engleski

Fluocortolon treatment and DNA studies in Duchenne boys

Results of steroid treatment in 7 boys with severe and intermediate type of DMD are presented as a pilot study. Fluocortolon (Ultraan) 0.75 mg/kg was applied randomly either every or every other day for 12 months followed by 0.75 mg/kg on alternate days (HSDAD) during next 12 months in all treated patients. We observed improvement in all of the treated patients in the first three months of treatment with plateau period of 3-5 months which continued in slow but steady declining phase of variable duration. Weight gain and cuschingoid face were the only adverse reactions that occurred. Control group consisted of 4 DMD boys who received no treatment. The DNA studies performed by multiplex PCR revealed deletions of exons round the hot spot regions in 2 of 5 boys in fluocortolon treated group and in each patient who received no treatment. The size of deletion was independent of phenotype and steroid response. Prenatal DNA study was successfully performed in a male foetus of affected DMD brother with 47-53 exon deletions. No deletion was found. No significant changes in cardiac function and psychological testing were observed in the period of 6-12 months of fluocortolon treatment in deleted and nondeleted patients, as well as in the control untreated group.

fluocortolon; Duchenne muscular dystrophy; steroid treatment

nije evidentirano