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J Neurol Sci. 2025 Jul 16;476:123628. doi: 10.1016/j.jns.2025.123628. Online ahead of print.

ABSTRACT

BACKGROUND: Complement component 5 inhibitors are indicated for the treatment of anti-acetylcholine receptor antibody-positive generalized myasthenia gravis (gMG). Clinical trials have demonstrated improved functional ability, muscle strength, and quality of life (QOL) in patients treated with eculizumab or ravulizumab. Evidence for their effectiveness and safety in clinical practice is reported here.

METHODS: Data from the global MG SPOTLIGHT Registry were collected from patients with gMG currently or previously treated with eculizumab or ravulizumab in routine clinical practice. Effectiveness was evaluated among patients with ≥3 outcome measurements using MG Activities of Daily Living (MG-ADL) and MG-QOL 15-revised (MG-QOL15r) patient-reported outcomes and/or Myasthenia Gravis Foundation of America Clinical Classification (MGFA class) physician assessments. Safety data were also assessed.

RESULTS: 189 patients were treated with eculizumab. Mean (95% CI) MG-ADL scores (n=110) decreased by 4.2 (-5.0, -3.4) points and MG-QOL15r scores (n=40) decreased by 7.5 (-9.9, -‍5.0) points from before eculizumab initiation to first assessment during/after treatment (median eculizumab treatment duration: 18.0 months). 45/103 (43.7%) patients were MGFA class 0-II before eculizumab versus 89/103 (86.4%) at first assessment. After transitioning to ravulizumab, improvements in MG-ADL scores (n=37), MG-QOL15r scores (n=14), and MGFA class (n=34) were maintained (median ravulizumab treatment duration: 8.7-11.4 months). Few treatment-related serious adverse events (eculizumab: Aspergillus infection, nervous system disorder [n=1 each]; ravulizumab: anaphylactic reaction [n=1]) and no meningococcal infections were reported with eculizumab or ravulizumab treatment.

CONCLUSIONS: In routine clinical practice, eculizumab was well tolerated and effective for patients with gMG. Treatment benefits were maintained after transition to ravulizumab.

PMID:40812271 | DOI:10.1016/j.jns.2025.123628


Source: https://pubmed.ncbi.nlm.nih.gov/4081227 ... t9+e462414