ADAPT
Trial question
What is the role of efgartigimod in patients with generalized myasthenia gravis?
Study design
Multi-center
Double blinded
RCT
Population
Characteristics of study participants
71.0% female
29.0% male
N = 167
167 patients (118 female, 49 male)
Inclusion criteria: adult patients with myasthenia gravis and a Myasthenia Gravis Activities of Daily Living score ≥ 5
Key exclusion criteria: receipt of rituximab or eculizumab within 6 months; thymectomy within 3 months; IVIG or plasma exchange within 1 month; active hepatitis B; hepatitis C seropositivity; HIV seropositivity with low CD4 count; serum IgG levels < 6 g/L; pregnancy
Interventions
N=84 efgartigimod (intravenous dose of 10 mg/kg, administered as four infusions per cycle; one infusion per week)
N=83 placebo (intravenous administration of matching placebo)
Primary outcome
Positive anti-acetylcholine receptor antibody patients with a clinically meaningful reduction in Myasthenia Gravis Activities of Daily Living score at cycle 1
68
30
68.0 %
51.0 %
34.0 %
17.0 %
0.0 %
Efgartigimod
Placebo
Significant increase ▲
NNT = 2
Significant increase in positive anti-AChR antibody patients with a clinically meaningful reduction in Myasthenia Gravis Activities of Daily Living score at cycle 1 (68% vs. 30%; OR 4.95, 95% CI 2.21 to 11.53)
Secondary outcomes
Significant increase in patients with a clinically meaningful reduction in Quantitative Myasthenia Gravis score (63% vs. 14%; OR 10.84, 95% CI 4.18 to 31.2)
Significant increase in patients with a clinically meaningful reduction in Myasthenia Gravis Activities of Daily Living score at cycle 1 (68% vs. 37%; OR 3.7, 95% CI 1.85 to 7.58)
Significant increase in patients with a clinically meaningful reduction in Myasthenia Gravis Activities of Daily Living score at day 126 (48.7% vs. 26.6%; AD 22.1%, 95% CI 11.06 to 33.14)
Safety outcomes
No significant difference in adverse events.
Conclusion
In adult patients with myasthenia gravis and a Myasthenia Gravis Activities of Daily Living score ≥ 5, efgartigimod was superior to placebo with respect to positive anti-AChR antibody patients with a clinically meaningful reduction in Myasthenia Gravis Activities of Daily Living score at cycle 1.
Reference
James F Howard Jr, Vera Bril, Tuan Vu 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 Jul;20(7):526-536.
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