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Measurements of Motor Function and Other Clinical Outcome Parameters in Ambulant Children with Duchenne Muscular Dystrophy
Published on: January 12, 2019
Clinical Profile and Pulmonary Function of Pediatric Patients with Duchenne Muscular Dystrophy at a Tertiary
Maria L Arquillo1, Elbert John V Layug2, Maria Cristina H Lozada1
1Division of Pediatric Pulmonology, Department of Pediatrics, Philippine General Hospital, University of the Philippines Manila.
Insights
This study assessed the clinical profile and pulmonary function in Filipino pediatric patients with Duchenne Muscular Dystrophy (DMD). Spirometry revealed patterns of restrictive lung disease, though no significant differences were found across disease stages.
Area of Science:
- Pediatric Pulmonology
- Neuromuscular Disorders
- Clinical Research
Background:
- Duchenne Muscular Dystrophy (DMD) is a progressive genetic disorder affecting muscle strength, including respiratory muscles.
- Pulmonary complications are a major cause of morbidity and mortality in DMD patients.
- Understanding the clinical profile and pulmonary function in pediatric DMD is crucial for management.
Purpose of the Study:
- To determine the clinical profile and pulmonary function of pediatric patients with Duchenne Muscular Dystrophy (DMD).
- To characterize disease progression stages in DMD.
- To investigate the association between disease progression and spirometry variables.
Main Methods:
- Cross-sectional study utilizing medical record review.
- Inclusion of pediatric patients (0-18 years) with DMD.
- Data collected from August 2018 to March 2020 at a tertiary government hospital.
Main Results:
- 30 pediatric DMD patients were analyzed, grouped by disease stage.
- Common comorbidities included overweight, obesity, and scoliosis in non-ambulatory patients.
- Sleep-disordered breathing symptoms were prevalent; spirometry showed patterns of restrictive lung disease (mean FVC 66.2%, FEV1 67.7%).
- No significant differences in pulmonary function were observed across disease progression stages.
Conclusions:
- This is the first study on Filipino pediatric DMD patients' pulmonary function.
- Spirometry patterns suggest restrictive lung disease.
- Further prospective studies are needed to identify respiratory variables correlating with pulmonary function.
Objective:
Our study aimed to determine the clinical profile and pulmonary function of pediatric patients with Duchenne Muscular Dystrophy (DMD). We also characterized the stages of progression of the disease and determined their potential association with spirometry variables.
Methods:
In this cross-sectional study, we used data obtained from a review of medical records of all pediatric patients (0-18 years old) with DMD seen in a multidisciplinary neuromuscular clinic of a tertiary government hospital from August 2018 until March 2020.
Results:
Included were 30 patients subdivided into groups according to the stage of disease progression. Overweight (26.7%), obesity (20%), and scoliosis (26.7%) were common among non-ambulatory patients. Only one late ambulatory patient had evidence of ineffective airway clearance. Symptoms of sleep-disordered breathing, particularly snoring (66.7%) and apnea (6.7%), were common across all disease stages. All patients had normal peripheral oxygen saturation on room air. The mean peak expiratory flow rate was 215.6 (±84) L/min. The mean Forced Vital Capacity (FVC), Forced Expiratory Volume in the first second (FEV1), and FEV1/FVC were 66.2% (±23.7), 67.7% (±23.8), and 97.5 (±3.2), respectively. Among patients with polysomnography results, the average apnea-hypopnea index (AHI) per hour was 3 (±1.6). When patients were compared according to their stage disease progression, however, no significant differences exist.
Conclusion:
This is the first study on the pulmonary function of Filipino pediatric patients with DMD. Spirometry patterns characteristic of restrictive lung disease were observed. Prospective studies may help identify respiratory variables that significantly correlate with pulmonary function.
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