Polskie Archiwum Medycyny Wewnętrznej
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Pol. Arch. Med. Wewn. · Jun 2022
ReviewParoxysmal nocturnal hemoglobinuria: advances in the understanding of pathophysiology, diagnosis, and treatment.
In recent years, "old" paroxysmal nocturnal hemoglobinuria (PNH) has achieved new advances in terms of the understanding of its pathophysiology, modern approach to diagnostics, optimization of therapy, and dynamic development of new therapeutic agents. This review emphasizes the greater than previously recognized importance of the reduced susceptibility of PNH stem cells to apoptosis in the selection of a defective clone. Some changes in cytokine and chemokine profiles in patients with PNH have been interpreted in the context of autoimmunity and apoptosis. ⋯ Determinants of modern treatment, such as strategies (complement C5 inhibitors vs hematopoietic stem cell allotransplantation), the safety and efficacy of treatment with eculizumab or ravulizumab, policy of initiation and monitoring of treatment, the criteria for response to treatment and final outcomes of treatment are described. Among the new therapeutic agents, crovalimab and C5 inhibitors at a less advanced stage of research are discussed: tesidolumab, pozelimab, zilucoplan, nomacopan, and cemdisiran. The first approved proximal complement pathway inhibitors that primarily prevent extravascular hemolysis, pegcetacoplan, danikopan, and iptacopan, are presented and their potential benefits are highlighted.
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Pol. Arch. Med. Wewn. · Jun 2022
Recent advances in the diagnosis and therapy of large vessel vasculitis.
Large vessel vasculitis (LVV), including Takayasu arteritis (TAK) and giant cell arteritis (GCA), causes granulomatous vascular inflammation mainly in large vessels, and is the most common primary vasculitis in adults. Vascular inflammation may evoke many clinical features including vision impairment, stroke, limb ischemia, and aortic aneurysms. The best way to diagnose LVV is to combine medical history, physical examination, various laboratory tests, and imaging modalities. ⋯ Tumor necrosis factor inhibitors are ineffective in GCA but effective in TAK. On the other hand, tocilizumab may be used to treat both diseases. Promising targeted therapies evaluated in ongoing clinical trials include, for example, anti‑IL‑12/23 (ustekinumab), anti‑IL‑17 (secukinumab), anti‑IL‑1 (anakinra), anti‑IL‑23 (guselkumab), anti‑cytotoxic T‑lymphocyte antigen 4 (abatacept), Janus kinase inhibitors (tofacitinib and upadacitinib), anti‑granulocyte / macrophage colony‑stimulating factor (mavrilimumab), and endothelin receptor (bosentan) therapies.
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Acromegaly is a chronic, slowly progressive disorder caused mostly by growth hormone (GH)-producing pituitary neuroendocrine tumors (PitNETs). Recently, the associations between sex and age at the time of diagnosis and the course of acromegaly have been a focus of debate. ⋯ According to our results, hypogonadism and greater IGF‑1 values were more frequently observed in men with acromegaly. Hyperprolactinemia, hypogonadism, and macroadenoma were more frequent in patients with acromegaly aged 40 years or younger.
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Pol. Arch. Med. Wewn. · Jun 2022
Observational StudyPrognostic value of acid-base balance parameters obtained from peripheral venous blood sample on admission in patients with myocardial infarction treated with percutaneous coronary intervention.
Peripheral venous blood sample may be used to obtain acid‑ base balance parameters (PVA‑BP) measured in rapid point‑of‑care test (POCT) analyzers on admission to an emergency department (ED). Thus, lactates, anion gap (AG), and base excess (BE) may be early prognostic markers in patients with myocardial infarction (MI). ⋯ In the patients admitted to the ED with MI treated with PCI the evaluation of PVA‑BP in POCT analyzers may be a reliable tool for early risk stratification.