Respiratory function and therapeutic expectations in DMD: Families experience and perspective

Marika Pane, Eugenio Maria Mercuri, Claudia Brogna, Simona Lucibello, Giorgia Coratti, Gloria Ferrantini, Elisa Pede, Gianluca Vita, Valeria A. Sansone, Sonia Messina, Emilio Albamonte, Francesca Salmin, Chiara Consulo

Research output: Contribution to journalArticle

Abstract

Objective. The aim of this study was to use a structured questionnaire in a large cohort of Duchenne Muscular Dystrophy (DMD) patients to assess caregivers and patients views on respiratory function and to establish if their responses were related to the patients' age or level of functional impairment. Methods. Questionnaires were administered to caregivers in 205 DMD patients of age between 3 and 36 years (115 ambulant, 90 non-ambulant), and to 64 DMD patients (3 ambulant, 61 non-ambulant) older than 18 years, subdivided into groups according to age, FVC, ambulatory and ventilatory status. Results. Some differences were found in relation to FVC % values (p = 0.014), ambulatory (p = 0.043) and ventilatory status (p = 0.014). Nearly half of the caregivers expected deterioration over the next years, with the perspective of deterioration more often reported by caregivers of non-ambulant (p = 0.018) and ventilated patients (p = 0.004). Caregivers appeared to be aware of the relevance of respiratory function on quality of life (84%) showing willingness to enter possible clinical trials if these were aiming to stabilize the progression of respiratory function with a very high number of positive responses across the spectrum of age, FVC, ambulatory and ventilatory status. The boys older than 18 years showed similar results. Conclusions. Our study showed that the concern for respiratory function increases with age and with the reduction of FVC or the need for ventilation, but the need for intervention was acknowledged across the whole spectrum of age and functional status.
Original languageEnglish
Pages (from-to)121-129
Number of pages9
JournalActa Myologica
Volume39
DOIs
Publication statusPublished - 2020

Keywords

  • Duchenne muscular dystrophy
  • Respiratory
  • Quality of life
  • Patient reported outcome measures

Fingerprint

Dive into the research topics of 'Respiratory function and therapeutic expectations in DMD: Families experience and perspective'. Together they form a unique fingerprint.

Cite this