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Published on: September 5, 2016
Emerging Therapies in Antiphospholipid Syndrome
1Department of Cardiovascular and Metabolic Sciences, Taussig Cancer Institute, Lerner Research Institute, Cleveland Clinic, Cleveland, OH, USA.
Antiphospholipid syndrome (APS) treatments like hydroxychloroquine and rituximab show promise for managing symptoms and enhancing anticoagulation. Complement inhibition, particularly eculizumab, is effective for catastrophic APS and may benefit other forms.
Area of Science:
- Immunology
- Hematology
- Rheumatology
Background:
- Antiphospholipid syndrome (APS) is a leading cause of acquired immune-mediated thrombophilia, characterized by thrombosis or pregnancy complications with antiphospholipid antibodies.
- Despite over 50 years of recognition, the underlying pathogenesis of APS remains unclear, limiting mechanism-based therapies.
- Current APS management involves long-term anticoagulation or aspirin and heparin for obstetric issues, highlighting the need for novel treatments.
Approach:
- This review critically examines clinical studies of immunomodulatory agents, including hydroxychloroquine, rituximab, and eculizumab (a complement C5 inhibitor).
- The focus is on evaluating the efficacy and potential mechanisms of these agents in managing APS manifestations.
- The review synthesizes current evidence, acknowledging the limitations of small study sizes.
Key Points:
- Hydroxychloroquine may improve the effectiveness of vitamin K antagonists in APS patients.
- Rituximab shows potential in alleviating "non-criteria" manifestations of APS.
- Eculizumab is effective in catastrophic APS (CAPS), suggesting a significant role for complement activation.
Conclusions:
- While conclusions are qualified due to small study sizes, hydroxychloroquine and rituximab offer potential therapeutic benefits in APS.
- Eculizumab's efficacy in CAPS underscores the importance of the complement system in severe APS.
- Further research is needed to elucidate complement's role in non-catastrophic APS, with emerging data suggesting complement inhibition's potential for thrombosis prevention.
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