Skip to main content

Advertising Disclaimer »

Main menu

  • Journals
    • Pediatrics
    • Hospital Pediatrics
    • Pediatrics in Review
    • NeoReviews
    • AAP Grand Rounds
    • AAP News
  • Authors/Reviewers
    • Submit Manuscript
    • Author Guidelines
    • Reviewer Guidelines
    • Open Access
    • Editorial Policies
  • Content
    • Current Issue
    • Online First
    • Archive
    • Blogs
    • Topic/Program Collections
    • AAP Meeting Abstracts
  • Pediatric Collections
    • COVID-19
    • Racism and Its Effects on Pediatric Health
    • More Collections...
  • AAP Policy
  • Supplements
  • Multimedia
    • Video Abstracts
    • Pediatrics On Call Podcast
  • Subscribe
  • Alerts
  • Careers
  • Other Publications
    • American Academy of Pediatrics

User menu

  • Log in
  • My Cart

Search

  • Advanced search
American Academy of Pediatrics

AAP Gateway

Advanced Search

AAP Logo

  • Log in
  • My Cart
  • Journals
    • Pediatrics
    • Hospital Pediatrics
    • Pediatrics in Review
    • NeoReviews
    • AAP Grand Rounds
    • AAP News
  • Authors/Reviewers
    • Submit Manuscript
    • Author Guidelines
    • Reviewer Guidelines
    • Open Access
    • Editorial Policies
  • Content
    • Current Issue
    • Online First
    • Archive
    • Blogs
    • Topic/Program Collections
    • AAP Meeting Abstracts
  • Pediatric Collections
    • COVID-19
    • Racism and Its Effects on Pediatric Health
    • More Collections...
  • AAP Policy
  • Supplements
  • Multimedia
    • Video Abstracts
    • Pediatrics On Call Podcast
  • Subscribe
  • Alerts
  • Careers

Discover Pediatric Collections on COVID-19 and Racism and Its Effects on Pediatric Health

American Academy of Pediatrics
SUPPLEMENT ARTICLE

Obesity and Endocrine Management of the Patient With Duchenne Muscular Dystrophy

David R. Weber, Stasia Hadjiyannakis, Hugh J. McMillan, Garey Noritz and Leanne M. Ward
Pediatrics October 2018, 142 (Supplement 2) S43-S52; DOI: https://doi.org/10.1542/peds.2018-0333F
David R. Weber
aGolisano Children’s Hospital, School of Medicine and Dentistry, University of Rochester, Rochester, New York;
  • Find this author on Google Scholar
  • Find this author on PubMed
  • Search for this author on this site
Stasia Hadjiyannakis
bDepartment of Pediatrics, Children’s Hospital of Eastern Ontario, University of Ottawa, Ottawa, Ontario, Canada; and
  • Find this author on Google Scholar
  • Find this author on PubMed
  • Search for this author on this site
Hugh J. McMillan
bDepartment of Pediatrics, Children’s Hospital of Eastern Ontario, University of Ottawa, Ottawa, Ontario, Canada; and
  • Find this author on Google Scholar
  • Find this author on PubMed
  • Search for this author on this site
Garey Noritz
cNationwide Children’s Hospital, The Ohio State University, Columbus, Ohio
  • Find this author on Google Scholar
  • Find this author on PubMed
  • Search for this author on this site
Leanne M. Ward
bDepartment of Pediatrics, Children’s Hospital of Eastern Ontario, University of Ottawa, Ottawa, Ontario, Canada; and
  • Find this author on Google Scholar
  • Find this author on PubMed
  • Search for this author on this site
  • Article
  • Figures & Data
  • Info & Metrics
  • Comments
Loading
Download PDF

Abstract

Duchenne muscular dystrophy (DMD) is associated with an increased risk of endocrine complications due to the effects of prolonged glucocorticoid therapy as well as progressive muscle weakness. Categories of complications include obesity and its comorbidities, short stature, pubertal delay, and adrenal insufficiency. Obesity prevention is important for long-term management of patients with DMD. Preventing glucocorticoid-induced weight gain fosters patient mobility, ease of transfer, and reduces sleep-disordered breathing. Metabolic complications from obesity (glucose intolerance, dyslipidemia) also can be avoided. Short stature and pubertal delay may negatively affect self-esteem and peer relationships, and careful monitoring of growth and pubertal development can allow anticipatory counseling. Adrenal insufficiency, a potentially life-threatening complication associated with prolonged glucocorticoid use, must be recognized so as to allow prompt treatment. In this article, we provide a summary of current guidance to ensure comprehensive endocrine management is followed in patients with DMD.

  • Abbreviations:
    DMD —
    Duchenne muscular dystrophy
    HPA —
    hypothalamic-pituitary-adrenal
    RCT —
    randomized controlled trial
    rhGH —
    recombinant human growth hormone
  • The effect of glucocorticoid therapy on the clinical phenotype in patients with Duchenne muscular dystrophy (DMD) has led to the need for clear recommendations about weight management and the monitoring and treatment of endocrine complications. The appetite-promoting and fat deposition effects of GC therapy are prominent in this condition and perpetuate serious obesity-related comorbidities such as diabetes, dyslipidemia, and sleep apnea. Glucocorticoid therapy also suppresses the hypothalamic and pituitary axes, causing an often profound linear growth failure and marked delay in pubertal development. The linear growth failure of DMD is further affected by a direct adverse effect of glucocorticoid therapy on growth plate function. Glucocorticoid dependence due to adrenal insufficiency is another serious consequence of the high-dose glucocorticoid therapy that is typically prescribed in DMD.

    All of these issues (obesity, growth failure, delayed puberty, and adrenal insufficiency) can adversely affect quality of life, and adequate treatment of adrenal insufficiency in patients with glucocorticoid dependence is a potential life-saving measure.1 In this review, we target pediatricians, neurologists, endocrinologists, and weight management clinicians involved in the care of patients with DMD by providing specific guidance on clinical practice in these areas. With appropriate prevention and treatment of glucocorticoid-related comorbidities, it is anticipated that the need to withdraw glucocorticoid therapy to treat the underlying disease will be largely or wholly obviated.

