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We retrospectively analyzed a large international cohort of 1113 patients with aplastic anemia to evaluate treatment choice and outcome in elderly patients as compared with a younger population. Overall, 319 (29%) patients were > 60 years old at diagnosis (60-64 years (n = 85), 106 65-69 years (n = 106), and 128 > 70 years (n = 128)). Elderly patients showed a more severe thrombocytopenia at onset and a significantly lower overall response (complete plus partial) to first-line therapy at 6 months as compared to younger patients (47% vs.

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Aplastic anemia (AA) is a life-threatening bone marrow failure syndrome. The advent of next-generation sequencing (NGS) has shed light on the link between somatic mutations (SM) and the efficacy of immunosuppressive therapy (IST) in AA patients. However, the relationship between SM and hematopoietic stem cell transplantation (HSCT) has not been extensively explored.

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Gingival Debulking Surgery for a Child With Severe Aplastic Anemia: A Case Report.

Spec Care Dentist

January 2025

Paediatric Dentistry, The University of Western Australia, Dental School, Perth, Australia.

Introduction: Aplastic anemia (AA) is a rare condition that frequently manifests with pancytopenia. Management of severe disease is through either allogenic stem cell transplantation or immunosuppressive therapy with supportive care. Drug-induced gingival overgrowth (DIGO) is a potential complication of a number of medications, including cyclosporine and amlodipine.

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Patients' preferences are crucial to formulating personalized treatment plans. We developed a self-reported questionnaire, Therapy Preference Scale (TPS), to examine treatment preferences of patients with cancer. TPS has 30 questions-19 on patients' preferences on safety, quality of life, and treatment effectiveness, 8 questions on importance of various treatment characteristics, and 3 on patients' preferred intent of therapy, expenses, and life expectancy gain.

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Introduction: Eculizumab is a C5 complement inhibitor approved by the FDA for the targeted treatment of four rare diseases, paroxysmal nocturnal hemoglobinuria (PNH), atypical hemolytic uremic syndrome (aHUS), generalized myasthenia gravis (gMG), and aquaporin-4 immunoglobulin G-positive optic neuromyelitis optica spectrum disorders (AQP4-IgG+NMOSD). The current study was conducted to assess real-world adverse events (AEs) associated with eculizumab through data mining of the FDA Adverse Event Reporting System (FAERS).

Methods: Disproportionality analyses, including Reporting Ratio Ratio (ROR), Proportional Reporting Ratio (PRR), Bayesian Confidence Propagation Neural Network (BCPNN), and Multi-Item Gamma Poisson Shrinker (MGPS) algorithms were used to quantify the signals of eculizumab-associated AEs.

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