Clinical aspects of patients with sarcoglycanopathies under steroids therapy

作者：Albuquerque Marco A V; Abath Neto Osorio; Maximino Jessica R; Chadi Gerson; Zanoteli Edmar; Reed Umbertina C^*

来源：Arquivos de Neuro-Psiquiatria, 2014, 72(10): 768-772.

DOI：10.1590/0004-282X20140126

• steroids
• limb-girdle muscular dystrophy
• sarcoglycan proteins
• myopathy

Patients with sarcoglycanopathies, which comprise four subtypes of autosomal recessive limb-girdle muscular dystrophies, usually present with progressive weakness leading to early loss of ambulation and premature death, and no effective treatment is currently available. Objective: To present clinical aspects and outcomes of six children with sarcoglycanopathies treated with steroids for at least one year. Method: Patient files were retrospectively analyzed for steroid use. Results: Stabilization of muscle strength was noted in one patient, a slight improvement in two, and a slight worsening in three. In addition, variable responses of forced vital capacity and cardiac function were observed. Conclusions: No overt clinical improvement was observed in patients with sarcoglycanopathies under steroid therapy. Prospective controlled studies including a larger number of patients are necessary to determine the effects of steroids for sarcoglycanopathies.

• 出版日期2014-10