Source:Pediatric Neurology
Author(s): Chanchan Liu, Mengcui Gui, Yayun Cao, Jing Lin, Yue Li, Suqiong Ji, Bitao Bu
BackgroundMyasthenia gravis tends to affect children in China. Oral pyridostigmine and prednisone could effectively improve the symptoms, but multiple side effects constitute the major concern after long-term oral prednisone. Try to overcome the flaws of prednisone therapy and to obtain more satisfied improvement, we tested the efficacy and safety of tacrolimus in children.MethodsChildren with myasthenia gravis who had not achieved satisfactory improvement and suffered from severe side effects after prednisone therapy were recruited between January 2015 and December 2016 at Tongji Hospital. All the children were treated with tacrolimus 1-2 mg daily and adjusted based on the response and serum concentration. The dosage of prednisone, severity of symptoms, blood samples, serum concentration of tacrolimus and titers of anti-AChR antibodies were evaluated every 4 weeks.ResultsFourteen children were enrolled. One child withdrew in 2 weeks after the enrollment. Thirteen children have completed the therapy for 1 year. At the endpoint, dosage of prednisone were significantly decreased (p<0.05), the symptoms evaluated by quantitative myasthenia gravis score, myasthenia gravis-specific manual muscle testing and myasthenia gravis-activities of daily living score were significantly improved (p<0.05, p<0.05, p<0.01,respectively). More importantly, 10 (76.9%) patients had completely discontinued prednisone, and the major side effects were nearly reversed. The mean titer of anti-AChR antibodies significantly dropped from 1.96±2.62 nmol/L to 0.70±1.04 nmol/L (P<0.05). No severe adverse events were reported.ConclusionsOur result suggested that tacrolimus is a promising agent for children with refractory myasthenia gravis. Randomized clinical trials are needed to confirm the observation.
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