Randomized Trial of Thymectomy in Myasthenia Gravis.
BACKGROUND: Thymectomy has been a mainstay in the treatment of myasthenia gravis, but there is no conclusive evidence of its benefit. We conducted a multicenter, randomized trial comparing thymectomy plus prednisone with prednisone alone. METHODS: We compared extended transsternal thymectomy plus alternate-day prednisone with alternate-day prednisone alone. Patients 18 to 65 years of age who had generalized nonthymomatous myasthenia gravis with a disease duration of less than 5 years were included if they had Myasthenia Gravis Foundation of America clinical class II to IV disease (on a scale from I to V, with higher classes indicating more severe disease) and elevated circulating concentrations of acetylcholine-receptor antibody. The primary outcomes were the time-weighted average Quantitative Myasthenia Gravis score (on a scale from 0 to 39, with higher scores indicating more severe disease) over a 3-year period, as assessed by means of blinded rating, and the time-weighted average required dose of prednisone over a 3-year period. RESULTS: A total of 126 patients underwent randomization between 2006 and 2012 at 36 sites. Patients who underwent thymectomy had a lower time-weighted average Quantitative Myasthenia Gravis score over a 3-year period than those who received prednisone alone (6.15 vs. 8.99, P<0.001); patients in the thymectomy group also had a lower average requirement for alternate-day prednisone (44 mg vs. 60 mg, P<0.001). Fewer patients in the thymectomy group than in the prednisone-only group required immunosuppression with azathioprine (17% vs. 48%, P<0.001) or were hospitalized for exacerbations (9% vs. 37%, P<0.001). The number of patients with treatment-associated complications did not differ significantly between groups (P=0.73), but patients in the thymectomy group had fewer treatment-associated symptoms related to immunosuppressive medications (P<0.001) and lower distress levels related to symptoms (P=0.003). CONCLUSIONS: Thymectomy improved clinical outcomes over a 3-year period in patients with nonthymomatous myasthenia gravis. (Funded by the National Institute of Neurological Disorders and Stroke and others; MGTX ClinicalTrials.gov number, NCT00294658.).
Wolfe, GI; Kaminski, HJ; Aban, IB; Minisman, G; Kuo, H-C; Marx, A; Ströbel, P; Mazia, C; Oger, J; Cea, JG; Heckmann, JM; Evoli, A; Nix, W; Ciafaloni, E; Antonini, G; Witoonpanich, R; King, JO; Beydoun, SR; Chalk, CH; Barboi, AC; Amato, AA; Shaibani, AI; Katirji, B; Lecky, BRF; Buckley, C; Vincent, A; Dias-Tosta, E; Yoshikawa, H; Waddington-Cruz, M; Pulley, MT; Rivner, MH; Kostera-Pruszczyk, A; Pascuzzi, RM; Jackson, CE; Garcia Ramos, GS; Verschuuren, JJGM; Massey, JM; Kissel, JT; Werneck, LC; Benatar, M; Barohn, RJ; Tandan, R; Mozaffar, T; Conwit, R; Odenkirchen, J; Sonett, JR; Jaretzki, A; Newsom-Davis, J; Cutter, GR; MGTX Study Group,
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