    Overweight, Obesity, and Related Comorbidities

    Vulnerability to obesity is largely driven by genetic, prenatal, developmental, and psychosocial risk factors. In individuals with DMD, this risk is increased further because of glucocorticoid use, decreased mobility, and reduced energy expenditure due to limited options for physical activity. Increased time and financial pressures for families with children who have special health care needs such as DMD also can contribute to risk of obesity and create barriers to successful weight management.2 Once weight is gained, biologic adaptations make it extremely difficult to lose weight and therefore prevention strategies are critical.3 In individuals with DMD, obesity can negatively affect mobility and physical functioning, resulting in falls and fractures; obesity can also exacerbate the metabolic complications associated with glucocorticoid use. Therefore, it is imperative that both obesity prevention strategies and management are incorporated into the care of individuals with DMD.

    Little research has been conducted in the area of obesity prevention or management strategies in DMD and in children with physical and developmental disabilities in general.4 Family-based behavioral interventions have shown modest reduction in BMI in the short-term for children and youth with obesity in the absence of developmental and physical disabilities.5,6 One small study (n = 3) on the treatment of obesity in children with DMD that targeted parents revealed inconsistent changes in body weight, increases in children’s perceived quality of life, and increases in healthy foods available at home. However, a larger randomized controlled trial (RCT) is needed to more effectively examine the efficacy of such an approach.7 Two small pharmacotherapeutic trials have been conducted, one with metformin8 and the other with topimarate,9 but there remains insufficient evidence regarding the benefit and safety of these medications in this clinical context. Given the paucity of evidence and research in this area, the unique determinants of obesity in individuals with DMD should be used to guide effective obesity prevention and management strategies that can feasibly be implemented in their care.

    Obesity Prevention

    Obesity prevention strategies should be introduced at 3 key time points: (1) diagnosis, (2) time of glucocorticoid initiation, and (3) time of loss of mobility. Particular attention should be given to patients who have a family history of obesity, given the highly hereditary nature of obesity.10 An increase in weight or BMI z score of ≥0.5 also should trigger intervention.11

    Obesity prevention strategies should aim to facilitate a healthy home food environment of mostly whole foods, regular and predictable times for meals and snacks that are mostly home prepared, family meals, limits on sugar-sweetened beverage consumption and restaurant meals, and encouragement of screen-free meals and snacks at the table. This should be done in consultation with a dietitian. Obesity prevention strategies also should include counseling around appropriate sleep hygiene and duration, limitations on screen time, and psychosocial assessments and support for both the patients and their caregivers.12,13

    Although physical activity is a mainstay of obesity prevention strategies, the approach to physical activity recommendations in patients with DMD is more measured, and the role of physical activity in DMD remains controversial.14 To avoid disuse atrophy and other secondary complications of inactivity, it is necessary that those who are ambulatory or in the early nonambulatory stage participate in gentle functional strengthening activity, including a combination of swimming pool exercises and recreation-based exercises in the community. Swimming is highly recommended from the early ambulatory to early nonambulatory phase and could be continued in the nonambulatory phase as long as it is medically safe. Additional benefits might be provided by low-resistance strength training and optimization of upper body function. Significant muscle pain or myoglobulinuria in the 24-hour period after a specific activity is a sign of overexertion and that the activity should be modified.15

    Monitoring of Weight Status

    Monitoring weight status should include measuring body weight and linear height in ambulatory patients or arm span and segmental length in nonambulatory patients at least every 6 months. Both BMI and weight should be plotted on the appropriate curve to determine the percentile for age. Optimal weight status is defined as BMI between the 10th and 85th percentiles. If height is unavailable or there are significant concerns about its accuracy, weight for age can be used, and an appropriate weight gain trajectory as per the growth curve can be another indicator of optimal weight gain. An increase in weight for age or BMI z scores by >0.5 between visits should raise concern and prompt intervention.11

    Although body composition is altered in individuals with DMD (lower lean body mass and higher fat mass), the role of direct measurement of body composition remains unclear and is not routinely recommended. Direct measures of adiposity have limited accessibility in most clinical settings and are not sufficiently accurate.16 Although reference data for a number of pediatric dual-energy x-ray absorptiometry body composition measures have been published,17–19 it remains unclear what their clinical applicability will be both within and outside of the population with DMD. Longitudinal data on the relation of adiposity in children to future disease risk in adults also is lacking; therefore, no agreement exists about cut-points for excess adiposity that would constitute obesity.20

    Weight Management

    More intensive weight management strategies should be introduced if weight for age or BMI z score is increased by >0.5 between visits and/or if BMI is >85th percentile. A referral to an intensive interdisciplinary weight management program or to a clinician with expertise in pediatric weight management should be made if BMI is >85th percentile with associated weight-related health complications and/or if BMI is >95th percentile.

    Evidence-based guidelines for managing obesity in individuals with DMD or children with physical and developmental disabilities do not currently exist4; therefore, an adaptation of current clinical practice guidelines for managing pediatric obesity, with the exception of physical activity recommendations, should be applied. The principles of weight management are in essence a more intensive application of obesity prevention strategies (as above). Family participation in lifestyle change is essential, and interventions must include nutritional and psychosocial reassessment and support, with frequent follow-up.6 High-intensity programs (>25 hours of contact with the child and/or family over a 6-month period) were able to demonstrate improvement in weight status 12 months after beginning the intervention.6 These interventions were focused on counseling and behavioral management techniques to assist with implementation of lifestyle change. Interventions may incorporate a structured daily eating plan with the addition of some self-monitoring (through the use of logs) under the supervision of a dietitian who has training in weight management, with careful attention paid to avoid overly restrictive dieting practices. Personnel should include a registered dietitian, physical activity expert, and mental health professional (social worker, counselor, and/or psychologist) who have training in motivational interviewing, goal setting, monitoring, and positive reinforcement techniques. Interventions must be mindful of, and adapted to, the unique needs of the patient and family. More specific recommendations can be found within the “Recommendations for Treatment of Child and Adolescent Overweight and Obesity.”21 Practical clinical resources include The 5As of Pediatric Obesity Management22 and the Healthy Active Living for Families Program.22

    Monitoring Weight-Related Health Complications

    Metabolic complications of obesity include glucose dysregulation, type 2 diabetes, dyslipidemia, and hypertension. The metabolic complications of obesity are often silent and need to be screened to identify and manage them. Table 1 lists comorbidities for which regular monitoring and possible intervention are recommended. Metabolic complications also are highly heritable, and a family history should result in increased vigilance around screening.23 Glucocorticoid use increases the risk of both obesity and its metabolic complications.

