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Neuromuscular disorders : NMD · 2026

Development of Italian centile curves for the North Star Ambulatory Assessment and timed items.

СтатьяНазвание и аннотация: Перевод пока не подготовлен
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Paolucci Sophia, Lentini Nicolò, Coratti Giorgia, Ricci Federica S, Rolle Enrica, Mongini Tiziana E, Gadaleta Giulio, Trucco Federica, Pedemonte Marina, Bruno Claudio, Morando Simone, Diella Eleonora, D'Angelo Grazia, Ghezzi Carlo, Catteruccia Michela, D'Amico Adele, De Luca Giacomo, Mizzoni Irene, Dosi Claudia, Zanin Riccardo L, Masson Riccardo, Sansone Valeria A, Albamonte Emilio, Maroni Sofia, Giacompolli Irene, Berardinelli Angela, Dragoni Francesca, Gardani Alice, Nigro Vincenzo, Picillo Esther, Messina Sonia, Sframeli Maria, Romeo Marika, Giannotta Melania, Pini Antonella, Frosini Silvia, Battini Roberta, Buchignani Bianca, Comi Giacomo, Magri Francesca, Pedrazzini Alessandra, Previtali Stefano C, Zambon Alberto, Ricci Giulia, Torri Francesca, Bello Luca, Pane Marika, Pastorino Roberta, Mercuri Eugenio, Italian DMD Registry

Аннотация

Centile curves may improve the interpretation of motor outcomes in specific populations. This study aimed to develop Italian age-specific centile curves for NSAA, rise-from-floor velocity (RFFV), and 10-meter walk/run velocity (10MWRV) in boys with Duchenne muscular dystrophy (DMD) and to assess their agreement with existing UK reference curves. Data from 627 patients (5080 assessments) with an age between 3-18 years were extracted from the Italian DMD Registry. GAMLSS models were used to estimate centile curves. Agreement with UK data (restricted to ages 5-16 years and who are actively on steroids) was evaluated using SDS deviation and percentile coverage analyses. NSAA scores peaked at approximately 6 years; RFFV and 10MWRV peaked at 5 years, then progressively declined. The Italian cohort showed small deviations from the UK centiles (all p<0.001): mean SDS values were significantly different across all outcomes, and age was significantly associated with SDS deviation through both linear and quadratic effects. The Italian centile curves extend reference data to wider age ranges. Available for both general and steroid-treated patients, they support clinical trials and real-world practice. The minor differences observed, especially in timed velocities, highlight the need for future international harmonisation.