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Early prednisone treatment in Duchenne muscular dystrophy
Author(s) -
Merlini Luciano,
Cicognani Alessandro,
Malaspina Elisabetta,
Gennari Monia,
Gnudi Saverio,
Talim Beril,
Franzoni Emilio
Publication year - 2003
Publication title -
muscle and nerve
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.025
H-Index - 145
eISSN - 1097-4598
pISSN - 0148-639X
DOI - 10.1002/mus.10319
Subject(s) - prednisone , duchenne muscular dystrophy , medicine , muscular dystrophy , surgery , pediatrics
The purpose of this long‐term, open parallel‐group, double‐consent study of alternate‐day, low‐dose prednisone in 2–4‐year‐old patients with Duchenne muscular dystrophy (DMD) was to determine whether prednisone produces a beneficial effect when given earlier than usual. Muscle function was evaluated by timed tests, and muscle strength with a hand‐held myometer. After 55 months of treatment, the five patients (mean age 8.3 years) in the prednisone group were still able to get up from the floor, whereas two of the three in the control group had lost this ability. Side effects included a decline in growth rate in the prednisone‐treated patients and excessive weight gain in one control and three treated patients. Because steroids are effective in prolonging function, but not in recovering lost function, we propose that treatment be started with low‐dose prednisone in DMD patients as soon as the diagnosis is definite. Muscle Nerve 27: 222–227, 2003