increases susceptibility to some types of cancer, indicating its role in immune surveillance for malignancy.
Article
Hematology
Ken Ishiyama, Yuji Yonemura, Tatsuya Kawaguchi, Kohei Hosokawa, Chiharu Sugimori, Yasutaka Ueda, Hiroyuki Takamori, Naoshi Obara, Hideyoshi Noji, Yukari Shirasugi, Kiyoshi Ando, Tsutomu Shichishima, Haruhiko Ninomiya, Shigeru Chiba, Jun-ichi Nishimura, Yuzuru Kanakura, Shinji Nakao
Summary: In this observational study, it was found that 52.6% of patients with acquired aplastic anemia (AA) and 13.7% of patients with myelodysplastic syndrome (MDS) had glycosylphosphatidylinositol-anchored protein-deficient (GPI[-]) cell populations (PNH-type cells). Furthermore, the presence of =1% PNH-type granulocytes was found to predict a higher likelihood of PNH-type cell expansion. This study provides valuable insights into the epidemiological characteristics and clinical significance of PNH-type cells in AA and MDS patients.
BRITISH JOURNAL OF HAEMATOLOGY
(2023)
Review
Pharmacology & Pharmacy
Young-A Heo
Summary: Some monkeys in the Asia-Pacific region have unique physiological characteristics that enable them to release oxygen during the night, enhancing their vitality and adaptation to dark environments.
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
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
Hematology
Alexander Roeth, Satoshi Ichikawa, Yoshikazu Ito, Jin Seok Kim, Zsolt Nagy, Naoshi Obara, Jens Panse, Hubert Schrezenmeier, Simona Sica, Juliette Soret, Kensuke Usuki, Sung-Soo Yoon, Nadiesh Balachandran, Muriel Buri, Pontus Lundberg, Himika Patel, Kenji Shinomiya, Alexandre Sostelly, Jun-ichi Nishimura
Summary: This study reports the long-term outcomes of crovalimab in patients with paroxysmal nocturnal haemoglobinuria. The results showed that crovalimab was well tolerated and achieved sustained C5 inhibition, with effective control of intravascular haemolysis, haemoglobin stabilization, and transfusion avoidance.
EUROPEAN JOURNAL OF HAEMATOLOGY
(2023)
Review
Hematology
Antonio Maria Risitano, Regis Peffault de Latour
Summary: Paroxysmal nocturnal haemoglobinuria (PNH) is a rare disease characterized by complement-mediated intravascular hemolysis, severe thrombophilia, and bone marrow failure. Treatment varies depending on the patient's condition, with the anti-C5 monoclonal antibody eculizumab revolutionizing treatment by controlling hemolysis and thrombotic risk effectively. New strategies of complement inhibition are emerging to improve patient outcomes.
BRITISH JOURNAL OF HAEMATOLOGY
(2022)
Article
Hematology
Bruno G. P. Pires da Silva, Natasha P. Fonseca, Luis Fernando B. Catto, Gabriel C. Pereira, Rodrigo T. Calado
Summary: This study retrospectively analyzed 87 cases of PNH in a Brazilian referral center and found that PNH presentation was variable, with most patients having subclinical disease or associated with bone marrow failure. The clone size remained stable or even disappeared in most cases.
ANNALS OF HEMATOLOGY
(2022)
Article
Hematology
Kohei Hosokawa, Shinji Nakao
Summary: Paroxysmal nocturnal hemoglobinuria (PNH) is an acquired hematopoietic stem cell disorder caused by a PIGA gene mutation. Clonal expansion of GPI(-) cells is common in PNH, but tiny GPI(-) cell populations can also be found in healthy individuals. In patients with acquired aplastic anemia, the expansion of PNH clones is related to immune attack on hematopoietic stem cells. However, the mechanisms underlying the selection and expansion of GPI(-) cells remain unclear.
SEMINARS IN HEMATOLOGY
(2022)
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
Oncology
Juri Alessandro Giannotta, Bruno Fattizzo, Wilma Barcellini
Summary: Paroxysmal nocturnal hemoglobinuria (PNH) is associated with aplastic anemia and myelodysplastic syndromes, with a prevalence of PNH clones in MPN patients around 10%, commonly associated with JAK2V617F-positive myelofibrosis. Thrombotic events are a common clinical presentation in this combination, sometimes refractory to treatment, and the use of eculizumab may only provide partial effectiveness in controlling hemolytic anemia, necessitating careful evaluation of risk/benefit in this peculiar setting.
FRONTIERS IN ONCOLOGY
(2021)
Letter
Genetics & Heredity
Sugat Adhikari, Surendra Sapkota, Suraj Shrestha, Kshitiz Karki, Anjan Shrestha
Summary: Paroxysmal nocturnal hemoglobinuria (PNH) is caused by a mutation in the phosphatidylinositol glycan class-A gene, resulting in uncontrolled complement activation and intravascular hemolysis. Eculizumab, a terminal complement inhibitor, has revolutionized the treatment of PNH but is expensive, posing challenges in low-middle income countries (LMICs) like Nepal. This article discusses potential approaches for PNH treatment in Nepal and other LMICs.
ORPHANET JOURNAL OF RARE DISEASES
(2023)
Article
Immunology
Andrea Kosta, Abdelilah Mekhloufi, Lorenzo Lucantonio, Alessandra Zingoni, Alessandra Soriani, Marco Cippitelli, Angela Gismondi, Francesca Fazio, Maria Teresa Petrucci, Angela Santoni, Helena Stabile, Cinzia Fionda
Summary: This study evaluated the role of the GAS6/TAM signaling pathway in the regulation of NKG2D ligand expression and NK cell recognition of MM. The results showed that GAS6, MERTK, and AXL depletion led to downregulation of MICA and inhibition of NKG2D-mediated NK cell degranulation. GAS6 derived from bone marrow stromal cells also increased MICA expression in MM cells.
FRONTIERS IN IMMUNOLOGY
(2022)
Article
Immunology
Xiaolong Wu, Amit Sharma, Johannes Oldenburg, Hans Weiher, Markus Essler, Dirk Skowasch, Ingo G. H. Schmidt-Wolf
Summary: CIK cells are a promising candidate for immunotherapy due to their convenient and relatively inexpensive expansion capability. NKG2D plays a crucial role in CIK cell-mediated antitumor activity, with NKG2D engagement alone being sufficient to activate CIK cells. The role of 2B4 in CIK cells is still unclear, but it provides limited synergy with NKG2D in certain aspects. Additional research is needed to optimize the functional potential of CIK cells for cancer therapy.
FRONTIERS IN IMMUNOLOGY
(2021)
Article
Hematology
Yali Du, Yuan Yang, Chen Yang, Miao Chen, Bing Han
Summary: Patients with classic PNH and BMF/PNH in China exhibit different clinical profiles, with classic PNH showing more hemolytic features and BMF/PNH patients being more prone to bleeding symptoms. There are differences in survival rate, PNH clone size, LDH level, ferritin level, renal impairment, and treatment modalities between the two groups. Less evolution to myeloid malignancies was observed in classic PNH than in BMF/PNH patients.