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
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)
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
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)
Article
Pharmacology & Pharmacy
Gaby A. M. Eliesen, Joris van Drongelen, Petra H. H. van den Broek, Andrei Sarlea, Olivier W. H. van der Heijden, Saskia Langemeijer, Rick Greupink, Elena B. Volokhina, Frans G. M. Russel
Summary: Eculizumab has been found to cross the placenta to some extent, with therapeutic drug levels observed in cord blood in a single case. In two pregnancies of a paroxysmal nocturnal haemoglobinuria patient receiving 900 mg eculizumab every 2 weeks, maternal, cord, and placental levels of unbound eculizumab, C5, and C5-eculizumab were measured. The levels of unbound eculizumab were higher than C5-eculizumab complexes in the placenta, suggesting selective transport of unbound eculizumab across the placenta.
BRITISH JOURNAL OF CLINICAL PHARMACOLOGY
(2021)
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)
Article
Hematology
Zlatko Pravdic, Mirjana Mitrovic, Andrija Bogdanovic, Marijana Virijevic, Nikica Sabljic, Nikola Pantic, Nada Suvajdzic Vukovic
Summary: Paroxysmal nocturnal haemoglobinuria (PNH) is a rare acquired hematological disease characterized by complement-mediated hemolysis, bone marrow failure, and venous thrombosis. Severe acute respiratory distress syndrome coronavirus 2 (SARS-CoV-2) disease 2019 (COVID-19) is associated with a high incidence of thrombosis in hospitalized pneumonia patients, with deep venous thrombosis (DVT) being a rare presenting symptom. This case describes a PNH patient on eculizumab for over 5 years who presented with DVT as the first sign of COVID-19.
Article
Public, Environmental & Occupational Health
Ilse Yolanda Osorio-Aragon, Sonia Toussaint-Caire, Simon Guzman-Bucio, Bibiana Monserrat Barbosa-Ramirez, Victor A. Vazquez-Aceituno, Juan Xicohtencatl-Cortes, Rigoberto Hernandez-Castro
Summary: We report a case of primary cutaneous mucormycosis in a patient with bone marrow failure due to paroxysmal nocturnal haemoglobinuria (PNH). The patient presented with papules on the lower limbs that rapidly progressed to necrotic plaques within 2 months. Histopathological examination showed granulomatous and suppurative dermatitis with tissue necrosis, and molecular identification confirmed Rhizopus arrhizus. Despite treatment with liposomal amphotericin B and surgical debridement, the patient developed severe anemia, thrombocytopenia, and septic shock, ultimately resulting in death after 6 days of hospitalization.
TROPICAL MEDICINE & INTERNATIONAL HEALTH
(2023)
Article
Pharmacology & Pharmacy
Mendy ter Avest, Saskia M. C. Langemeijer, Saskia E. M. Schols, David M. Burger, Nicole C. A. J. van de Kar, Nicole M. A. Blijlevens, Wietske Kievit, Rob ter Heine
Summary: By exploring alternative dosing regimens of ravulizumab, including extending the dosing interval to 10 weeks or even individualizing it to a mean of 12.8 weeks based on pharmacokinetic monitoring, noninferior efficacy in terms of lactate dehydrogenase normalization was achieved with significant drug cost savings up to 37%. This study demonstrates the potential of individualized dosing of ravulizumab to improve patient-friendliness while reducing costs.
BRITISH JOURNAL OF CLINICAL PHARMACOLOGY
(2021)
Article
Neurosciences
George Abraham Ninan, T. Angel Miraclin, Sujith Karumathil, John Davis Prasad, Sarath Kumar, Deepti Bal, Aditya Nair, Shaikh Atif Ahmed, Prabhakar Appaswamy Thirumal, Biju George, Ajith Sivadasan, Sanjith Aaron
Summary: Paroxysmal nocturnal haemoglobinuria (PNH) is a rare clonal hematopoietic disorder that leads to increased complement sensitivity of red blood cells, intravascular hemolysis, and vascular inflammation. Management of cerebrovascular emergencies in patients with PNH is complex.
JOURNAL OF STROKE & CEREBROVASCULAR DISEASES
(2023)
Article
Hematology
Ingvild Jenssen Laegreid, Thomas Wilson, Kristoffer Hjerto Naess, Siw Leiknes Ernstsen, Vibeke Schou, Mirjana Grujic Arsenovic
Summary: This study revealed a contraindication to minor incompatible whole blood transfusion and prompted a discussion on the distinction between whole blood and erythrocyte concentrates, highlighting the importance of these differences. It also drew attention to patient groups where minor incompatibility can have major consequences.
Article
Hematology
Bosi Alessandro, Barcellini Wilma, Fattizzo Bruno
Summary: Thrombosis risk in thrombocytopenic patients is complex, with COVID-19 infection adding additional risk. This case highlights challenges in managing thrombotic risk in cytopenic patients and the impact of COVID-19.
THROMBOSIS JOURNAL
(2022)
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)
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)
Article
Medicine, General & Internal
Arcangelo Iannuzzi, Antonio Parrella, Francesca De Ritis, Anna Cammarota, Lucia Berloco, Francesca Paudice, Giovanni D'Angelo, Emilio Aliberti, Gabriella Iannuzzo
Summary: A 78-year-old woman was diagnosed with Aplastic Anaemia associated with Paroxysmal Nocturnal Haemoglobinuria during an acute SARS-CoV-2 infection. The virus may have directly contributed to the disease, or altered the phosphatidylinositol glycan class A (PIGA) gene pathway.
MEDICINA-LITHUANIA
(2022)
