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A Rare But Fatal Toxicity: Immune Checkpoint Inhibitor-Related Acquired Thrombotic Thrombocytopenic Purpura.
J Immunother Precis Oncol
February 2025
Section of Benign Hematology, The University of Texas MD Anderson Cancer Center, Houston, TX, USA.
Thrombotic thrombocytopenic purpura (TTP) is characterized by thrombotic microangiopathy resulting from decreased activation of the von Willebrand factor-cleaving protease (ADAMTS13). TTP can cause organ damage and is often fatal if the appropriate treatment is not started immediately. Although primary immune TTP is the most common form of TTP, secondary immune etiologies, including complications from immune checkpoint inhibitors (ICIs), have also been reported.
View Article and Find Full Text PDFIntroduction: Thrombotic microangiopathies (TMA) represent distinct pathological and clinical entities with known chronicity and recurrence. Kidney biopsy is the gold standard to diagnose TMA in patients with renal manifestations but the prognostic significance of acute or chronic phase of the disease has not been well studied. We examined the clinical characteristics, management, and predictors of acute vs.
View Article and Find Full Text PDFAn Unusual Case of Hemolytic Anemia: A Case Report.
Cureus
December 2024
Internal Medicine, Hospital Beatriz Ângelo, Lisboa, PRT.
Vitamin B12 deficiency is a potentially severe condition with clinical manifestations ranging from nonspecific symptoms, such as asthenia and glossitis, to severe hematological problems, including pancytopenia and megaloblastic anemia. One of the rare phenomena associated with this condition is pseudo-thrombotic microangiopathy (pseudo-TMA), which can mimic diseases such as thrombotic thrombocytopenic purpura (TTP), leading to possible misdiagnosis and inappropriate treatment. In this article, we present the case of a 62-year-old man with a history of intravenous drug use, untreated hepatitis C, smoking, and alcoholism.
View Article and Find Full Text PDFSafety and Efficacy of IV Onasemnogene Abeparvovec for Pediatric Patients With Spinal Muscular Atrophy: The Phase 3b SMART Study.
Neurology
January 2025
The Dubowitz Neuromuscular Centre, Developmental Neurosciences Department, University College London, Great Ormond Street Institute of Child Health, United Kingdom.
Background And Objectives: Safety and efficacy of IV onasemnogene abeparvovec has been demonstrated for patients with spinal muscular atrophy (SMA) weighing <8.5 kg. SMART was the first clinical trial to evaluate onasemnogene abeparvovec for participants weighing 8.
View Article and Find Full Text PDFManagement of Intraoperative Sickle Crisis During Kidney Transplant in a Patient With Sickle Cell Disease: A Case Report.
Cureus
December 2024
Surgery, George Washington University School of Medicine and Health Sciences, Washington, D.C., USA.
A 31-year-old male patient with a history of sickle cell disease (SCD) with stage V chronic kidney disease (CKD) presented for a deceased donor kidney transplant. During surgery, the transplanted kidney showed mottling and limited cortical flow, raising concerns for an intraoperative sickle cell crisis versus hyperacute rejection. Postoperative imaging revealed decreased vascularity, and the patient was treated with RBC exchange.
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