6 Minute walk test in Duchenne MD patients with different mutations: 12 month changes.
Обработка статьи · этапы, ошибки и управление
Ошибки и ограничения
История · было → стало
Состояние обновляется каждые 5 секунд, пока блок открыт. Процент показывается только там, где известен объём работы. Пауза останавливает продолжение этапа; текущий запрос к источнику может завершиться.
Pane Marika, Mazzone Elena S, Sormani Maria Pia, Messina Sonia, Vita Gian Luca, Fanelli Lavinia, Berardinelli Angela, Torrente Yvan, D'Amico Adele, Lanzillotta Valentina, Viggiano Emanuela, D'Ambrosio Paola, Cavallaro Filippo, Frosini Silvia, Bello Luca, Bonfiglio Serena, Scalise Roberta, De Sanctis Roberto, Rolle Enrica, Bianco Flaviana, Van der Haawue Marlene, Magri Francesca, Palermo Concetta, Rossi Francesca, Donati Maria Alice, Alfonsi Chiara, Sacchini Michele, Arnoldi Maria Teresa, Baranello Giovanni, Mongini Tiziana, Pini Antonella, Battini Roberta, Pegoraro Elena, Previtali Stefano C, Napolitano Sara, Bruno Claudio, Politano Luisa, Comi Giacomo P, Bertini Enrico, Morandi Lucia, Gualandi Francesca, Ferlini Alessandra, Goemans Nathalie, Mercuri Eugenio
Аннотация
METHODS: The 6MWT was performed in 191 ambulant DMD boys at baseline and 12 months later. The results were analysed using a test for heterogeneity in order to establish possible differences among different types of mutations (deletions, duplications, point mutations) and among subgroups of deletions eligible to skip individual exons.
RESULTS: At baseline the 6MWD ranged between 180 and 560,80 metres (mean 378,06, SD 74,13). The 12 month changes ranged between -325 and 175 (mean -10.8 meters, SD 69.2). Although boys with duplications had better results than those with the other types of mutations, the difference was not significant. Similarly, boys eligible for skipping of the exon 44 had better baseline results and less drastic changes than those eligible for skipping exon 45 or 53, but the difference was not significant.
CONCLUSIONS: even if there are some differences among subgroups, the mean 12 month changes in each subgroup were all within a narrow Range: from the mean of the whole DMD cohort. This information will be of help at the time of designing clinical trials with small numbers of eligible patients.