    View this table:
    • View inline
    • View popup
    TABLE 1

    Recommended Monitoring for Obesity-Related Comorbidities

    Signs and symptoms of obesity-induced biomechanical complications can typically be elicited on routine history and physical examination. Effective management of biomechanical complications, especially sleep apnea and sleep-disordered breathing, may help improve success with weight management.24

    Children and youth with obesity are at increased risk of social isolation and stigmatization.25 Childhood psychiatric disorders (depression, anxiety), school difficulties, body dissatisfaction, dysregulated eating behaviors, teasing, and bullying have all been linked with pediatric obesity.26,27 Mental health disorders, as well as some of the pharmacotherapeutic agents that are used to manage them, can complicate weight management, promote weight gain, and affect prognosis and therefore should be routinely monitored through history at clinic visits.28

    Endocrine Disorders: Short Stature, Pubertal Delay, and Adrenal Insufficiency

    Growth Failure Causing Short Stature

    Short stature is common in individuals with DMD. Observational studies of glucocorticoid-naïve, ambulatory patients with DMD have described a growth pattern marked by (1) normal length at birth, (2) below-average growth velocity in early life, and (3) age-appropriate growth velocity at a below-average height percentile for the remainder of childhood.28,29 A study of ambulatory patients with DMD revealed that participants were shorter than expected for age by an average of 4.3 cm compared with reference data.30,31 The authors of a natural history study reported that the median height of participants at age 18 years was below the fifth percentile.32 The etiology of the impaired growth in individuals with DMD remains unclear because growth hormone secretion in response to provocative testing,33 circulating growth factors,28 and skeletal maturation29 were normal in patients with DMD.

    Growth impairment in DMD is exacerbated by glucocorticoid treatment.34–36 Current recommendations support the initiation of glucocorticoid therapy before functional decline; therefore, patients are exposed to the deleterious effects of glucocorticoid for the majority of their growth.37 Glucocorticoid treatment regimens vary and may affect growth differently. Growth impairment was observed irrespective of agent (prednisone, prednisolone, deflazacort) in a natural history study.38 However, an RCT revealed that a weekend-only prednisone regimen resulted in greater growth compared with daily dosing.39 The mechanisms underlying the growth inhibitory effects of glucocorticoids are complex. Glucocorticoid exposure appears to inhibit growth hormone release,40 antagonize the peripheral action of growth hormone and IGF-1,41–43 and induce chondrocyte apoptosis at the growth plate.44

    Growth should be verified every 6 months in all patients with DMD until puberty has finished and final adult height has been reached.37 Standing height is acceptable for patients who are still walking, with the results compared with a standardized growth curve. A DMD-specific growth curve derived from glucocorticoid-naïve patients is available for patients ages 2 to 12 years, although its clinical usefulness is untested.30 Growth is evaluated differently in nonambulatory patients, ideally starting before loss of ambulation to permit tracking of the growth rate during transition from walking to nonambulatory. Arm span, ulnar and tibia lengths, knee height, and segmental measurements of recumbent length have been proposed.45 However, these methods have not been specifically validated in DMD. A finding of impaired growth (downward crossing of percentile for height, height velocity <4 cm/year, or height below the third percentile) should prompt consultation with an endocrinologist. A standard clinical evaluation should be performed in all patients with DMD with growth failure to identify treatable hormonal or other causes (Fig 1, Table 2).

    FIGURE 1
    • Download figure
    • Open in new tab
    • Download powerpoint
    FIGURE 1

    Assessments and interventions for impaired growth and delayed puberty in patients with DMD. (Reproduced with permission from Birnkrant DJ, Bushby K, Bann CM, et al; DMD Care Considerations Working Group. Diagnosis and management of Duchenne muscular dystrophy, an update, part 1: diagnosis, and neuromuscular, rehabilitation, endocrine, and gastrointestinal and nutritional management. Lancet Neurol. 2018;17[3]:258.)

    View this table:
    • View inline
    • View popup
    TABLE 2

    Recommended Biochemical and Radiographic Tests for the Evaluation of Impaired Growth and Delayed Puberty in Individuals With DMD

    The treatment of DMD-related short stature is controversial. Recombinant human growth hormone (rhGH) is commonly used to treat hypothalamic and/or pituitary growth hormone deficiency and a handful of other childhood conditions.46 To date, no RCTs have been conducted to evaluate the efficacy and safety of rhGH to improve growth in individuals with DMD. The only RCT of rhGH conducted in this population was designed to investigate cardiac outcomes. The researchers found that rhGH was well tolerated and that left ventricular mass increased with treatment. However, no height outcomes were reported.40 The largest report of clinical rhGH use in individuals with DMD included 39 boys and revealed that growth velocity increased from 1.3 to 5.2 cm/year over 12 months.47 No detrimental musculoskeletal or pulmonary effects were observed, although 3 boys developed known rhGH-related adverse events (impaired fasting glucose, worsening of scoliosis, and benign intracranial hypertension). Adding to the controversy are theoretical concerns that increased growth may worsen muscle function in this context.48,49 The relevance of this evidence to humans remains uncertain, however, and clinical trials investigating the use of mazindol, a postulated inhibitor of growth hormone release, yielded inconsistent effects on growth suppression and no benefits on muscle strength.33,50–53 In light of limited data and the ongoing controversy, regular use of rhGH in the population with DMD is not recommended. The decision to use rhGH should be made on a case-by-case basis with biochemical evidence of growth hormone deficiency, and after a discussion of the benefits and known plus unknown risks. For example, it is unknown whether rhGH adversely affects muscle strength.

