In three patients with march haemoglobinuria a well-defined protein abnormality has been revealed by high-resolution sodium-dodecyl-sulphate/polyacrylamide-gel electrophoresis.
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More Than a Haematoma: A Case of Aplastic Anemia.
Cureus
December 2024
Family Medicine, Family Health Unit (USF) Almedina, Local Health Unit of Trás-os-Montes and Alto Douro (ULSTMAD), Lamego, PRT.
Easy bruising and ecchymosis are common symptoms in clinical practice, yet distinguishing benign from clinically significant cases can be challenging. We report the case of a 46-year-old woman who presented in December 2023 with easy bruising and increased menstrual flow, revealing new-onset pancytopenia in laboratory tests. Initially diagnosed with Acute Myeloid Leukemia inversion (inv) (16), subsequent results were inconclusive, leading to a diagnosis of Paroxysmal Nocturnal Hemoglobinuria (PNH).
View Article and Find Full Text PDFOral complement factor D inhibitor danicopan for paroxysmal nocturnal hemoglobinuria.
Expert Rev Clin Pharmacol
September 2024
School of Pharmaceutical Science, Hengyang Medical School, University of South China, Hengyang, Hunan, China.
Article Synopsis
- Paroxysmal nocturnal hemoglobinuria (PNH) is a rare blood disorder leading to hemolysis, thrombosis, and bone marrow failure, with danicopan (Voydeya™) recently approved for its treatment.
- A systematic review of studies on danicopan showcased its efficacy in increasing hemoglobin levels in patients with PNH, particularly highlighted in a phase 3 trial where 60% of participants saw significant improvement compared to none in the placebo group.
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Crovalimab: First Approval.
Drugs
June 2024
Springer Nature, Private Bag 65901, Mairangi Bay, Auckland, 0754, New Zealand.
Article Synopsis
- Crovalimab, developed by Chugai Pharmaceutical and Roche, is a monoclonal antibody targeting complement component C5, aimed at treating various complement-mediated diseases like PNH and lupus nephritis.
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Activity and safety of eltrombopag in combination with cyclosporin A as first‑line treatment of adults with severe aplastic anaemia (SOAR): a phase 2, single-arm study.
Lancet Haematol
March 2024
Samsung Medical Center, Sungkyunkwan University School of Medicine, Seoul, South Korea.
Background: Antithymocyte globulin (ATG)-based immunosuppression is standard in front-line treatment for people with severe aplastic anaemia without a histocompatible donor or who are 40 years or older. However, ATG requires in-hospital administration, is associated with infusion-related toxicities and has limited availability worldwide. In this study, we investigated the activity and safety of an ATG-free regimen of eltrombopag with cyclosporin A as a potential treatment for patients with severe aplastic anaemia who might not have access to or cannot tolerate horse-ATG.
View Article and Find Full Text PDFThe Features of COVID-19's Course and the Efficacy of the Gam-COVID-Vac Vaccine in Patients with Paroxysmal Nocturnal Hemoglobinuria.
Hematol Rep
September 2023
Moscow City Hematology Center, S.P. Botkin City Clinical Hospital, Moscow 125284, Russia.
COVID-19 and other infectious diseases can exacerbate the course of paroxysmal nocturnal hemoglobinuria (PNH). The efficacy and safety of the Gam-COVID-Vac vaccine in patients with PNH has not been adequately studied. A retrospective, observational, cohort, non-comparative study was performed to assess the course of COVID-19 as well as the safety and efficacy of the Gam-COVID-Vac (Sputnik V) vaccine in patients with paroxysmal nocturnal hemoglobinuria (PNH).
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