Table 1.
Baseline Demographic and Clinical Characteristics
| Efgartigimod (n = 21) | Ravulizumab (n = 41) | Total (N = 62) | |
| Age (y), median (IQR) | 61.00 (43.00–73.00) | 69.00 (47.00–77.00) | 64.00 (44.00–75.50) |
| Early-onset myasthenia gravis, n (%) | 7 (33.3) | 16 (39.0) | 23 (37.1) |
| Sex, n (%) | |||
| Female | 12 (57.1) | 27 (65.9) | 39 (62.9) |
| Male | 9 (42.9) | 14 (34.1) | 23 (37.1) |
| Weight (kg), median (IQR) | 77.00 (65.00–87.00) | 77.00 (60.00–88.00) | 77.00 (62.00–87.75) |
| Disease duration (y), median (IQR) | 4.00 (2.00–6.00) | 7.00 (3.00–10.00) | 5.00 (2.00–9.75) |
| MGFA class at screening, n (%) | |||
| II | 8 (38.1) | 10 (24.4) | 18 (29.0) |
| III | 12 (57.1) | 27 (65.9) | 39 (62.9) |
| IV | 1 (4.8) | 4 (9.8) | 5 (8.1) |
| Previous thymectomy, n (%) | 13 (61.9) | 25 (61.0) | 38 (61.3) |
| Thymoma, n (%) | 2 (11.8) | 7 (19.4) | 9 (17.0) |
| n missing | 4 | 5 | 9 |
| History of myasthenic crisis, n (%) | 12 (57.1) | 24 (58.5) | 36 (58.1) |
| Total MG-ADL score, median (IQR) | 10.00 (8.00–11.00) | 9.00 (7.00–12.00) | 10.00 (7.00–12.00) |
| n missing | 0 | 2 | 2 |
| Total QMG score, median (IQR) | 14.00 (10.00–17.00) | 14.50 (11.00–19.25) | 14.00 (11.00–18.00) |
| n missing | 0 | 1 | 1 |
| Total MG-QOL15r score, median (IQR) | 37.00 (31.00–40.00) | 22.50 (19.00–26.50) | 25.00 (19.50–36.50) |
| n missing | 8 | 11 | 19 |
| At least one previous IST, n (%) | 21 (100) | 41 (100) | 62 (100) |
| Myasthenia gravis therapy at baseline, n (%) | |||
| Any steroid | 12 (63.2) | 26 (63.4) | 38 (63.3) |
| n missing | 2 | 0 | 2 |
| Any immunosuppressive therapy | 18 (85.7) | 27 (65.9) | 45 (72.6) |
| Rituximab | 0 (0.0) | 5 (12.2) | 5 (8.1) |
| Steroid and any immunosuppressive therapy | 10 (52.6) | 20 (48.8) | 30 (50.0) |
| No steroid and no immunosuppressive therapy | 1 (4.8) | 8 (19.5) | 9 (14.5) |
| Refractory MG at baselinea, n (%) | 15 (71.4) | 23 (59.0) | 38 (63.3) |
| n missing | 0 | 2 |
Abbreviations: AChR = acetylcholine receptor; IQR = interquartile range; IST = immunosuppressive therapy; MG-ADL = MG specific activity of daily life score; MGFA = Myasthenia Gravis Foundation of America classification; MG-QoL15 = MG-Specific Quality of Life Score; QMG = Quantitative MG Score.
Reported are median (IQR) for continuous variables and n (%) for categorical variables. Disease duration is the time from diagnosis until baseline. The data on the thymoma findings refer to the number of patients in the respective group, regardless of whether a thymectomy was performed. We cannot exclude the possibility that a thymoma was present in the non-thymectomized patients, but there was no evidence of a thymoma on the CT scan of the thorax.
Immunotherapy refractory status was defined according to the randomized controlled phase 3 trial of eculizumab in refractory, AChR-Ab-positive, generalized MG (REGAIN): diagnosis of a generalized AChR-Ab–positive MG with a MG-ADL score at baseline of >5 and a history of either 1 2 or more immunosuppressive therapies, or 2 at least one immunosuppressive therapy with IVIg or plasma exchange given at least 4 times per year, for 12 months without symptom control.