    Delayed Pubertal Development

    Delayed or absent pubertal development in glucocorticoid-treated patients with DMD is due to hypogonadotropic hypogonadism54 and may negatively affect physical health, psychosocial development, and self-esteem. Studies have revealed that pubertal delay in individuals with DMD is common, with a prevalence of 50% to 100% in glucocorticoid-treated boys.55,56 No published RCTs have contained assessments of the safety and efficacy of testosterone to induce puberty in individuals with DMD. However, the authors of a retrospective study of 14 boys treated with testosterone reported patient satisfaction with treatment and increased growth velocity in the first year of therapy.57 Testosterone is widely used in pediatrics to induce puberty and is specifically advised in treatment of adult men with glucocorticoid-induced hypogonadism.58

    All patients with DMD should undergo monitoring of pubertal development by physical examination every 6 months starting at age 9 years (Fig 1).37 A finding of delayed puberty (absence of testicular enlargement ≥4 cm3 by age 14 years) should prompt referral to an endocrinologist. Testosterone is recommended starting by age 14 years (or by 12 years in those on glucocorticoids) at a low dose and should be increased gradually over 2 to 3 years until adult testosterone levels are achieved. Examples of commonly used testosterone regimens are provided in Table 3. Patients and families should be counseled about expected effects, including body odor, facial hair, acne, growth spurt, growth plate closure, and increased libido. Testosterone levels should be monitored to adjust dose. Annual monitoring of hemoglobin and/or hematocrit, lipids, and serum glucose should be considered.58 An unexpected adverse effect on muscle or cardiac health may warrant discontinuation or dose reduction.

    View this table:
    • View inline
    • View popup
    TABLE 3

    Example Testosterone Replacement Regimens in DMD Patients With Confirmed Hypogonadism

    Currently, the expected benefit of restoring testosterone to normal physiologic levels is felt to outweigh potential risks of treatment. However, future studies are needed to identify the optimal timing and regimen for testosterone replacement in individuals with DMD.

    Glucocorticoid Dependence and Adrenal Insufficiency

    Chronic glucocorticoid therapy at the doses used on individuals with DMD leads to a suppressed hypothalamic-pituitary-adrenal (HPA) axis. Patients are therefore at risk for life-threatening adrenal crisis should glucocorticoid therapy be stopped suddenly and also during times of severe injury or illness.59 All patients on glucocorticoids should be taught the symptoms, signs, and appropriate management of adrenal crisis at the time of initial glucocorticoid prescription, including intramuscular hydrocortisone administrative for home use in the event the patient cannot take his usual glucocorticoid therapy because of vomiting. Patient education should also include emergency administration of intramuscular hydrocortisone and instructions for stress dosing during times of illness, trauma, or surgical intervention (Table 4). The need for education around intramuscular hydrocortisone is testament to the potentially life-threatening nature of adrenal suppression in the face of illness and surgery. Glucocorticoid therapy is never discontinued abruptly, but rather tapered slowly to allow HPA recovery. A steroid taper should adhere to the following guidance: (1) gradual dose reduction; (2) monitoring for signs and/or symptoms of adrenal insufficiency (fatigue, headache, nausea and/or vomiting, hypoglycemia, hypotension); (3) dose increase and slowing of taper in response to signs or symptoms; and (4) continuation of stress steroid coverage until the taper is complete and the HPA axis has been proven normal by an adequate cortisol response to corticorelin or corticotropin stimulation (peak cortisol >20 μg/dL [550 nmol/L]).60 HPA axis recovery can take months to years.61,62 Periodic monitoring of 8:00 am cortisol levels for a return to a normal value (eg, >6 μg/dL [165 nmol/L])63 can guide the decision around when to discontinue daily steroid therapy and to perform stimulation testing, recognizing that the precise threshold that is used to define a normal 8:00 am cortisol may vary depending on institution-specific practice standards. Irrespective of the specific threshold that is used to signal discontinuation of daily physiologic steroid therapy, an expert in adrenal suppression management should interpret the 8:00 am cortisol results in light of the child’s clinical status and in accordance with local practice standards. These decisions are typically made in collaboration with an endocrinologist. A protocol for the management of adrenal suppression has been developed previously64 and endorsed by the DMD Care Considerations Working Group.

    View this table:
    • View inline
    • View popup
    TABLE 4

    Management of Adrenal Suppression in Patients on Chronic Glucocorticoid Therapy

    Conclusions

    Because of the positive effects of glucocorticoid therapy on ambulation, mitigation of scoliosis, and cardiorespiratory function in individuals with DMD, glucocorticoid therapy has been widely adopted as the standard of care for pediatric and adult patients with DMD. At the same time, the adverse effects of glucocorticoid therapy on weight management and the hormonal milieu are often troubling for patients and in some cases are potentially life-threatening (in the case of glucocorticoid-dependence and adrenal insufficiency). To maintain a positive benefit-to-toxicity ratio, glucocorticoid therapy to treat the underlying disease should be integrated with clinical programs that effectively address the resulting side effects. In most cases, this will require a multidisciplinary effort, with input from neuromuscular specialists, endocrinologists, primary health care providers, and clinicians with expertise in obesity management. A comprehensive approach to monitoring and managing these important side effects should not be viewed as optional but rather a mandatory component of the approach to glucocorticoid therapy in this setting.

    Acknowledgments

    We thank Victor Konji for his assistance in the preparation of references for the manuscript. Dr Leanne M. Ward was supported by the Canadian Institutions for Health Research Operating Grants Program, the Canadian Child Health Clinician Scientist Program, the Children’s Hospital of Eastern Ontario Research Institute, the University of Ottawa Research Chair Program, and the Children’s Hospital of Eastern Ontario Departments of Pediatrics and Surgery.

    Footnotes

      • Accepted July 26, 2018.
    • Address correspondence to Leanne M. Ward, MD, Children’s Hospital of Eastern Ontario, University of Ottawa, 401 Smyth Rd, Ottawa, ON, K1H 8L1, Canada. E-mail: lward{at}cheo.on.ca
    • The guidelines or recommendations in this article are not American Academy of Pediatrics policy and publication herein does not imply endorsement.

    • FINANCIAL DISCLOSURE: Other than those listed under Potential Conflict of Interest; the other authors have indicated they have no financial relationships relevant to this article to disclose.

    • FUNDING: Supported in part by the Cooperative Agreement, NU38OT000167, funded by the Centers for Disease Control and Prevention. Dr Ward was supported by a Research Chair from the University of Ottawa.

