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Journal of neuromuscular diseases · 2023

Longitudinal Analysis of PUL 2.0 Domains in Ambulant and Non-Ambulant Duchenne Muscular Dystrophy Patients: How do they Change in Relation to Functional Ability?

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Pane Marika, Coratti Giorgia, Brogna Claudia, Bovis Francesca, D'Amico Adele, Pegoraro Elena, Bello Luca, Sansone Valeria, Albamonte Emilio, Ferraroli Elisabetta, Mazzone Elena Stacy, Fanelli Lavinia, Messina Sonia, Catteruccia Michela, Cicala Gianpaolo, Ricci Martina, Frosini Silvia, De Luca Giacomo, Rolle Enrica, De Sanctis Roberto, Forcina Nicola, Norcia Giulia, Passamano Luigia, Gardani Alice, Pini Antonella, Monaco Giulia, D'Angelo Maria Grazia, Capasso Anna, Leone Daniela, Zanin Riccardo, Vita Gian Luca, Panicucci Chiara, Bruno Claudio, Mongini Tiziana, Ricci Federica, Berardinelli Angela, Battini Roberta, Masson Riccardo, Baranello Giovanni, Dosi Claudia, Bertini Enrico, Politano Luisa, Mercuri Eugenio

Аннотация

BACKGROUND: The performance of upper limb 2.0 (PUL) is widely used to assess upper limb function in DMD patients. The aim of the study was to assess 24 month PUL changes in a large cohort of DMD patients and to establish whether domains changes occur more frequently in specific functional subgroups.

METHODS: The PUL was performed in 311 patients who had at least one pair of assessments at 24 months, for a total of 808 paired assessments. Ambulant patients were subdivided according to the ability to walk: >350, 250-350, ≤250 meters. Non ambulant patients were subdivided according to the time since they lost ambulation: <1, 1-2, 2-5 or >5 years.

RESULTS: At 12 months, the mean PUL 2.0 change on all the paired assessments was -1.30 (-1.51--1.05) for the total score, -0.5 (-0.66--0.39) for the shoulder domain, -0.6 (-0.74--0.5) for the elbow domain and -0.1 (-0.20--0.06) for the distal domain.At 24 months, the mean PUL 2.0 change on all the paired assessments was -2.9 (-3.29--2.60) for the total score, -1.30 (-1.47--1.09) for the shoulder domain, -1.30 (-1.45--1.11) for the elbow domain and -0.4 (-1.48--1.29) for the distal domain.Changes at 12 and 24 months were statistically significant between subgroups with different functional abilities for the total score and each domain (p < 0.001).

CONCLUSION: There were different patterns of changes among the functional subgroups in the individual domains. The time of transition, including the year before and after loss of ambulation, show the peak of negative changes in PUL total scores that reflect not only loss of shoulder but also of elbow activities. These results suggest that patterns of changes should be considered at the time of designing clinical trials.

Предметные рубрики MeSH

ИП Урсолов Владлен Сергеевич

ИНН 500118482352 · ОГРНИП 314500117700049

Реквизиты и контакты