- Wolfe, Gil I;
- Kaminski, Henry J;
- Aban, Inmaculada B;
- Minisman, Greg;
- Kuo, Hui-Chien;
- Marx, Alexander;
- Ströbel, Philipp;
- Mazia, Claudio;
- Oger, Joel;
- Cea, J Gabriel;
- Heckmann, Jeannine M;
- Evoli, Amelia;
- Nix, Wilfred;
- Ciafaloni, Emma;
- Antonini, Giovanni;
- Witoonpanich, Rawiphan;
- King, John O;
- Beydoun, Said R;
- Chalk, Colin H;
- Barboi, Alexandru C;
- Amato, Anthony A;
- Shaibani, Aziz I;
- Katirji, Bashar;
- Lecky, Bryan RF;
- Buckley, Camilla;
- Vincent, Angela;
- Dias-Tosta, Elza;
- Yoshikawa, Hiroaki;
- Waddington-Cruz, Márcia;
- Pulley, Michael T;
- Rivner, Michael H;
- Kostera-Pruszczyk, Anna;
- Pascuzzi, Robert M;
- Jackson, Carlayne E;
- Garcia Ramos, Guillermo S;
- Verschuuren, Jan JGM;
- Massey, Janice M;
- Kissel, John T;
- Werneck, Lineu C;
- Benatar, Michael;
- Barohn, Richard J;
- Tandan, Rup;
- Mozaffar, Tahseen;
- Conwit, Robin;
- Odenkirchen, Joanne;
- Sonett, Joshua R;
- Jaretzki, Alfred;
- Newsom-Davis, John;
- Cutter, Gary R
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.).