    • POTENTIAL CONFLICT OF INTEREST: Dr Weber has previously served as a paid consultant for Marathon Pharmaceuticals; Drs Ward, Hadjiyannakis, McMillan, and Noritz have indicated they have no potential conflicts of interest to disclose.

    References

    1. ↵
      1. Kinnett K,
      2. Noritz G
      . The PJ Nicholoff steroid protocol for Duchenne and Becker muscular dystrophy and adrenal suppression. PLoS Curr. 2017;9:ecurrents.md.d18deef7dac96ed135e0dc8739917b6epmid:28744411
      OpenUrlPubMed
    2. ↵
      1. Must A,
      2. Curtin C,
      3. Hubbard K,
      4. Sikich L,
      5. Bedford J,
      6. Bandini L
      . Obesity prevention for children with developmental disabilities. Curr Obes Rep. 2014;3(2):156–170pmid:25530916
      OpenUrlPubMed
    3. ↵
      1. Schwartz AV
      . Diabetes mellitus: does it affect bone? Calcif Tissue Int. 2003;73(6):515–519pmid:14517715
      OpenUrlCrossRefPubMed
    4. ↵
      1. Bandini L,
      2. Danielson M,
      3. Esposito LE, et al
      . Obesity in children with developmental and/or physical disabilities. Disabil Health J. 2015;8(3):309–316pmid:26058685
      OpenUrlPubMed
    5. ↵
      1. Vincent C,
      2. Gagnon D,
      3. Routhier F, et al; ADMI Group
      . Service dogs in the province of Quebec: sociodemographic profile of users and the dogs’ impact on functional ability. Disabil Rehabil Assist Technol. 2015;10(2):132–140pmid:24369043
      OpenUrlPubMed
    6. ↵
      1. Barton M; US Preventive Services Task Force
      . Screening for obesity in children and adolescents: US Preventive Services Task Force recommendation statement. Pediatrics. 2010;125(2):361–367pmid:20083515
      OpenUrlAbstract/FREE Full Text
    7. ↵
      1. Arikian A,
      2. Boutelle K,
      3. Peterson CB,
      4. Dalton J,
      5. Day JW,
      6. Crow SJ
      . Targeting parents for the treatment of pediatric obesity in boys with Duchenne muscular dystrophy: a case series. Eat Weight Disord. 2010;15(3):e161–e165pmid:21150251
      OpenUrlPubMed
    8. ↵
      1. Casteels K,
      2. Fieuws S,
      3. van Helvoirt M, et al
      . Metformin therapy to reduce weight gain and visceral adiposity in children and adolescents with neurogenic or myogenic motor deficit. Pediatr Diabetes. 2010;11(1):61–69pmid:19496972
      OpenUrlCrossRefPubMed
    9. ↵
      1. Carter GT,
      2. Yudkowsky MP,
      3. Han JJ,
      4. McCrory MA
      . Topiramate for weight reduction in Duchenne muscular dystrophy. Muscle Nerve. 2005;31(6):788–789pmid:15779019
      OpenUrlCrossRefPubMed
    10. ↵
      1. Farooqi S,
      2. O’Rahilly S
      . Genetics of obesity in humans. Endocr Rev. 2006;27(7):710–718pmid:17122358
      OpenUrlCrossRefPubMed
    11. ↵
      1. Ford AL,
      2. Hunt LP,
      3. Cooper A,
      4. Shield JP
      . What reduction in BMI SDS is required in obese adolescents to improve body composition and cardiometabolic health? Arch Dis Child. 2010;95(4):256–261pmid:19966092
      OpenUrlAbstract/FREE Full Text
    12. ↵
      1. Barlow SE; Expert Committee
      . Expert committee recommendations regarding the prevention, assessment, and treatment of child and adolescent overweight and obesity: summary report. Pediatrics. 2007;120(suppl 4):S164–S192pmid:18055651
      OpenUrlAbstract/FREE Full Text
    13. ↵
      1. Tremblay MS,
      2. Carson V,
      3. Chaput JP, et al
      . Canadian 24-hour movement guidelines for children and youth: an integration of physical activity, sedentary behaviour, and sleep. Appl Physiol Nutr Metab. 2016;41(6 suppl 3):S311–S327pmid:27306437
      OpenUrlPubMed
    14. ↵
      1. Markert CD,
      2. Ambrosio F,
      3. Call JA,
      4. Grange RW
      . Exercise and Duchenne muscular dystrophy: toward evidence-based exercise prescription. Muscle Nerve. 2011;43(4):464–478pmid:21404285
      OpenUrlCrossRefPubMed
    15. ↵
      1. Bushby K,
      2. Finkel R,
      3. Birnkrant DJ, et al; DMD Care Considerations Working Group
      . Diagnosis and management of Duchenne muscular dystrophy, part 2: implementation of multidisciplinary care. Lancet Neurol. 2010;9(2):177–189pmid:19945914
      OpenUrlCrossRefPubMed
    16. ↵
      1. Wells JC,
      2. Fewtrell MS
      . Measuring body composition. Arch Dis Child. 2006;91(7):612–617pmid:16790722
      OpenUrlAbstract/FREE Full Text
    17. ↵
      1. Ogden CL,
      2. Li Y,
      3. Freedman DS,
      4. Borrud LG,
      5. Flegal KM
      . Smoothed percentage body fat percentiles for U.S. children and adolescents, 1999-2004. Natl Health Stat Rep. 2011;(43):1–7pmid:22164513
      OpenUrlPubMed
      1. Wells JC,
      2. Williams JE,
      3. Chomtho S, et al
      . Body-composition reference data for simple and reference techniques and a 4-component model: a new UK reference child. Am J Clin Nutr. 2012;96(6):1316–1326pmid:23076617
      OpenUrlAbstract/FREE Full Text
    18. ↵
      1. Weber DR,
      2. Moore RH,
      3. Leonard MB,
      4. Zemel BS
      . Fat and lean BMI reference curves in children and adolescents and their utility in identifying excess adiposity compared with BMI and percentage body fat. Am J Clin Nutr. 2013;98(1):49–56pmid:23697708
      OpenUrlAbstract/FREE Full Text
    19. ↵
      1. Freedman DS,
      2. Sherry B
      . The validity of BMI as an indicator of body fatness and risk among children. Pediatrics. 2009;124(suppl 1):S23–S34pmid:19720664
      OpenUrlAbstract/FREE Full Text
    20. ↵
      1. Spear BA,
      2. Barlow SE,
      3. Ervin C, et al
      . Recommendations for treatment of child and adolescent overweight and obesity. Pediatrics. 2007;120(suppl 4):S254–S288pmid:18055654
      OpenUrlAbstract/FREE Full Text
    21. ↵
      1. American Academy of Pediatrics
