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Eculizumab in thymoma-associated myasthenia gravis: a real-world cohort study.
Ther Adv Neurol Disord
December 2024
Huashan Rare Disease Center and Department of Neurology, Huashan Hospital, Shanghai Medical College, National Center for Neurological Disorders, Fudan University, No.12 Urumqi Middle Road, Jing 'an District, Shanghai 200040, China.
Background: Thymoma-associated myasthenia gravis (TAMG) is a subtype of myasthenia gravis (MG) that is associated with more severe symptoms and a relatively poor prognosis. Eculizumab, an inhibitor to target human C5 component of the complement cascade, is considered a treatment option for refractory generalized MG (gMG).
Objectives: To explore the safety and efficacy of eculizumab in patients with TAMG.
Catastrophic antiphospholipid syndrome: a CAPS-tivating hematologic disease.
Hematology Am Soc Hematol Educ Program
December 2024
Department of Medicine, McMaster University, Hamilton, Ontario, Canada.
Catastrophic antiphospholipid syndrome (CAPS) is a rare but life-threatening form of antiphospholipid syndrome (APS) defined by the rapid onset of large and small vessel thrombosis occurring simultaneously across multiple sites, resulting in multiorgan dysfunction. The presence of underlying immune dysfunction causing activation of coagulation and, in many cases, abnormal complement regulation predisposes these patients to thrombotic events. CAPS is often preceded by triggering factors such as infection, surgery, trauma, anticoagulation discontinuation, and malignancy.
View Article and Find Full Text PDFSuccessful eculizumab treatment as an adjunctive therapy to desensitization in ABO-incompatible living donor kidney transplantation and its molecular phenotypes.
Front Immunol
November 2024
Department of Internal Medicine, College of Medicine, Yonsei University, Seoul, Republic of Korea.
Article Synopsis
- ABO-incompatible kidney transplantation (KT) is increasingly used to address organ shortages, but high levels of anti-ABO antibodies can hinder successful transplantation despite existing desensitization methods.
- Eculizumab was used as an additional treatment to help patients with very high anti-ABO antibodies who did not respond to standard therapies, showing improvements in kidney function and antibody levels after transplant.
- The study indicates that short-term eculizumab treatment may effectively support ABOi KT patients by maintaining stable kidney function and reducing antibody levels, even in cases previously deemed challenging.
Article Synopsis
- Antibody-mediated rejection (AMR) is a major cause of long-term kidney transplant failure, even with advancements in immunosuppressive therapies.
- * A case is described where late active AMR progressed to severe chronic active AMR, treated with a multidrug approach.
- * The current treatment options and understanding of AMR, including the role of donor-specific antibodies and various therapeutic strategies, highlight a need for improved guidelines based on stronger evidence.*
This unique case of generalized myasthenia gravis shows sustained stability of a patient's condition for 3 years with eculizumab/ravulizumab treatment following 16 years of refractory disease. It highlights the long-term effectiveness of C5 inhibitors in a real-world setting, aiding physicians in their decision-making for refractory cases and treatment discontinuation scenarios.
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