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Limited literature is available on stimulus induced after discharges (SIAD) in patients with peripheral nerve hyperexcitability (PNH). The aim of the study was to examine the diagnostic utility of SIAD in the diagnosis and monitoring of primary PNH disorders. In this retrospective study, we studied 26 patients who were admitted with a diagnosis of primary PNH to the department of Neurology from January 2013 to April 2019. Their clinical profile, immunological characteristics were extracted from the database and nerve conduction studies were relooked for the presence of SIAD. 76% of patients in the primary PNH cohort had SIAD with 90% of them being voltage-gated potassium channel complex antibody positive; predominantly against contactin-associated protein-2 antigen and rest being paraneoplastic. There was also resolution of SIAD following treatment indicating reversible hyperexcitability. SIAD is a sensitive marker for Primary PNH syndrome with monitoring and diagnostic implications.
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http://dx.doi.org/10.1111/jns.12422 | DOI Listing |
Ann Hematol
August 2025
U.O.C. di Ematologia, Ospedale di Ravenna e Università di Bologna, Ravenna, Italy.
The prevalence of paroxysmal nocturnal hemoglobinuria (PNH) clones is little investigated in myeloproliferative neoplasms (MPN) patients. The aim of this multicenter study was to evaluate the prevalence of PNH clones (glycosyl-phosphatidyl-inositol lacking) in 119 Ph- negative MPN patients having anemia, LDH elevation, asthenia and history of thrombosis. All the participating centers performed the standardized diagnostic test by using a single lyophilized template for granulocytes, monocytes, and erythrocytes.
View Article and Find Full Text PDFMedicina (Kaunas)
July 2025
Department of Nephrology, Hypertension, Transplantation and Internal Medicine, Central University Hospital, Medical University of Lodz, Pomorska 251, 92-213 Lodz, Poland.
: The field of nephrology is increasingly embracing advanced treatments and clinical trials that focus on inhibiting specific components of the complement cascade, a key driver in complement-mediated kidney diseases. : This review aims to summarize innovative therapies targeting various pathways, including the inhibition of the terminal part of the complement pathway (mainly C5), the alternative pathway (factor B inhibitors), and the lectin pathway (MASP inhibitors. C5 inhibitors play a critical role in preventing the formation of the membrane attack complex (MAC), offering effective solutions for conditions like atypical hemolytic uremic syndrome (aHUS) and paroxysmal nocturnal hemoglobinuria (PNH).
View Article and Find Full Text PDFBMJ Open
July 2025
Yardhura Walani, National Centre for Aboriginal and Torres Strait Islander Wellbeing Research, National Centre for Epidemiology and Population Health, The Australian National University, Canberra, ACT, Australia.
Introduction: The tobacco and nicotine industry fuels tobacco-related addiction, disease and death. Indigenous peoples experience a disproportionate burden of commercial tobacco-related morbidity and mortality. Over the past two decades, significant progress has been made in reducing smoking prevalence among Indigenous peoples; however, smoking remains a leading contributor to the burden of death and disease.
View Article and Find Full Text PDFBr J Haematol
August 2025
Chinese Academy of Medical Sciences, Peking Union Medical College Hospital, Beijing, China.
Paroxysmal nocturnal haemoglobinuria (PNH) is a rare, life-threatening disorder characterized by complement-mediated haemolysis, leading to anaemia and thrombosis. HRS-5965 is a novel, oral, selective complement factor B inhibitor targeting the alternative pathway, potentially reducing both intra- and extravascular haemolysis. In this randomized, open-label phase II study, we evaluated the efficacy and safety of HRS-5965 monotherapy in complement inhibitor-naïve adult PNH patients.
View Article and Find Full Text PDFPharm Res
July 2025
Small Molecule Research, Alexion, AstraZeneca Rare Disease, 100 College St, New Haven, 06510, CT, USA.
Objective: The goal of the study was to discover small molecular inhibitors of complement factor D (FD), an essential protease for activation of the alternative pathway (AP) of complement, that possess the characteristics for clinical investigation in complement-mediated diseases such as paroxysmal nocturnal hemoglobinuria (PNH).
Methods: Compounds were synthesized and tested in vitro for potency, selectivity, and metabolic stability. The optimized compounds were subjected further to a panel of in vitro tests for primary and secondary pharmacology including inhibitory effects on FD, different complement pathways and disease models, as well as to pharmacokinetic and pharmacodynamic evaluations in animals.