Efgartigimod and Ravulizumab for Treating Acetylcholine Receptor Auto-antibody-Positive (AChR-Ab plus ) Generalized Myasthenia Gravis: Indirect Treatment Comparison

被引:5
作者
van Steen, Cecile [1 ]
Celico, Lorenzo [2 ]
Spaepen, Erik [2 ]
Hagenacker, Tim [3 ]
Meuth, Sven G. [4 ]
Ruck, Tobias [4 ]
Smith, A. Gordon [5 ]
Bodicoat, Danielle H. [2 ]
de Francesco, Maria [2 ]
Iannazzo, Sergio [1 ]
机构
[1] Argenx BV, B-9052 Ghent, Belgium
[2] HEOR Value Hub, Brussels, Belgium
[3] Univ Med Essen, D-45147 Essen, Germany
[4] Univ Hosp Dusseldorf, D-40225 Dusseldorf, Germany
[5] VCU Dept Neurol, Richmond, VA USA
关键词
Acetylcholine receptor auto-antibodies positive; AChR-Ab plus; Efgartigimod; Generalized myasthenia gravis; MG-ADL; MG-QoL15; QMG; Ravulizumab; ADJUSTED INDIRECT COMPARISONS;
D O I
10.1007/s12325-024-02856-3
中图分类号
R-3 [医学研究方法]; R3 [基础医学];
学科分类号
1001 ;
摘要
Introduction 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.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).Results 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)].Conclusion 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.
引用
收藏
页码:2486 / 2499
页数:14
相关论文
共 13 条
[1]   Reliability testing of the quantitative myasthenia gravis score [J].
Barohn, RJ ;
McIntire, D ;
Herbelin, L ;
Wolfe, GI ;
Nations, S ;
Bryan, WW .
MYASTHENIA GRAVIS AND RELATED DISEASES: DISORDERS OF THE NEUROMUSCULAR JUNCTION, 1998, 841 :769-772
[2]   CONSTRUCT AND CONCURRENT VALIDATION OF THE MG-QOL15 IN THE PRACTICE SETTING [J].
Burns, Ted M. ;
Grouse, C. K. ;
Conaway, Mark R. ;
Sanders, Donald B. .
MUSCLE & NERVE, 2010, 41 (02) :219-226
[3]   Safety, efficacy, and tolerability of efgartigimod in patients with generalised myasthenia gravis (ADAPT) : a multicentre, randomised, placebo-controlled, phase 3 trial [J].
Howard, James F. Jr Jr ;
Bril, Vera ;
Vu, Tuan ;
Karam, Chafic ;
Perk, Stojan ;
Margania, Temur ;
Murai, Hiroyuki ;
Bilinska, Malgorzata ;
Shakarishvili, Roman ;
Smilowski, Marek ;
Guglietta, Antonio ;
Ulrichts, Peter ;
Vangeneugden, Tony ;
Utsugisawa, Kimiaki ;
Verschuuren, Jan ;
Mantegazza, Renato .
LANCET NEUROLOGY, 2021, 20 (07) :526-536
[4]   International Consensus Guidance for Management of Myasthenia Gravis 2020 Update [J].
Narayanaswami, Pushpa ;
Sanders, Donald B. ;
Wolfe, Gil ;
Benatar, Michael ;
Cea, Gabriel ;
Evoli, Amelia ;
Gilhus, Nils Erik ;
Illa, Isabel ;
Kuntz, Nancy L. ;
Massey, Janice ;
Melms, Arthur ;
Murai, Hiroyuki ;
Nicolle, Michael ;
Palace, Jacqueline ;
Richman, David ;
Verschuuren, Jan .
NEUROLOGY, 2021, 96 (03) :114-122
[5]   Methods for Population-Adjusted Indirect Comparisons in Health Technology Appraisal [J].
Phillippo, David M. ;
Ades, Anthony E. ;
Dias, Sofia ;
Palmer, Stephen ;
Abrams, Keith R. ;
Welton, Nicky J. .
MEDICAL DECISION MAKING, 2018, 38 (02) :200-211
[6]   Efficacy of innovative therapies in myasthenia gravis: A systematic review, meta-analysis and network meta-analysis [J].
Sacca, Francesco ;
Pane, Chiara ;
Espinosa, Pablo Ezequiel ;
Sormani, Maria Pia ;
Signori, Alessio .
EUROPEAN JOURNAL OF NEUROLOGY, 2023, 30 (12) :3854-3867
[7]   Matching-Adjusted Indirect Comparisons: A New Tool for Timely Comparative Effectiveness Research [J].
Signorovitch, James E. ;
Sikirica, Vanja ;
Erder, M. Haim ;
Xie, Jipan ;
Lu, Mei ;
Hodgkins, Paul S. ;
Betts, Keith A. ;
Wu, Eric Q. .
VALUE IN HEALTH, 2012, 15 (06) :940-947
[8]   Determinants of Quality of Life in Myasthenia Gravis Patients [J].
Szczudlik, Piotr ;
Sobieszczuk, Ewa ;
Szyluk, Beata ;
Lipowska, Marta ;
Kubiszewska, Justyna ;
Kostera-Pruszczyk, Anna .
FRONTIERS IN NEUROLOGY, 2020, 11
[9]  
Vu T., 2022, NEUROLOGY, DOI [10.1212/WNL.98.18_supplement.791, DOI 10.1212/WNL.98.18_SUPPLEMENT.791]
[10]   Ravulizumab for adults with generalized myasthenia gravis: a plain language summary of three studies [J].
Aguirre, Florencia ;
Andrade, Renata .
JOURNAL OF COMPARATIVE EFFECTIVENESS RESEARCH, 2024, 13 (11)