Physical training and high-protein diet improved muscle strength, parent-reported fatigue, and physical quality of life in children with Pompe disease.
Study Goal
The researchers aimed to investigate the effects of a 12-week tailored lifestyle intervention, including a high-protein diet (2 grams/kg), on exercise capacity and other health outcomes in children with Pompe disease.
Results Summary
The intervention improved muscle strength, core stability, quality of life, and parent-reported fatigue, with significant increases in absolute Peak VO2 and specific muscle groups' strength. However, improvements in exercise capacity were not significant compared to the control period.
Population
Children with Pompe disease (median age 10.6 years, including six classic infantile patients).
Effective Dosage
2 grams/kg of body weight.
Duration
12 weeks.
Interactions
None mentioned.
| Intervention | Direction | Endpoint | Population | Dosage | Impact | Claim # |
|---|---|---|---|---|---|---|
a 12-week tailored lifestyle intervention, consisting of physical training and a high protein diet (2 grams/kg) | no change | safety | children with Pompe disease | - | seemed safe | #1 |
a 12-week tailored lifestyle intervention, consisting of physical training and a high protein diet (2 grams/kg) | increase | absolute Peak VO2 | children with Pompe disease | 1279 mL/min [1012.5-2006] vs. 1352 mL/min [1101.5-2069] | improved significantly | #2 |
a 12-week tailored lifestyle intervention, consisting of physical training and a high protein diet (2 grams/kg) | increase | Muscle strength of the hip flexors, hip abductors, elbow extensors, neck extensors, knee extensors | children with Pompe disease | - | improved significantly | #3 |
a 12-week tailored lifestyle intervention, consisting of physical training and a high protein diet (2 grams/kg) | increase | core stability | children with Pompe disease | - | improved significantly | #4 |
a 12-week tailored lifestyle intervention, consisting of physical training and a high protein diet (2 grams/kg) | increase | the change in health domain of quality of life | children with Pompe disease | - | reported a significant increase | #5 |
a 12-week tailored lifestyle intervention, consisting of physical training and a high protein diet (2 grams/kg) | increase | quality of life domains: physical functioning, change in health, family cohesion | children with Pompe disease | - | reported significantly better scores | #6 |
a 12-week tailored lifestyle intervention, consisting of physical training and a high protein diet (2 grams/kg) | decrease | fatigue | children with Pompe disease | - | reported significantly better scores | #7 |
a 12-week tailored lifestyle intervention, consisting of physical training and a high protein diet (2 grams/kg) | increase | muscle strength | children with Pompe disease | - | led to improvements | #8 |
a 12-week tailored lifestyle intervention, consisting of physical training and a high protein diet (2 grams/kg) | increase | core stability | children with Pompe disease | - | led to improvements | #9 |
a 12-week tailored lifestyle intervention, consisting of physical training and a high protein diet (2 grams/kg) | increase | quality of life | children with Pompe disease | - | led to improvements | #10 |
a 12-week tailored lifestyle intervention, consisting of physical training and a high protein diet (2 grams/kg) | decrease | parent-reported fatigue | children with Pompe disease | - | led to improvements | #11 |
a 12-week tailored lifestyle intervention, consisting of physical training and a high protein diet (2 grams/kg) | increase | disease trajectory | Pompe patients with a stable disease trajectory | - | seemed to benefit the most | #12 |
Exercise has proven to be an effective adjuvant treatment to enzyme replacement therapy (ERT) in mildly affected adult Pompe patients. The aim of this study was to investigate the effects of a 12-week tailored lifestyle intervention, consisting of physical training and a high protein diet (2 grams/kg), in children with Pompe disease. This randomized controlled semi-crossover trial investigated the effects of a lifestyle intervention on the primary outcome: exercise capacity. Secondary outcomes were: muscle strength, core stability, motor function, physical activity levels, quality of life, fatigue, fear of exercise, caloric intake, energy balance, body composition, and safety. Fourteen Pompe patients with a median age of 10.6 [IQR: 7.2-14.5], of whom six classic infantile patients, participated in the lifestyle intervention. At baseline, patients had a lower exercise capacity compared to healthy peers (median 70.3% [IQR: 54.8%-98.6%] of predicted). After the intervention, absolute Peak VO2 improved significantly (1279 mL/min [1012.5-2006] vs. 1352 mL/min [1101.5-2069], p = 0.039), but not compared to the control period. Muscle strength of the hip flexors, hip abductors, elbow extensors, neck extensors, knee extensors, and core stability improved significantly compared to the control period. Children reported a significant increase on the change in health domain of quality of life, parents reported significantly better scores on the quality of life domains: physical functioning, change in health, family cohesion, and fatigue. A 12-week tailored lifestyle intervention for children with Pompe disease seemed safe and led to improvements in muscle strength, core stability, quality of life, and parent-reported fatigue. Pompe patients with a stable disease trajectory seemed to benefit the most from the intervention.