TY - JOUR
T1 - Glucocorticoid-Associated Demise of a Patient with Duchenne Muscular Dystrophy
AU - Brahmsteadt, Alexander E.
AU - Bach, John R.
AU - Pishdad, Reza
AU - Cespedes, Lissette
AU - Pierucci, Paola
N1 - Publisher Copyright:
© Wolters Kluwer Health, Inc. All rights reserved.
PY - 2020/12
Y1 - 2020/12
N2 - We describe the clinical deterioration of a 26-yr-old man with Duchenne muscular dystrophy on oral daily high-dose deflazacort. Although this daily regimen was targeted to benefit ambulation and respiration, it resulted in premature death with lethal sequelae from liver failure, decubiti, diabetes mellitus, and morbid obesity. This case illustrates the need for further research weighing risk versus benefit of daily glucocorticoid therapy, specifically deflazacort, in Duchenne muscular dystrophy patients. Thus, curtailment of daily dosing to eliminate dire sequelae in patients living longer into adulthood than ever before is recommended.
AB - We describe the clinical deterioration of a 26-yr-old man with Duchenne muscular dystrophy on oral daily high-dose deflazacort. Although this daily regimen was targeted to benefit ambulation and respiration, it resulted in premature death with lethal sequelae from liver failure, decubiti, diabetes mellitus, and morbid obesity. This case illustrates the need for further research weighing risk versus benefit of daily glucocorticoid therapy, specifically deflazacort, in Duchenne muscular dystrophy patients. Thus, curtailment of daily dosing to eliminate dire sequelae in patients living longer into adulthood than ever before is recommended.
KW - Deflazacort
KW - Duchenne Muscular Dystrophy
KW - Glucocorticoid
KW - Neuromuscular Disease
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U2 - 10.1097/PHM.0000000000001426
DO - 10.1097/PHM.0000000000001426
M3 - Article
C2 - 32195738
AN - SCOPUS:85096508903
SN - 0894-9115
VL - 99
SP - e146-e148
JO - American Journal of Physical Medicine and Rehabilitation
JF - American Journal of Physical Medicine and Rehabilitation
IS - 12
ER -