1. 413P Natural history of renal dysfunction in Duchenne muscular dystrophy.
- Author
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Arahata, H.
- Subjects
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DUCHENNE muscular dystrophy , *OLDER patients , *BLOOD urea nitrogen , *MUSCULAR atrophy , *HEART failure patients - Abstract
The main clinical features in patients with Duchenne muscular dystrophy (DMD) are (1) progressive muscle atrophy and weakness, (2) cardiac dysfunction, and (3) intellectual disability. However, there are few reports about natural history of patients with DMD develop renal dysfunction with age. Previous studies have shown that as skeletal muscle mass decreases, serum creatinine levels are less likely to reflect renal functions. In this study, we retrospectively examined how blood urea nitrogen (BUN) levels change over time. Patients clinically or genetically diagnosed with DMD were included in this study. The study period was from April 1st, 2012 to August 31st, 2023. The eligible patients underwent BUN levels' measurement at least twice. High BUN was defined as 30mg/dl or higher. Two or more consecutive increases in BUN are counted as one time. A total of 52 patients were included in the study, and their mean duration of observation was 7.7 years. Those with a BUN level ≤30 mg/dl obtained during the study period were considered to have renal dysfunction, accounting to 15 patients. High BUN was initially detected at 28.7 years of age (±8.0) and mean peak BUN level was 116.5 mg/dl. Their mean total duration of rising periods was 108 days, respectively. And these periods occurred 2.33 times/person on average. Two patients died from renal failure, according to the attending clinician. However, renal function improved in the remaining patients. No patients died of heart failure in the study period. The results for the remaining 37 patients showed that the mean age at the time of the first measurement was 14.8 years old (±6.1) and the mean observation period was 7.03 years. The average number of BUN tests was 20.5/7.03 y. (1 test /4.1month). The average maximum BUN value was 16.0 mg/dL (±5.6), the average minimum BUN value was 5.8 (±3.0), and the average value was 10.2. Although increase in BUN levels can be found in conditions other than renal dysfunction, BUN levels changed in 30% of patients with DMD. Since renal dysfunction is an inhibitory factor of treatment with morpholino-based drugs and affects survival, a future prospective study is required for elderly patients with DMD, renal function tests are recommended once every 3 to 4 months, and shorter follow-up is recommended if abnormalities are detected. 30% of patients with elderly DMD may have renal dysfunctions. About 10% in these patients may die by renal dysfunction, although in 90% of these patients' recovery can be expected within 3 months of natural course. [ABSTRACT FROM AUTHOR]
- Published
- 2024
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