Glucocorticoid Use in DMD Patients and Impact on Growth Effect

Patients with Duchenne muscular dystrophy (DMD) experience considerable impairments in their quality of life due to the severe impairment of their motor abilities and growth. Glucocorticoid therapies may halt the progression, but they may also impede growth. Find out more about treating the course of the disease and these individuals' quality of life.

A childhood hereditary disorder known as Duchenne muscular dystrophy (DMD) is characterized by increasing muscle weakening and degradation. It mainly affects men and results in a deficiency of dystrophin, which is essential for stable and strong muscles. Children with DMD have a limited life expectancy and frequently experience cardiac or respiratory failure before or during their 30s; regrettably, there is no known cure.

The musculoskeletal effects of DMD significantly impair patients’ motor skills, causing substantial impacts on quality of life. Additionally, short stature and low BMI (Body Mass Index( are common due to impaired growth and development arising from reduced muscle mass, which hinders bone turnover. This is typically exacerbated by glucocorticoid (GC) treatment, a cornerstone of DMD management.

Prednisone and deflazacort are examples of GC treatment that is used to increase muscle strength, extend walking, improve pulmonary function, and reduce inflammation to aid in muscle recovery. But long-term GC use can inhibit the generation of growth hormones, which can lead to stunted growth. It is still debatable whether growth suppression should be lessened or promoted.

Findings on Height Variations in Patients with DMD

Research indicates that the degree of growth impairment in DMD patients is influenced by the kind, dosage, and length of GC therapy. For instance, it has been demonstrated that in the ambulatory phase of DMD, an alternate prednisone regimen has no discernible negative effects on height or weight. Furthermore, DMD patients who get growth hormone therapy have shown increased development velocity without compromising their cardiac, pulmonary, or motor abilities.

Fascinatingly, studies suggest that persons with DMD may benefit clinically from being shorter. Young children with DMD can walk, but as they get older, they lose this capacity, indicating a decrease of function with height.

Height and clinical outcomes in male children with DMD were found to be significantly correlated in an early investigation. Over the years, related research has confirmed this, indicating that growth inhibition may be a useful strategy for delaying the course of DMD. This phenomena is probably caused by the mechanical strain brought on by the exponential increase in load per muscle fiber, which accelerates the loss of muscle fibers and damages structures. Consequently, reducing stress on muscle fibers and preserving their function can be achieved by using non-weight-bearing activities and keeping a controlled weight.

For both patients and caretakers, DMD poses particular emotional and physical difficulties. Plans for treatment should be customized for each patient, taking into account both the possible effects on growth and the advantages of GC therapy. A thorough approach that takes into account both illness progression and quality of life is still crucial as science advances.

Read More: FDA-Approved Treatments for DMD (Duchenne Muscular Dystrophy)

References:

  1. Why short stature is beneficial in Duchenne muscular dystrophy.
  2. Growth hormone therapy for children with Duchenne muscular dystrophy and glucocorticoid induced short stature.
  3. Short stature in Duchenne muscular dystrophy: a study of 34 patients.
  4. Normal height and weight in a series of ambulant Duchenne muscular dystrophy patients using the 10 day on/10 day off prednisone regimen.
  5. Growth, pubertal development, and skeletal health in boys with Duchenne Muscular Dystrophy.
  6. Relation between height and clinical course in Duchenne muscular dystrophy.

LEAVE A REPLY

Please enter your comment!
Please enter your name here


Hot Topics

Related Articles