Clinical aspects of patients with sarcoglycanopathies under steroids therapy.

Abstract

UNLABELLED:

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.

• Authors: Marco A V Albuquerque, Osório Abath-Neto, Jéssica R Maximino, Gerson Chadi, Edmar Zanoteli, Umbertina C Reed
• Journal: Arquivos de neuro-psiquiatria (Arq Neuropsiquiatr) Vol. 72 Issue 10 Pg. 768-72 (Oct 2014) ISSN: 1678-4227 [Electronic] Germany
• PMID: 25337728 (Publication Type: Journal Article, Research Support, Non-U.S. Gov't)

Chemical References

• Glucocorticoids
• Pregnenediones
• Prednisolone
• deflazacort

Topics

• Child
• Female
• Glucocorticoids (therapeutic use)
• Humans
• Male
• Prednisolone (therapeutic use)
• Pregnenediones (therapeutic use)
• Retrospective Studies
• Sarcoglycanopathies (drug therapy)
• Treatment Outcome