Steroid therapy and fracture prevention in Duchenne Muscular Dystrophy

Duchenne muscular dystrophy (DMD) is a hereditary neuromuscular disease that leads to progressive muscle fiber degeneration and weakness. There is no cure for this disease and current therapy consists on treatment with glycocorticoids (GC). GC therapy is linked to risk of bone loss and increased fracture risk. Despite their adverse effects GC remain the standard care to slow down disease progression (2).

Steroid therapy and fracture prevention in Duchenne Muscular Dystrophy

This recent study explored factors that are associated with incident fracture risk in glucocorticoid (GC)-treated patients with Duchenne muscular dystrophy (DMD): Risk Factors Associated with Incident Vertebral Fractures in Steroid-treated Males with Duchenne Muscular Dystrophy. Brief, vertical fractures (VF) were prospectively evaluated in 38 males with Duchenne muscular dystrophy at study baseline and 12 months . The authors concluded the following: ” The observation that ≥ 1 prevalent VF and/or non-VF were the strongest predictors of incident VFs at 12 months supports the need for prevention of first fractures in this high-risk setting. Bone age delay, a marker of GC exposure, may assist in the prioritization of patients in efforts to prevent first fractures.”

Steroid therapy and fracture prevention in Duchenne Muscular Dystrophy – The Biomedica IL-6 and Sclerostin ELISA were highlighted in this study.

High Sensitivity IL-6 ELISA  (cat. no. BI-IL6) – measurable values in serum and plasma samples.

• Format: 12×8 wells
• Sensitivity: 0.28 pg/ml
• Dynamic Range: 0-200 pg/ml
• Assay Time: 4 hours 30 minutes
• Sample Type: Serum, Plasma, Cell Culture Supernatants, Urine
• Sample Volume: 100 µl
• Alternative Name: Interleukin 6
• Kit includes 7 pre-diluted calibrators and 2 controls

 

Sclerostin ELISA (cat. no. BI-20492) 

• Format: 12×8 wells
• Sensitivity: 3.2 pmol/l (= 72 pg/ml)
• Dynamic Range: 0 to 240 pmol/l (=0-5400 pg/ml)
• Assay Time: 18-25h (overnight incubation) / 1 h /30 min
• Sample Type: Serum, Plasma, Cell Culture Supernatants, Urine
• Sample Volume: 20 µl
• Alternative Name: SOST
• Kit includes 6 pre-diluted calibrators and 1 control

 

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Literature

1.Risk Factors Associated with Incident Vertebral Fractures in Steroid-treated Males with Duchenne Muscular Dystrophy. Phung K, McAdam L, Ma J, McMillan HJ, Jackowski S, Scharke M, Matzinger MA, Shenouda N, Koujok K, Jaremko JL, Wilson N, Walker S, Hartigan C, Khan N, Page M, Robinson ME, Saleh DS, Smit K, Rauch F, Siminoski K, Ward LM.J Clin Endocrinol Metab. 2023 Aug 23:dgad435. doi: 10.1210/clinem/dgad435. Epub ahead of print. PMID: 37610420.

Abstract

Purpose: Prevention of fractures is an unmet need in glucocorticoid (GC)-treated Duchenne muscular dystrophy. This study explored factors associated with incident vertebral fractures (VFs) to inform future fracture prevention efforts. Methods: VFs were evaluated prospectively at study baseline and 12 months on lateral spine radiographs in participants aged 4 to 25 years with Duchenne muscular dystrophy. Clinical factors were analyzed for their association with the change in Spinal Deformity Index (sum of the Genant-defined VF grades from T4 to L4) between baseline and 12 months. Results: Thirty-eight males were evaluated (mean ± SD age at baseline 11.0 ± 3.6 years; mean ± SD GC duration at baseline 4.1 ± 3.1 years; 74% ambulatory). Nine of 38 participants (24%) had 17 incident VFs, of which 3/17 VFs (18%) were moderate/severe. Participants with 12-month incident VF had lower mean ± SD baseline lumbar spine areal bone mineral density Z-scores (-2.9 ± 1.0 vs -1.9 ± 1.1; P = .049) and lower total body less head areal bone mineral density Z-scores (-3.1 ± 1.2 vs -1.6 ± 1.7; P = .036). Multivariable linear regression showed that at least 1 VF at baseline (P < .001), a higher number of antecedent non-VF (P < .001), and greater bone age delay at baseline (P = .027) were significant predictors of an increase in the Spinal Deformity Index from baseline to 12 months. Conclusion: The observation that ≥ 1 prevalent VF and/or non-VF were the strongest predictors of incident VFs at 12 months supports the need for prevention of first fractures in this high-risk setting. Bone age delay, a marker of GC exposure, may assist in the prioritization of patients in efforts to prevent first fractures.

2. Emerging therapies for Duchenne muscular dystrophy. Markati T, Oskoui M, Farrar MA, Duong T, Goemans N, Servais L. Lancet Neurol. 2022 Sep;21(9):814-829. doi: 10.1016/S1474-4422(22)00125-9. Epub 2022 Jul 15. PMID: 35850122.