title: Renal function in children and adolescents with Duchenne muscular dystrophy.
abstract: Improved life expectancy and the need for robust tools to monitor renal safety of emerging new therapies have fueled the interest in renal function in Duchenne muscular dystrophy (DMD) patients. We aimed to establish a methodology to accurately assess their renal function. Twenty DMD patients (5-22 years) were included in this prospective study. After obtaining medical history, all patients underwent a clinical examination, 24-hour ambulatory blood pressure monitoring, ultrasound of the kidneys, direct GFR measurement ((51)Cr-EDTA, mGFR), complete blood and urine analysis. Seventeen of 20 patients were treated with corticosteroids and 5/20 with angiotensin converting enzyme inhibitor (lisinopril). No patient suffered from urinary tract infections or other renal diseases. Hypertension (systolic or diastolic blood pressure >P95) was found in 9/20 patients (8/9 patients were on steroid treatment) and a non-dipping blood pressure profile in 13/20 subjects (10/13 patients were on steroid treatment). Urinary protein to creatinine ratio was elevated in 17/18 patients, whereas 24-hour urine protein excretion was normal in all subjects. Median interquartile range (IQR) mGFR was 130.4 (29.1) mL/min/1.73 m(2). Hyperfiltration (mGFR >150 mL/min/1.73 m(2)) was found in 5/20 patients. Inverse correlation between mGFR and age was observed (R(2) = 0.45, p = 0.001). Serum creatinine based estimated GFR (eGFR) equations overestimated mGFR up to 300%. eGFR based on cystatin C Filler equation was closest to the mGFR (median eGFR (IQR) of 129.5 (39.7) mL/min/1.73 m(2)). Our study demonstrates a high prevalence of hyperfiltration and hypertension in children and adolescents with DMD. Because the majority of hypertensive patients were under corticosteroid treatment, the iatrogenic cause of hypertension cannot be excluded. Serum or urine creatinine measurements are of no value to evaluate renal function in DMD patients due to the reduced skeletal muscle mass.
authors: Department of Pediatrics, University Hospitals Leuven, Leuven, Belgium.
authors: Elke
authors: E
authors: Braat
authors: Department of Public Health and Primary Care @ Kulak, KU Leuven Kulak, Kortrijk, Belgium.
authors: Liesbeth
authors: L
authors: Hoste
authors: Department of Pediatrics, University Hospitals Leuven, Leuven, Belgium.
authors: Liesbeth
authors: L
authors: De Waele
authors: Department of Nuclear Medicine, University Hospitals Leuven, Leuven, Belgium.
authors: Olivier
authors: O
authors: Gheysens
authors: Laboratory of Medicine, University Hospitals Leuven, Leuven, Belgium.
authors: Pieter
authors: P
authors: Vermeersch
authors: Department of Nuclear Medicine, University Hospitals Leuven, Leuven, Belgium.
authors: Karolien
authors: K
authors: Goffin
authors: Department of Public Health and Primary Care @ Kulak, KU Leuven Kulak, Kortrijk, Belgium.
authors: Hans
authors: H
authors: Pottel
authors: Department of Pediatrics, University Hospitals Leuven, Leuven, Belgium.
authors: Nathalie
authors: N
authors: Goemans
authors: Department of Pediatrics, University Hospitals Leuven, Leuven, Belgium. Electronic address: elena.levtchenko@uzleuven.be.
authors: Elena
authors: E
authors: Levtchenko
mesh_terms: D000293:Adolescent; D000328:Adult; D001794:Blood Pressure; D002648:Child; D002675:Child, Preschool; D005919:Glomerular Filtration Rate; D006801:Humans; D007668:Kidney / Q000000981:diagnostic imaging / Q000503:physiopathology*; D008297:Male; D020388:Muscular Dystrophy, Duchenne / Q000097:blood / Q000503:physiopathology* / Q000652:urine; D011446:Prospective Studies; D014463:Ultrasonography; D001743:Urinary Bladder / Q000000981:diagnostic imaging / Q000503:physiopathology*; D055815:Young Adult
keywords: Blood pressure; Duchenne muscular dystrophy; GFR; Hyperfiltration; Kidney function