Abstract | BACKGROUND: OBJECTIVE: To determine safety and tolerability of once-weekly prednisone in patients with LGMD and BMD. METHODS: We conducted an open label, exploratory single center study of of once-weekly prednisone at 0.75-1 mg/Kg in LGMD (n = 19) and BMD (n = 1) (mean age 35, range 18-60). The LGMD participants represented multiple different LGMD subtypes, and the study included ambulatory and non-ambulatory participants. Participants were assessed at baseline and 24 weeks for vital signs, blood biomarkers, and for patient-reported side effects. As secondary endpoints, functional muscle testing and body composition were measured. RESULTS: Over the 24-week study, there were no significant changes in blood pressure, HgbA1C, or lipid profiles. We observed a reduction in serum creatine kinase over the study interval. Whole body DEXA scanning suggested a possible increase in lean mass and a reduction in adiposity. Functional measures suggested trends in improved muscle performance. CONCLUSIONS: In this single center, open label pilot study, once-weekly prednisone was safe and well tolerated. Additional investigation of once-weekly prednisone in a larger cohort and for a longer period of time is warranted.
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Authors | Aaron S Zelikovich, Benjamin C Joslin, Patricia Casey, Elizabeth M McNally, Senda Ajroud-Driss |
Journal | Journal of neuromuscular diseases
(J Neuromuscul Dis)
Vol. 9
Issue 2
Pg. 275-287
( 2022)
ISSN: 2214-3602 [Electronic] Netherlands |
PMID | 35124660
(Publication Type: Clinical Trial, Journal Article)
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Chemical References |
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Topics |
- Drug Administration Schedule
- Humans
- Muscular Dystrophies, Limb-Girdle
(drug therapy)
- Muscular Dystrophy, Duchenne
(drug therapy)
- Pilot Projects
- Prednisone
(administration & dosage, adverse effects)
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