Efgartigimod and Ravulizumab for Treating Acetylcholine Receptor Auto-antibody-Positive (AChR-Ab+) Generalized Myasthenia Gravis: Indirect Treatment Comparison.
AChR-Ab+
Acetylcholine receptor auto-antibodies positive
Efgartigimod
Generalized myasthenia gravis
MG-ADL
MG-QoL15
QMG
Ravulizumab
Journal
Advances in therapy
ISSN: 1865-8652
Titre abrégé: Adv Ther
Pays: United States
ID NLM: 8611864
Informations de publication
Date de publication:
20 Apr 2024
20 Apr 2024
Historique:
received:
26
02
2024
accepted:
22
03
2024
medline:
20
4
2024
pubmed:
20
4
2024
entrez:
20
4
2024
Statut:
aheadofprint
Résumé
Efgartigimod and ravulizumab, both approved for treating acetylcholine receptor auto-antibody-positive (AChR-Ab+) generalized myasthenia gravis (gMG), have not been directly compared. This paper assessed comparative effects of efgartigimod vs. ravulizumab for treating adults with AChR-Ab+ gMG using indirect treatment comparison methods. The matching-adjusted indirect comparison used data from two randomized trials of adult men and women. The ADAPT (efgartigimod vs. placebo; individual patient data available) population was reweighted to match the CHAMPION (ravulizumab vs. placebo; index study; aggregate data available) population. The relative effect of efgartigimod versus placebo was estimated in this reweighted population and compared with the observed ravulizumab versus placebo effect to estimate the efgartigimod versus ravulizumab effect. The outcomes were Myasthenia Gravis Activities of Daily Living (MG-ADL), Quantitative Myasthenia Gravis (QMG), and Myasthenia Gravis Quality of Life 15-item-revised scale (MG-QoL15r) assessed as cumulative effect (area under the curve; AUC) over 26 weeks (primary) and change from baseline at 4 weeks and time of best response (week 4 for efgartigimod; week 26 for ravulizumab). For MG-QoL15r, efgartigimod had a statistically significant improvement compared with ravulizumab over 26 weeks [mean difference (95% confidence interval): - 52.6 (- 103.0, - 2.3)], at week 4 [- 4.0 (- 6.6, - 1.4)], and at time of best response [- 3.9 (- 6.5, - 1.3)]. Efgartigimod had a statistically significant improvement over ravulizumab in MG-ADL at week 4 [- 1.9 (- 3.3, - 0.5)] and at time of best response [- 1.4 (- 2.8, 0.0)] and in QMG at week 4 [- 3.2 (- 5.2, - 1.2)] and at time of best response [- 3.0 (- 5.0, - 1.0)]. For AUC over 26 weeks, improvements were not significantly different between efgartigimod and ravulizumab for MG-ADL [- 8.7 (- 36.1, 18.8)] and QMG [- 13.7 (- 50.3, 22.9)]. Efgartigimod may provide a faster and greater improvement over 26 weeks in quality of life than ravulizumab in adults with AChR-Ab+ gMG. Efgartigimod showed faster improvements in MG-ADL and QMG than ravulizumab.
Identifiants
pubmed: 38642198
doi: 10.1007/s12325-024-02856-3
pii: 10.1007/s12325-024-02856-3
doi:
Types de publication
Journal Article
Langues
eng
Sous-ensembles de citation
IM
Informations de copyright
© 2024. The Author(s).
Références
Howard JF, et al. Safety, efficacy, and tolerability of efgartigimod in patients with generalised myasthenia gravis (ADAPT): a multicentre, randomised, placebo-controlled, phase 3 trial. Lancet Neurol. 2021;20(7):526–36.
doi: 10.1016/S1474-4422(21)00159-9
pubmed: 34146511
Szczudlik P, et al. Determinants of quality of life in myasthenia gravis patients. Front Neurol. 2020;11: 553626.
doi: 10.3389/fneur.2020.553626
pubmed: 33071942
pmcid: 7538807
Yang Y, et al. Quality of life in 188 patients with myasthenia gravis in China. Int J Neurosci. 2016;126(5):455–62.
doi: 10.3109/00207454.2015.1038712
pubmed: 26000922
Narayanaswami P, et al. International consensus guidance for management of myasthenia gravis: 2020 update. Neurology. 2021;96(3):114–22.
doi: 10.1212/WNL.0000000000011124
pubmed: 33144515
pmcid: 7884987
Wolfe GI, et al. Myasthenia gravis activities of daily living profile. Neurology. 1999;52(7):1487–9.
doi: 10.1212/WNL.52.7.1487
pubmed: 10227640
Vu T, et al. Terminal complement inhibitor ravulizumab in generalized myasthenia gravis. NEJM Evid. 2022. https://doi.org/10.1056/EVIDoa2100066 .
doi: 10.1056/EVIDoa2100066
pubmed: 38319212
Vu T, et al. Efficacy and safety of ravulizumab, a long-acting terminal complement inhibitor, in adults with anti-acetylcholine receptor antibody-positive generalized myasthenia gravis: results from the phase 3 CHAMPION MG study (P1–1.Virtual). Neurology. 2022. https://doi.org/10.1212/WNL.98.18_supplement.791 .
doi: 10.1212/WNL.98.18_supplement.791
pubmed: 34921103
Phillippo DM, et al. Methods for population-adjusted indirect comparisons in health technology appraisal. Med Decis Mak. 2018;38(2):200–11.
doi: 10.1177/0272989X17725740
Signorovitch JE, et al. Matching-adjusted indirect comparisons: a new tool for timely comparative effectiveness research. Value Health. 2012;15(6):940–7.
doi: 10.1016/j.jval.2012.05.004
pubmed: 22999145
Barohn RJ, et al. Reliability testing of the quantitative myasthenia gravis score. Ann N Y Acad Sci. 1998;841:769–72.
doi: 10.1111/j.1749-6632.1998.tb11015.x
pubmed: 9668327
Burns TM, et al. Construct and concurrent validation of the MG-QOL15 in the practice setting. Muscle Nerve. 2010;41(2):219–26.
doi: 10.1002/mus.21609
pubmed: 19941339
Saccà F, et al. Efficacy of innovative therapies in myasthenia gravis: a systematic review, meta-analysis and network meta-analysis. Eur J Neurol. 2023;30(12):3854–67.
doi: 10.1111/ene.15872
pubmed: 37204031
Wiendl H, Meisel A. Diagnostics and Therapy of myasthenic syndromes. S2k-Guideline 2023. 2023.