Upper limb disease evolution in exon 53 skipping eligible patients with Duchenne muscular dystrophy.
Ann Clin Transl Neurol
; 8(10): 1938-1950, 2021 10.
Article
em En
| MEDLINE
| ID: mdl-34453498
ABSTRACT
OBJECTIVE:
To understand the natural disease upper limb progression over 3 years of ambulatory and non-ambulatory patients with Duchenne muscular dystrophy (DMD) using functional assessments and quantitative magnetic resonance imaging (MRI) and to exploratively identify prognostic factors.METHODS:
Forty boys with DMD (22 non-ambulatory and 18 ambulatory) with deletions in dystrophin that make them eligible for exon 53-skipping therapy were included. Clinical assessments, including Brooke score, motor function measure (MFM), hand grip and key pinch strength, and upper limb distal coordination and endurance (MoviPlate), were performed every 6 months and quantitative MRI of fat fraction (FF) and lean muscle cross sectional area (flexor and extensor muscles) were performed yearly.RESULTS:
In the whole population, there were strong nonlinear correlations between outcome measures. In non-ambulatory patients, annual changes over the course of 3 years were detected with high sensitivity standard response mean (|SRM| ≥0.8) for quantitative MRI-based FF, hand grip and key pinch, and MFM. Boys who presented with a FF<20% and a grip strength >27% were able to bring a glass to their mouth and retained this ability in the following 3 years. Ambulatory patients with grip strength >35% of predicted value and FF <10% retained ambulation 3 years later.INTERPRETATION:
We demonstrate that continuous decline in upper limb strength, function, and MRI measured muscle structure can be reliably measured in ambulatory and non-ambulatory boys with DMD with high SRM and strong correlations between outcomes. Our results suggest that a combination of grip strength and FF can be used to predict important motor milestones.
Texto completo:
1
Base de dados:
MEDLINE
Assunto principal:
Força da Mão
/
Progressão da Doença
/
Distrofia Muscular de Duchenne
/
Extremidade Superior
/
Adiposidade
Tipo de estudo:
Prognostic_studies
Limite:
Adolescent
/
Child
/
Humans
/
Male
Idioma:
En
Revista:
Ann Clin Transl Neurol
Ano de publicação:
2021
Tipo de documento:
Article
País de afiliação:
França