Testosterone Treatment of Pubertal Delay in Duchenne Muscular Dystrophy

Wood, Claire; Cheetham, Timothy; Guglieri, Michela; Bushby, K.; Owen, Catherine J.; Johnstone, Helen; Straub, Volker

doi:10.1055/s-0035-1563696

Cited by 26 publications

(6 citation statements)

References 23 publications

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“…The increase in BMD LS could not be solely explained by very small increases in height, far less than the 25-cm height increase over puberty that is seen in healthy boys and less than that previously reported in a small cohort of boys with DMD treated briefly with testosterone for pubertal delay [21]. The majority accrual of bone mass of our cohort is thus due to testosterone effect.…”

Section: Discussion/conclusioncontrasting

confidence: 72%

Effect of Testosterone Treatment for Delayed Puberty in Duchenne Muscular Dystrophy

et al. 2020

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Objective: To evaluate the impact of pubertal induction with testosterone on bone health, body composition, and motor function in boys with Duchenne muscular dystrophy (DMD) receiving long-term glucocorticoid. Study Design: A retrospective, observational, pre-post study investigating the impact of testosterone therapy on bone mass accrual, vertebral fracture incidence, body composition, motor function, and quality of life in boys with DMD. All those boys aged ≥14 years, on chronic steroid therapy, who had delayed puberty, and were receiving oral testosterone or oral and then transitioned to intramuscular testosterone, to complete virilization, were included. Prior/concomitant zoledronic acid use was included. The primary outcome was lumbar spine areal bone mineral density (BMD LS). Results: Puberty was induced, using oral testosterone undecanoate in 16 individuals, 10 of whom had transited to intramuscular testosterone at time of assessment. Median age at testosterone onset was 14.5 years (range 14-17.7). Median duration of testosterone therapy was 2.5 years (range 1.0-4.5). There was statistically significant increase in median BMD LS (0.523-0.700, p < 0.001) and median annualized percentage change of BMD LS (-1.34 to +10.08%, p < 0.001), with median Tanner stage 4 at evaluation (range 2-4). Ten of 14 assessed had no progression in vertebral fractures. Fat mass index (FMI) standard deviation score (SDS), lean body mass index (LBMI) SDS, and percentage change of FMI and LBMI were statistically unchanged. Cardiac function remained stable. Motor function in non-ambulatory individuals with Egen Klassifikation scores improved in 7 of 8. Conclusion: Testosterone for delayed puberty acted as an adjunct to bisphosphonates to increase bone density and stabilize vertebral fracture in most boys with DMD.

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Section: Discussion/conclusioncontrasting

confidence: 72%

Effect of Testosterone Treatment for Delayed Puberty in Duchenne Muscular Dystrophy

et al. 2020

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“…A recent retrospective review found that testosterone was generally well tolerated and perceived to be beneficial by individuals with DMD and their families. 103 …”

Section: Endocrine Managementmentioning

confidence: 99%

Diagnosis and management of Duchenne muscular dystrophy, part 1: diagnosis, and neuromuscular, rehabilitation, endocrine, and gastrointestinal and nutritional management

Birnkrant

Bushby

Bann

et al. 2018

The Lancet Neurology

878

1,080

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Since the publication of the Duchenne muscular dystrophy (DMD) care considerations in 2010, multidisciplinary care of this severe, progressive neuromuscular disease has evolved. In conjunction with improved patient survival, a shift to more anticipatory diagnostic and therapeutic strategies has occurred, with a renewed focus on patient quality of life. In 2014, a steering committee of experts from a wide range of disciplines was established to update the 2010 DMD care considerations, with the goal of improving patient care. The new care considerations aim to address the needs of patients with prolonged survival, to provide guidance on advances in assessments and interventions, and to consider the implications of emerging genetic and molecular therapies for DMD. The committee identified 11 topics to be included in the update, eight of which were addressed in the original care considerations. The three new topics are primary care and emergency management, endocrine management, and transitions of care across the lifespan. In part 1 of this three-part update, we present care considerations for diagnosis of DMD and neuromuscular, rehabilitation, endocrine (growth, puberty, and adrenal insufficiency), and gastrointestinal (including nutrition and dysphagia) management.

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“…It is possible that some boys with DMD have a contiguous gene deletion which may be associated with hypogonadotrophic hypogonadism. Thus, in contrast, in boys with DMD, testosterone therapy is commenced and gradually increased until adult replacement dose is attained over a period of 2-3 years, evaluating for testicular growth during treatment indicating endogenous gonadotrophin activity [72]. …”

Section: A Proposed Pathway Of Clinical Monitoring Of Bone Health Andmentioning

confidence: 99%

Skeletal Fragility in Children with Chronic Disease

2016

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Skeletal fragility associated with underlying childhood chronic disease is a systemic disorder of poor bone growth and reduction in bone turnover which can lead to abnormal bone mass, geometry and microarchitecture. Due to the growth potential unique to children, remarkable bone recovery following a transient threat to the bone can occur if there is concurrent growth. Addressing bone health in these children should focus on improvement in growth, puberty and removing the primary insult. In conditions where there is a little scope for bone recovery and limited residual growth, bone-targeted therapy may need to be considered, even though there is currently limited evidence. The importance of early detection of signs of bone fragility, by active screening for vertebral fracture using newer imaging techniques such as dual-energy X-ray absorptiometry lateral vertebral morphometry, may now be possible. There is currently, a paucity of evidence to support prophylactic use of anti-resorptive therapy. Where poor growth and low bone turnover are seen, the use of growth-promoting therapies and anabolic bone-protective agents may be more physiological and should be evaluated in well-designed trials. Collaborative studies on long-term fracture outcome and well-designed trials of bone-protective therapies are needed and to be encouraged.

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Testosterone Treatment of Pubertal Delay in Duchenne Muscular Dystrophy

Cited by 26 publications

References 23 publications

Effect of Testosterone Treatment for Delayed Puberty in Duchenne Muscular Dystrophy

Effect of Testosterone Treatment for Delayed Puberty in Duchenne Muscular Dystrophy

Diagnosis and management of Duchenne muscular dystrophy, part 1: diagnosis, and neuromuscular, rehabilitation, endocrine, and gastrointestinal and nutritional management

Skeletal Fragility in Children with Chronic Disease

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