      . Healthy Active Living for Families (HALF) program. 2016. Available at: https://www.aap.org/en-us/advocacy-and-policy/aap-health-initiatives/HALF-Implementation-Guide/Pages/About-HALF.aspx. Accessed July 20, 2016
    22. ↵
      1. Expert Panel on Integrated Guidelines for Cardiovascular Health and Risk Reduction in Children and Adolescents
      2. National Heart, Lung, and Blood Institute
      . Expert panel on integrated guidelines for cardiovascular health and risk reduction in children and adolescents: summary report. Pediatrics. 2011;128(suppl 5):S213–S256pmid:22084329
      OpenUrlFREE Full Text
    23. ↵
      1. Harsch IA,
      2. Konturek PC,
      3. Koebnick C, et al
      . Leptin and ghrelin levels in patients with obstructive sleep apnoea: effect of CPAP treatment. Eur Respir J. 2003;22(2):251–257pmid:12952256
      OpenUrlAbstract/FREE Full Text
    24. ↵
      1. Puhl RM,
      2. Latner JD
      . Stigma, obesity, and the health of the nation’s children. Psychol Bull. 2007;133(4):557–580pmid:17592956
      OpenUrlCrossRefPubMed
    25. ↵
      1. Gonzalez A,
      2. Boyle MH,
      3. Georgiades K,
      4. Duncan L,
      5. Atkinson LR,
      6. MacMillan HL
      . Childhood and family influences on body mass index in early adulthood: findings from the Ontario Child Health Study. BMC Public Health. 2012;12:755pmid:22958463
      OpenUrlPubMed
    26. ↵
      1. Russell-Mayhew S,
      2. McVey G,
      3. Bardick A,
      4. Ireland A
      . Mental health, wellness, and childhood overweight/obesity. J Obes. 2012;2012:281801
      OpenUrlPubMed
    27. ↵
      1. Nagel BH,
      2. Mortier W,
      3. Elmlinger M,
      4. Wollmann HA,
      5. Schmitt K,
      6. Ranke MB
      . Short stature in Duchenne muscular dystrophy: a study of 34 patients. Acta Paediatr. 1999;88(1):62–65pmid:10090550
      OpenUrlCrossRefPubMed
    28. ↵
      1. Eiholzer U,
      2. Boltshauser E,
      3. Frey D,
      4. Molinari L,
      5. Zachmann M
      . Short stature: a common feature in Duchenne muscular dystrophy. Eur J Pediatr. 1988;147(6):602–605pmid:3181201
      OpenUrlCrossRefPubMed
    29. ↵
      1. West NA,
      2. Yang ML,
      3. Weitzenkamp DA, et al
      . Patterns of growth in ambulatory males with Duchenne muscular dystrophy. J Pediatr. 2013;163(6):1759–1763.e1
      OpenUrlCrossRefPubMed
    30. ↵
      1. Kuczmarski RJ,
      2. Ogden CL,
      3. Guo SS, et al
      . 2000 CDC growth charts for the United States: methods and development. Vital Health Stat 11. 2002;(246):1–190pmid:12043359
      OpenUrlPubMed
    31. ↵
      1. McDonald CM,
      2. Abresch RT,
      3. Carter GT, et al
      . Profiles of neuromuscular diseases. Duchenne muscular dystrophy. Am J Phys Med Rehabil. 1995;74(suppl 5):S70–S92pmid:7576424
      OpenUrlCrossRefPubMed
    32. ↵
      1. Zatz M,
      2. Rapaport D,
      3. Vainzof M, et al
      . Effect of mazindol on growth hormone levels in patients with Duchenne muscular dystrophy. Am J Med Genet. 1988;31(4):821–833pmid:3239574
      OpenUrlCrossRefPubMed
    33. ↵
      1. Biggar WD,
      2. Harris VA,
      3. Eliasoph L,
      4. Alman B
      . Long-term benefits of deflazacort treatment for boys with Duchenne muscular dystrophy in their second decade. Neuromuscul Disord. 2006;16(4):249–255pmid:16545568
      OpenUrlCrossRefPubMed
      1. Houde S,
      2. Filiatrault M,
      3. Fournier A, et al
      . Deflazacort use in Duchenne muscular dystrophy: an 8-year follow-up. Pediatr Neurol. 2008;38(3):200–206pmid:18279756
      OpenUrlCrossRefPubMed
    34. ↵
      1. Moxley RT III,
      2. Pandya S,
      3. Ciafaloni E,
      4. Fox DJ,
      5. Campbell K
      . Change in natural history of Duchenne muscular dystrophy with long-term corticosteroid treatment: implications for management. J Child Neurol. 2010;25(9):1116–1129pmid:20581335
      OpenUrlCrossRefPubMed
    35. ↵
      1. Birnkrant DJ,
      2. Bushby K,
      3. Bann CM, et al; DMD Care Considerations Working Group
      . Diagnosis and management of Duchenne muscular dystrophy, part 2: respiratory, cardiac, bone health, and orthopaedic management. Lancet Neurol. 2018;17(4):347–361pmid:29395990
      OpenUrlPubMed
    36. ↵
      1. Bello L,
      2. Gordish-Dressman H,
      3. Morgenroth LP, et al; CINRG Investigators
      . Prednisone/prednisolone and deflazacort regimens in the CINRG Duchenne Natural History Study. Neurology. 2015;85(12):1048–1055pmid:26311750
      OpenUrlAbstract/FREE Full Text
    37. ↵
      1. Escolar DM,
      2. Hache LP,
      3. Clemens PR, et al
      . Randomized, blinded trial of weekend vs daily prednisone in Duchenne muscular dystrophy. Neurology. 2011;77(5):444–452pmid:21753160
      OpenUrlAbstract/FREE Full Text
    38. ↵
      1. Cittadini A,
      2. Ines Comi L,
      3. Longobardi S, et al
      . A preliminary randomized study of growth hormone administration in Becker and Duchenne muscular dystrophies. Eur Heart J. 2003;24(7):664–672pmid:12657225
      OpenUrlCrossRefPubMed
    39. ↵
      1. Wehrenberg WB,
      2. Janowski BA,
      3. Piering AW,
      4. Culler F,
      5. Jones KL
      . Glucocorticoids: potent inhibitors and stimulators of growth hormone secretion. Endocrinology. 1990;126(6):3200–3203pmid:1972061
      OpenUrlCrossRefPubMed
      1. Allen DB,
      2. Julius JR,
      3. Breen TJ,
      4. Attie KM
      . Treatment of glucocorticoid-induced growth suppression with growth hormone. National Cooperative Growth Study. J Clin Endocrinol Metab. 1998;83(8):2824–2829pmid:9709954
      OpenUrlCrossRefPubMed
    40. ↵
      1. Jux C,
      2. Leiber K,
      3. Hügel U, et al
      . Dexamethasone impairs growth hormone (GH)-stimulated growth by suppression of local insulin-like growth factor (IGF)-I production and expression of GH- and IGF-I-receptor in cultured rat chondrocytes. Endocrinology. 1998;139(7):3296–3305pmid:9645706
