Report on the Second Endocrine Aspects Of Duchenne Muscular Dystrophy Conference December 1-2, 2010, Baltimore, Maryland, USA (original) (raw)
Related papers
Muscular Dystrophies at Different Ages: Metabolic and Endocrine Alterations
International Journal of Endocrinology, 2012
Common metabolic and endocrine alterations exist across a wide range of muscular dystrophies. Skeletal muscle plays an important role in glucose metabolism and is a major participant in different signaling pathways. Therefore, its damage may lead to different metabolic disruptions. Two of the most important metabolic alterations in muscular dystrophies may be insulin resistance and obesity. However, only insulin resistance has been demonstrated in myotonic dystrophy. In addition, endocrine disturbances such as hypogonadism, low levels of testosterone, and growth hormone have been reported. This eventually will result in consequences such as growth failure and delayed puberty in the case of childhood dystrophies. Other consequences may be reduced male fertility, reduced spermatogenesis, and oligospermia, both in childhood as well as in adult muscular dystrophies. These facts all suggest that there is a need for better comprehension of metabolic and endocrine implications for muscular...
Corticosteroid Treatment and Growth Patterns in Ambulatory Males with Duchenne Muscular Dystrophy
The Journal of pediatrics, 2016
To evaluate growth patterns of ambulatory males with Duchenne muscular dystrophy (DMD) treated with corticosteroids compared with ambulatory, steroid-naïve males with DMD and age-matched unaffected general-population males and to test associations between growth and steroid treatment patterns among treated males. Using data from the Muscular Dystrophy Surveillance, Tracking, and Research Network, we identified a total of 1768 height, 2246 weight, and 1755 body mass index (BMI) measurements between age 2 and 12 years for 324 ambulatory males who were treated with corticosteroids for at least 6 months. Growth curve comparisons and linear mixed-effects modeling, adjusted for race/ethnicity and birth year, were used to evaluate growth and steroid treatment patterns (age at initiation, dosing interval, duration, cumulative dose). Growth curves for ambulatory males treated with corticosteroids showed significantly shorter stature, heavier weight, and greater BMI compared with ambulatory, ...
The effect of steroid treatment on weight in nonambulatory males with Duchenne muscular dystrophy
American journal of medical genetics. Part A, 2018
To describe the long-term effect of steroid treatment on weight in nonambulatory males with Duchenne Muscular Dystrophy (DMD), we identified 392 males age 7-29 years with 4,512 weights collected after ambulation loss (176 steroid-naïve and 216 treated with steroids ≥6 months) from the Muscular Dystrophy Surveillance, Tracking, and Research Network (MD STARnet). Comparisons were made between the weight growth curves for steroid-naïve males with DMD, steroid-treated males with DMD, and the US pediatric male population. Using linear mixed-effects models adjusted for race/ethnicity and birth year, we evaluated the association between weight-for-age and steroid treatment characteristics (age at initiation, dosing interval, cumulative duration, cumulative dose, type). The weight growth curves for steroid-naïve and steroid-treated nonambulatory males with DMD were wider than the US pediatric male growth curves. Mean weight-for-age z scores were lower in both steroid-naïve (mean = -1.3) and...
Muscle & nerve, 2013
Contemporary natural history data in Duchenne muscular dystrophy (DMD) is needed to assess care recommendations and aid in planning future trials. The Cooperative International Neuromuscular Research Group (CINRG) DMD Natural History Study (DMD-NHS) enrolled 340 individuals, aged 2-28 years, with DMD in a longitudinal, observational study at 20 centers. Assessments obtained every 3 months for 1 year, at 18 months, and annually thereafter included: clinical history; anthropometrics; goniometry; manual muscle testing; quantitative muscle strength; timed function tests; pulmonary function; and patient-reported outcomes/health-related quality-of-life instruments. Glucocorticoid (GC) use at baseline was 62% present, 14% past, and 24% GC-naive. In those ≥6 years of age, 16% lost ambulation over the first 12 months (mean age 10.8 years). Detailed information on the study methodology of the CINRG DMD-NHS lays the groundwork for future analyses of prospective longitudinal natural history dat...
Journal of the American Medical Directors Association, 2016
Introduction: Aging is a process that involves a reduction in muscle strength and anabolic hormone concentrations, which impacts significantly on health. Aim: To study the hormone/total strength (H/TS) ratio as a proxy of anabolic insensitivity status in elders, and its relationship with disability, hospitalization, and mortality risk. Design: A total of 1462 persons aged !65 years from the Toledo Study of Healthy Aging participated in this study. Serum concentrations of insulin like growth factor 1, total and free testosterone, dehydroepiandrosterone, dehydroepiandrosterone sulfate, and 17b-estradiol were measured. Total maximal voluntary isometric strength was obtained (handgrip, shoulder, hip, and knee) using standardized techniques and equipment. Physical activity was recorded by physical activity scale for the elderly questionnaire. Associations of the H/TS ratio with hospitalization and mortality were assessed using logistic regression models, and participants stratified into quartiles for each H/TS ratio. Results: In women, all individual ratio H/TS models showed a strong to moderate increased risk for death and hospitalization. In men, all models revealed a significant positive association of the ratio H/TS with mortality rate but not for hospitalization (P < .01). Participants who have 2 or more H/TS ratios in the worst quartile increased the risk of hospitalization and mortality at least by 2-fold. Conclusions: We demonstrate the main role that muscle function plays in the relationship between the hormonal status and hospitalization and mortality risk; this could be taken into consideration as a way to classify patients for hormonal therapy.
The role of corticosteroids in muscular dystrophy: A critical appraisal
Muscle & Nerve, 2007
Over the years various steroid trials have been conducted in Duchenne muscular dystrophy (DMD). In children who are still able to walk as well as in those who are wheelchair-bound, corticosteroids have been found to stabilize muscle strength for a period of time. Controlled clinical observations have shown that some boys remain ambulatory for years longer than reported in natural history data. The two main steroids used are prednisone/prednisolone and deflazacort. They are probably equally effective in stabilizing muscle strength but may have different side-effect profiles; for instance, deflazacort causes less weight gain. The exact mechanism by which steroids slow the dystrophic process is under investigation. DMD children treated long term also seem to develop other complications of the condition less frequently. For instance, they develop respiratory insufficiency later and have fewer cardiac symptoms. The therapeutic value of corticosteroids is limited, but these drugs represent the best treatment option currently available.
Neuromuscular Disorders, 2014
Steroids are nowadays routinely used as a long-term treatment in Duchenne muscular dystrophy (DMD). Their effects on body composition were assessed using dual X-ray absorptiometry. The study followed over 2 years 29 genetically confirmed DMD patients: 21 in the steroid-treated group and 8 in the steroid-naïve group. After 2 years of steroid treatment, the lean tissue mass values increased significantly (p < 0.0001), the percentage of body fat mass remained practically constant (p = 0.94) in comparison with the initial visit. In the steroid-naïve patients, there were no significant increases in the lean tissue mass but deterioration in body composition confirmed by a significant increase in the percentage of body fat mass. Besides, significant negative correlations were found between the percentage of body fat mass and the MFM total score (R = À0.79, n = 76, p < 0.0001). A 2-year steroid treatment improves significantly body composition of boys with DMD through a significant increase in lean tissue mass. We suggest that a thorough check of body composition should be carried out before steroid treatment discontinuation in case of overweight gain.