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.).

Clinical description of intracranial hemorrhage associated with bleeding disorders.

BACKGROUND: Intracerebral hemorrhage (ICH) is an unusual but serious complication of bleeding disorders. ICH is believed to follow thrombocytopenia, alterations in coagulation, and vascular fragility. Information regarding its distribution is nonconclusive, and the mechanism of bleeding is not fully understood. The aim of this study was to examine the clinical and neuroimaging features of ICH in patients with bleeding disorders to predict risk factors for this condition. METHODS: All cases of ICH diagnosed from 1987 to 2004 were retrospectively identified using the centralized database of our institution. Cases were included whenever ICH was caused by a primary hematologic disorder. The clinical characteristics, neuroimages, and outcome were analyzed. RESULTS: A total of 31 patients were identified. ICH was the initial presentation of the bleeding disorder in 9 patients. Overall, 71% had systemic bleeding concurrent to the ICH. All patients had altered mental status. In 45.2% of the patients simultaneous intracranial hemorrhages were found. Eight patients had recurrent ICH. Severe thrombocytopenia (platelet count < 10,000/mm(3)) was present in 41% and very low platelets (