Physical capacity in non-ambulatory people with Duchenne muscular dystrophy or spinal muscular atrophy: a longitudinal study

Abstract

The purpose of this study was to describe functional ability, muscle strength, forced vital capacity, and clinical events in participants with Duchenne muscular dystrophy (DMD) or spinal muscular atrophy (SMA) in the non-ambulatory stages of the diseases. Nineteen non-ambulatory participants with DMD (all males; 13 to 24 years) and 13 with SMA (six males, seven females; 11 to 57 years) were assessed once a year over 5 years. The assessments comprised functional ability measured with the EK scale and upper extremity grade, muscle strength measured with the manual muscle test, and forced vital capacity defined as a percentage of normal values (FVC%). In the DMD group all variables measured deteriorated and there was a direct correlation between them. In the SMA group only muscle strength and FVC% deteriorated and there was no close relation between the variables measured. In the DMD group, 16 participants had cardiorespiratory clinical events leading to death in five cases. In the SMA group only four participants had respiratory clinical events, none leading to death. Although the participants with SMA had been extremely weak and non-ambulatory since early childhood they were older and less exposed to life-threatening events than the participants with DMD.

Sniff nasal inspiratory pressure in the longitudinal assessment of young Duchenne muscular dystrophy children

“…This study included DMD children from a younger age (5 years) than previous studies [4,7]. Our PImax results, showing no significant evolution of PImax from 5 to 20 years of age, are in agreement with another longitudinal study [5] but differ from a transverse study showing a PImax increase from 7 to 14 years of age followed by a decrease in 15-18-year-old DMD patients [27].…”

“…Respiratory impairment in neuromuscular diseases is assessed by the measurement of maximal inspiratory pressure (PImax), slow vital capacity (VC) and, more recently, sniff nasal inspiratory pressure (SNIP). Studies describing longitudinal follow-up of respiratory function for o2 years in children with BMD or DMD have been based on PImax measurements [2][3][4] and lung volumes [2][3][4][5][6][7]. At the time of writing, there was no published prospective study describing longitudinal assessment of inspiratory muscle strength by SNIP measurements.…”

Duchenne muscular dystrophy: quality of life among 95 patients evaluated using the Life Satisfaction Index for Adolescents

“…DMD has a progressive and irreversible clinical course that initially is predominantly in the proximal limb-girdle muscles 5 . The symmetrical and progressive decline in muscle strength and motor skills over the course of time was well established by Scott et al 6 , and the annual decline in muscle strength is around 2% 7 .…”

O Uso das Escalas Funcionais para Avaliação Clínica da Distrofia Muscular de Duchenne

“…A avaliação compreendeu a capacidade funcional e o grau da extremidade superior medida pela EK, a força muscular medida por meio de teste manual e a capacidade vital forçada. De todas as variáveis, somente a EK obteve significância estatística, podendo discriminar aqueles que necessitaram de ventilação assistida e os que não necessitavam, durante todo o período pesquisado 24 .…”