Efficacia del Neridronato nella Distrofia Muscolare di Duchenne: risultati di uno studio monocentrico

Abstract

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In Duchenne muscular dystrophy (DMD), muscle degeneration and oral glucocorticoid (GC) treatment lead to secondary osteoporosis and fragility fractures (FF). The first-line therapy for osteoporosis are bisphosphonates; alendronate, clodronate, neridronate, pamidronate, risendronate and zolendronate have been previously investigated in DMD patients; neridronate, already used for osteogenesis imperfecta has only been used for 12 months in a cohort of eight DMD patients. The aim of this study was to evaluate efficacy of neridronate in GC-treated DMD patients followed at Institute Giannina Gaslini presenting with FF, focusing on bone mineral density, fracture incidence and treatment tolerability. We conducted a retrospective, single-center study; lumbar spine (LS), total body less head (TB) bone mineral density (BMD), bone mineral content (BMC), LS bone mineral apparent density (BMAD) were assessed by dual-energy X-ray absorptiometry before treatment (T0) and after 24 (T24) and 36 months (T36). Spine X-ray was performed to quantify vertebral fractures, according to Genant score. 31/86 patients were enrolled in the study. 16/31 had vertebral fractures, 15/31 had long-bone fractures; 8/31 reported multiple fractures at T0. Fractures occurred at a median age of 12.2 years. At T0, the median age was 14 years. Twenty and 16 patients completed a 24 and 36-month follow up. A significant improvement in LS BMD and BMD Z-score (+ 0.49 SDS at T24, + 0.76 SDS at T36), BMC and BMAD (+0.02 at T24, +0.049 at T36) was observed over time. In contrast, TB BMD and BMD Z-scores didn't significantly change. Two and one patients at T24 and T36 presented incident fractures. Neridronate was well tolerated, with only mild adverse events (arthralgia, fever), and asymptomatic hypocalcemia. In GC-treated DMD patients presenting with FF, neridronate was safe and associated with a significant improvement in LS BMD, low incidence of new fragility fractures, supporting its potential in DMD patients