Summary

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• Soliris (eculizumab) is indicated for the treatment of patients with paroxysmal nocturnal hemoglobinuria (PNH) to reduce hemolysis, the treatment of patients with atypical hemolytic uremic syndrome (aHUS) to inhibit complement-mediated thrombotic microangiopathy, the treatment of generalized myasthenia gravis (gMG) in adult patients who are anti-acetylcholine receptor (AchR) antibody positive, and the treatment of neuromyelitis optica spectrum disorder (NMOSD) in adult patients who are anti-aquaporin-4 (AQP4) antibody positive.
• This summary is based on the review of 12 systematic review(s)/meta-analysis(es). ^[1-12]
• Generalized Myasthenia Gravis (gMG): Eculizumab ranked second in effectiveness for reducing the Quantitative Myasthenia Gravis Score (SMD = -0.67; 95% CrI, -1.43, 0.01) compared to other treatments, being less effective than batoclimab (SMD = -1.61; 95% CrI, -2.78, -0.43) but more effective than zilucoplan (SMD = -0.54; 95% CrI, -1.56, 0.46) and nipoclimab (SMD = -0.02; 95% CrI, -1.04, 1.00).
• Atypical Hemolytic Uremic Syndrome (aHUS): Eculizumab and ravulizumab demonstrated comparable efficacy in treating aHUS, with patient preference leaning towards ravulizumab due to lower financial burden and less frequent dosing.
• Renal Transplant Patients with aHUS: Eculizumab significantly reduced the likelihood of aHUS recurrence (OR = 0.05; 95% CI: 0.00-0.13), decreased the need for dialysis (OR = 0.13; 95% CI: 0.01-0.32), and improved renal function.
• Neuromyelitis Optica Spectrum Disorder (NMOSD): Eculizumab was more effective in reducing on-trial relapse risk in AQP-4 positive patients compared to other monoclonal antibodies (Chi² = 9.84, P = 0.002).
• Generalized Myasthenia Gravis (gMG): Eculizumab had a risk ratio (RR) of 0.99 (95% CrI, 0.85, 1.21) for adverse events (AEs), showing no significant difference from placebo. Batoclimab significantly reduced AEs (RR, 0.19; 95% CrI, 0, 0.97).
• Renal Transplant Patients with aHUS: Infection was the most common adverse event, with infection and graft-versus-host disease (GvHD) being the main causes of death.
• Paroxysmal Nocturnal Hemoglobinuria (PNH): Common adverse events included nasopharyngitis, headache, upper respiratory tract infection, nausea, fatigue, diarrhea, cough, pyrexia, abdominal pain, pain in extremities, and contusion.
• There is no population types or subgroups information available in the reviewed studies.