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?

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

Research output: Contribution to journalArticle

Abstract

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.
Original languageEnglish
Pages (from-to)567-574
Number of pages8
JournalJournal of Neuromuscular Diseases
Volume10
DOIs
Publication statusPublished - 2023

Keywords

  • Duchenne muscular dystrophy
  • PUL 2.0
  • performance of upper limb
  • functional abilities
  • ambulatory status

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