      OpenUrlCrossRefPubMed
    41. ↵
      1. Chrysis D,
      2. Ritzen EM,
      3. Sävendahl L
      . Growth retardation induced by dexamethasone is associated with increased apoptosis of the growth plate chondrocytes. J Endocrinol. 2003;176(3):331–337pmid:12630918
      OpenUrlAbstract
    42. ↵
      1. Lohman TG,
      2. Roche AF,
      3. Martorell R
      , eds. Anthropometric Standardization Reference Manual. Champaign, IL: Human Kinetics Books; 1988
    43. ↵
      1. Hardin DS,
      2. Kemp SF,
      3. Allen DB
      . Twenty years of recombinant human growth hormone in children: relevance to pediatric care providers. Clin Pediatr (Phila). 2007;46(4):279–286pmid:17475983
      OpenUrlCrossRefPubMed
    44. ↵
      1. Rutter MM,
      2. Collins J,
      3. Rose SR, et al
      . Growth hormone treatment in boys with Duchenne muscular dystrophy and glucocorticoid-induced growth failure. Neuromuscul Disord. 2012;22(12):1046–1056pmid:22967789
      OpenUrlCrossRefPubMed
    45. ↵
      1. Zatz M,
      2. Rapaport D,
      3. Vainzof M, et al
      . Relation between height and clinical course in Duchenne muscular dystrophy. Am J Med Genet. 1988;29(2):405–410pmid:3354613
      OpenUrlCrossRefPubMed
    46. ↵
      1. Bodor M,
      2. McDonald CM
      . Why short stature is beneficial in Duchenne muscular dystrophy. Muscle Nerve. 2013;48(3):336–342pmid:23893308
      OpenUrlCrossRefPubMed
    47. ↵
      1. Coakley JH,
      2. Moorcraft J,
      3. Hipkin LJ,
      4. Smith CS,
      5. Griffiths RD,
      6. Edwards RH
      . The effect of mazindol on growth hormone secretion in boys with Duchenne muscular dystrophy. J Neurol Neurosurg Psychiatry. 1988;51(12):1551–1557pmid:3221222
      OpenUrlAbstract/FREE Full Text
      1. Zatz M,
      2. Rapaport D,
      3. Pavanello RC,
      4. Rocha JM,
      5. Vainzof M,
      6. Nicolau W
      . Nocturnal rhythm of growth hormone in Duchenne patients: effect of different doses of mazindol and/or cyproheptadine. Am J Med Genet. 1989;33(4):457–467pmid:2596504
      OpenUrlPubMed
      1. Collipp PJ,
      2. Kelemen J,
      3. Chen SY,
      4. Castro-Magana M,
      5. Angulo M,
      6. Derenoncourt A
      . Growth hormone inhibition causes increased selenium levels in Duchenne muscular dystrophy: a possible new approach to therapy. J Med Genet. 1984;21(4):254–256pmid:6492089
      OpenUrlAbstract/FREE Full Text
    48. ↵
      1. Griggs RC,
      2. Moxley RT III,
      3. Mendell JR, et al
      . Randomized, double-blind trial of mazindol in Duchenne dystrophy. Muscle Nerve. 1990;13(12):1169–1173pmid:2266990
      OpenUrlCrossRefPubMed
    49. ↵
      1. Rosen H,
      2. Jameel ML,
      3. Barkan AL
      . Dexamethasone suppresses gonadotropin-releasing hormone (GnRH) secretion and has direct pituitary effects in male rats: differential regulation of GnRH receptor and gonadotropin responses to GnRH. Endocrinology. 1988;122(6):2873–2880pmid:2836177
      OpenUrlCrossRefPubMed
    50. ↵
      1. Merlini L,
      2. Gennari M,
      3. Malaspina E, et al
      . Early corticosteroid treatment in 4 Duchenne muscular dystrophy patients: 14-year follow-up. Muscle Nerve. 2012;45(6):796–802pmid:22581531
      OpenUrlCrossRefPubMed
    51. ↵
      1. Dooley JM,
      2. Bobbitt SA,
      3. Cummings EA
      . The impact of deflazacort on puberty in Duchenne muscular dystrophy. Pediatr Neurol. 2013;49(4):292–293pmid:23921283
      OpenUrlPubMed
    52. ↵
      1. Wood CL,
      2. Cheetham TD,
      3. Guglieri M, et al
      . Testosterone treatment of pubertal delay in Duchenne muscular dystrophy. Neuropediatrics. 2015;46(6):371–376pmid:26408798
      OpenUrlPubMed
    53. ↵
      1. Bhasin S,
      2. Cunningham GR,
      3. Hayes FJ, et al; Task Force, Endocrine Society
      . Testosterone therapy in men with androgen deficiency syndromes: an Endocrine Society clinical practice guideline. J Clin Endocrinol Metab. 2010;95(6):2536–2559pmid:20525905
      OpenUrlCrossRefPubMed
    54. ↵
      1. Shulman DI,
      2. Palmert MR,
      3. Kemp SF; Lawson Wilkins Drug and Therapeutics Committee
      . Adrenal insufficiency: still a cause of morbidity and death in childhood. Pediatrics. 2007;119(2). Available at: www.pediatrics.org/cgi/content/full/119/2/e484pmid:17242136
      OpenUrlAbstract/FREE Full Text
    55. ↵
      1. Puar TH,
      2. Stikkelbroeck NM,
      3. Smans LC,
      4. Zelissen PM,
      5. Hermus AR
      . Adrenal crisis: still a deadly event in the 21st century. Am J Med. 2016;129(3):339.e1–339.e9
      OpenUrl
    56. ↵
      1. Huber BM,
      2. Bolt IB,
      3. Sauvain MJ,
      4. Flück CE
      . Adrenal insufficiency after glucocorticoid withdrawal in children with rheumatic diseases. Acta Paediatr. 2010;99(12):1889–1893pmid:20649769
      OpenUrlCrossRefPubMed
    57. ↵
      1. Jamilloux Y,
      2. Liozon E,
      3. Pugnet G, et al
      . Recovery of adrenal function after long-term glucocorticoid therapy for giant cell arteritis: a cohort study. PLoS One. 2013;8(7):e68713pmid:23894335
      OpenUrlCrossRefPubMed
    58. ↵
      1. Charmandari E,
      2. Nicolaides NC,
      3. Chrousos GP
      . Adrenal insufficiency. Lancet. 2014;383(9935):2152–2167pmid:24503135
      OpenUrlCrossRefPubMed
    59. ↵
      1. Birnkrant DJ,
      2. Bushby K,
      3. Bann CM, et al; DMD Care Considerations Working Group
      . Diagnosis and management of Duchenne muscular dystrophy, part 1: diagnosis, and neuromuscular, rehabilitation, endocrine, and gastrointestinal and nutritional management. Lancet Neurol. 2018;17(3):251–267pmid:29395989
      OpenUrlPubMed
    • Copyright © 2018 by the American Academy of Pediatrics
    PreviousNext
    Back to top

