Summary

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• This summary is based on the review of 22 systematic review(s)/meta-analysis(es). ^[1-22]
• Myasthenia Gravis (MG): Batoclimab showed the highest effectiveness on quantitative MG and MG Composite scores with SUCRA (surface under the cumulative ranking curve) values of 99% and 92%, respectively. Rozanolixzumab was most effective in improving MG Activities of Daily Living with a SUCRA value of 85%. Eculizumab demonstrated the best improvement in MG Quality of Life Score (SUCRA 96%).
• Paroxysmal Nocturnal Hemoglobinuria (PNH): Complement inhibitors, including eculizumab, reduced LDH levels for treatment durations ≤26 weeks and >26 weeks; mean hemoglobin levels increased by 1.4 g/dL (≤26 weeks) and 1.9 g/dL (>26 weeks); transfusion avoidance was achieved in at least 50% of patients. FACIT-F scores improved in both time frames, indicating reduced fatigue.
• Infection-Associated Hemolytic Uremic Syndrome (IA-HUS): Eculizumab did not show statistically significant positive effects on medium- to long-term outcomes, with studies indicating a high risk of bias.
• Atypical Hemolytic Uremic Syndrome (aHUS): Eculizumab was associated with improved renal function, reduced recurrence rates, and decreased need for dialysis. Both eculizumab and ravulizumab showed comparable efficacy and safety, though eculizumab required more frequent dosing and had a higher financial burden.
• MG: Rozanolixzumab had a higher likelihood of adverse events (AEs) compared to placebo.
• PNH: Complement inhibitors, including eculizumab, generally demonstrated an acceptable safety profile, though specific adverse events were not detailed among different inhibitors. 8. aHUS: Eculizumab was linked to serious adverse events in 42% of patients, including meningococcal infections, with a similar safety profile to ravulizumab, which had the benefit of less frequent dosing.
• Eculizumab and other complement inhibitors demonstrated clinically relevant efficacy across diverse subgroups, including generalized and refractory myasthenia gravis (MG) patients with improved quality of life; treatment-naive and long-term-treated paroxysmal nocturnal hemoglobinuria (PNH) patients with reduced hemolysis and increased hemoglobin; severely ill infection-associated and atypical hemolytic uremic syndrome (IA-HUS and aHUS) patients showing mixed results but high infection risk; AQP4-IgG seropositive neuromyelitis optica spectrum disorder (NMOSD) patients with reduced relapse risk; and lupus nephritis (LN) patients with thrombotic microangiopathy showing benefit in managing TMA.