    Advertising Disclaimer »

    In this issue

    Pediatrics
    Vol. 142, Issue Supplement 2
    1 Oct 2018
    • Table of Contents
    • Index by author
    View this article with LENS
    PreviousNext
    Email Article

    Thank you for your interest in spreading the word on American Academy of Pediatrics.

    NOTE: We only request your email address so that the person you are recommending the page to knows that you wanted them to see it, and that it is not junk mail. We do not capture any email address.

    Enter multiple addresses on separate lines or separate them with commas.
    Obesity and Endocrine Management of the Patient With Duchenne Muscular Dystrophy
    (Your Name) has sent you a message from American Academy of Pediatrics
    (Your Name) thought you would like to see the American Academy of Pediatrics web site.
    CAPTCHA
    This question is for testing whether or not you are a human visitor and to prevent automated spam submissions.
    Request Permissions
    Article Alerts
    Log in
    You will be redirected to aap.org to login or to create your account.
    Or Sign In to Email Alerts with your Email Address
    Citation Tools
    Obesity and Endocrine Management of the Patient With Duchenne Muscular Dystrophy
    David R. Weber, Stasia Hadjiyannakis, Hugh J. McMillan, Garey Noritz, Leanne M. Ward
    Pediatrics Oct 2018, 142 (Supplement 2) S43-S52; DOI: 10.1542/peds.2018-0333F

    Citation Manager Formats

    • BibTeX
    • Bookends
    • EasyBib
    • EndNote (tagged)
    • EndNote 8 (xml)
    • Medlars
    • Mendeley
    • Papers
    • RefWorks Tagged
    • Ref Manager
    • RIS
    • Zotero
    Share
    Obesity and Endocrine Management of the Patient With Duchenne Muscular Dystrophy
    David R. Weber, Stasia Hadjiyannakis, Hugh J. McMillan, Garey Noritz, Leanne M. Ward
    Pediatrics Oct 2018, 142 (Supplement 2) S43-S52; DOI: 10.1542/peds.2018-0333F
    del.icio.us logo Digg logo Reddit logo Twitter logo CiteULike logo Facebook logo Google logo Mendeley logo
    Print
    Download PDF
    Insight Alerts
    • Table of Contents

    Jump to section

    • Article
      • Abstract
      • Overweight, Obesity, and Related Comorbidities
      • Endocrine Disorders: Short Stature, Pubertal Delay, and Adrenal Insufficiency
      • Conclusions
      • Acknowledgments
      • Footnotes
      • References
    • Figures & Data
    • Info & Metrics
    • Comments

    Related Articles

    • No related articles found.
    • PubMed
    • Google Scholar

    Cited By...

    • No citing articles found.
    • Google Scholar

    More in this TOC Section

    • Part 4: Pediatric Basic and Advanced Life Support 2020 American Heart Association Guidelines for Cardiopulmonary Resuscitation and Emergency Cardiovascular Care
    • Pediatric Life Support 2020 International Consensus on Cardiopulmonary Resuscitation and Emergency Cardiovascular Care Science With Treatment Recommendations
    • Part 5: Neonatal Resuscitation 2020 American Heart Association Guidelines for Cardiopulmonary Resuscitation and Emergency Cardiovascular Care
    Show more Supplement Article

    Similar Articles

    • Journal Info
    • Editorial Board
    • Editorial Policies
    • Overview
    • Licensing Information
    • Authors/Reviewers
    • Author Guidelines
    • Submit My Manuscript
    • Open Access
    • Reviewer Guidelines
    • Librarians
    • Institutional Subscriptions
    • Usage Stats
    • Support
    • Contact Us
    • Subscribe
    • Resources
    • Media Kit
    • About
    • International Access
    • Terms of Use
    • Privacy Statement
    • FAQ
    • AAP.org
    • shopAAP
    • Follow American Academy of Pediatrics on Instagram
    • Visit American Academy of Pediatrics on Facebook
    • Follow American Academy of Pediatrics on Twitter
    • Follow American Academy of Pediatrics on Youtube
    • RSS
    American Academy of Pediatrics

    © 2021 American Academy of Pediatrics