Paediatric thyroid disease.

The spectrum of thyroid disorders presenting to paediatricians is different to that seen by adult physicians. Referrals reflect cases detected by the neonatal screening programme for congenital hypothyroidism and many of the inherited defects of thyroid hormone generation or action will be manifest in early life. Autoimmune thyroid disease can be particularly challenging to manage in the young and the potential impact of thyroid status on neurodevelopment and schooling are key considerations throughout childhood and adolescence.

Sexual health and fertility in Duchenne muscular dystrophy-An exploratory study.

INTRODUCTION/AIMS: Recent clinical guidelines recommend that adolescents with Duchenne muscular dystrophy (DMD) who are on daily glucocorticoid treatment should be offered pubertal induction in order to ensure adult levels of sex hormones as they reach adulthood. However, it remains unclear how gonadal status, including androgen concentrations, impacts physical function and future fertility. The aim of this study was to give a voice to adults with DMD, exploring their perspectives around sexual health, hormone treatment, and fertility. METHODS: Qualitative data was collected from six adults with DMD through two online focus groups. Participants were recruited through Pathfinders Neuromuscular Alliance and Duchenne UK and invited to take part if they had DMD and were 18 years of age or older. Conversations were transcribed verbatim and an interpretivist paradigm was used with thematic analysis. RESULTS: The main themes identified were (1) the need for communication and information about sexual health, (2) dealing with the potential fear of rejection, (3) physical barriers to relationships including sex, (4) testosterone supplementation in DMD, and (5) parenthood and fertility. DISCUSSION: We recommend that clinicians work with young people with DMD individually, to explore the benefits of testosterone treatment for them and their personal sexual health needs. If they are offered treatment, this should always be accompanied by the opportunity for psychological support. This work highlights the need for further research to establish the role of testosterone supplementation in adults with DMD and its effects on fertility and the value of specific emotional and practical support for sexual health.

An evaluation of first responders' intention to refer to post-overdose services following SHIELD training.

BACKGROUND: First responders [law enforcement officers (LEO) and Fire/Emergency Medical Services (EMS)] can play a vital prevention role, connecting overdose survivors to treatment and recovery services. This study was conducted to examine the effect of occupational safety and harm reduction training on first responders' intention to refer overdose survivors to treatment, syringe service, naloxone distribution, social support, and care-coordination services, and whether those intentions differed by first responder profession. METHODS: First responders in Missouri were trained using the Safety and Health Integration in the Enforcement of Laws on Drugs (SHIELD) model. Trainees' intent to refer (ITR) overdose survivors to prevention and supportive services was assessed pre- and post-training (1-5 scale). A mixed model analysis was conducted to assess change in mean ITR scores between pre- and post-training, and between profession type, while adjusting for random effects between individual trainees and baseline characteristics. RESULTS: Between December 2020 and January 2023, 742 first responders completed pre- and post-training surveys. SHIELD training was associated with higher first responders' intentions to refer, with ITR to naloxone distribution (1.83-3.88) and syringe exchange (1.73-3.69) demonstrating the greatest changes, and drug treatment (2.94-3.95) having the least change. There was a significant increase in ITR score from pre- to post-test (ß = 2.15; 95% CI 1.99, 2.30), and LEO-relative to Fire/EMS-had a higher score at pre-test (0.509; 95% CI 0.367, 0.651) but a lower score at post-test (0.148; 95% CI - 0.004, 0.300). CONCLUSION: Training bundling occupational safety with harm reduction content is immediately effective at increasing first responders' intention to connect overdose survivors to community substance use services. When provided with the rationale and instruction to execute referrals, first responders are amenable, and their positive response highlights the opportunity for growth in increasing referral partnerships and collaborations. Further research is necessary to assess the extent to which ITR translates to referral behavior in the field.

Outcomes of State Targeted/Opioid Response Grants and the Medication First Approach: Evidence of Racial Inequities in Improved Treatment Access and Retention.

BACKGROUND: Since 2017, Missouri has increased access to medication for opioid use disorder (OUD) within the State's publicly-funded substance use specialty treatment system through a "Medication First" approach. Results from a statewide assessment of the first year of State Targeted Response implementation showed increases and improvements in overall treatment admissions, medication utilization, and treatment retention. The current study, which focuses on the St. Louis region, the epicenter of Missouri's overdose crisis, examines whether improvements were experienced equally among Black and White clients. METHODS: This study is a retrospective analysis using state-level billing records for individuals with OUD receiving services through publicly-funded substance use treatment programs between July 1, 2016, and June 30, 2019, with claimed services updated through November 1, 2020. Comparisons across time periods, treatment groups, and Black and White clients were assessed using chi-square tests of independence and multivariate negative binomial regressions. RESULTS: White individuals in St. Louis experienced larger increases in treatment admissions and utilization of medications for OUD than Black individuals, and Black clients were retained in treatment for shorter lengths of time than White clients. CONCLUSION: In Missouri, rates of drug overdose deaths are more than three times higher for Black people than White people. Racial inequities in OUD treatment utilization and retention must be intentionally targeted and corrected as one component of reducing this sizable disparity in fatalities.

Early diet in preterm infants and later cognition: 10-year follow-up of a randomized controlled trial.

BACKGROUND: Achieving adequate nutrition in preterm infants is challenging. The post-discharge period may be critical for influencing growth and cognitive outcomes. We studied the effects of post-discharge nutrition on childhood cognition. METHODS: Preterm-born children were randomized at ~36 weeks corrected age (CGA) to either preterm formula (PTF) or term formula (TF) until 6 months, or PTF until 40 weeks CGA, then TF until 6 months (crossover group). Childhood cognition was assessed using the short form Wechsler Intelligence Scale for Children III, allowing computation of full-scale intelligence quotient (FSIQ) and four-factor index scores; verbal comprehension, freedom from distractibility (FDI), perceptual organization (POI), and processing speed (PSI). RESULTS: Ninety-two children were recruited (mean 10.1 years). FSIQ did not differ by group. PTF-fed children had 10-point higher PSI (p = 0.03) compared to crossover. Faster weight gain from term to 12 weeks CGA was associated with 5-point higher FSIQ (p = 0.02) and four-point higher POI (p = 0.04). Infant head growth was positively associated with FSIQ (+3.8 points, p = 0.04) and FDI (+6 points, p = 0.003). CONCLUSIONS: While there is no long-term impact of post-discharge macronutrient enrichment on childhood cognition, greater weight and head growth in specific epochs is associated with better outcomes. Further studies are needed to determine optimal early diet in preterm infants. IMPACT: Achieving adequate nutrient intakes in preterm infants before and after hospital discharge is challenging. Nutrient intakes prior to discharge affect later cognitive and metabolic outcomes. Follow-up of a randomized controlled trial shows no cognitive benefit in later childhood from a more nutrient-dense formula compared to standard formula after hospital discharge. Growth in the first year of life is strongly correlated with childhood cognition and emphasizes the importance of nutrition in early life.

Measurement of salivary testosterone in adolescents and young men with Duchenne muscular dystrophy.

BACKGROUND: Many young adults with Duchenne Muscular Dystrophy (DMD) receive long-term glucocorticoids (GC). GC can cause hypogonadotrophic hypogonadism and adolescents may therefore be candidates for pubertal induction. It is unclear whether men with DMD on or off GC have age-appropriate endogenous testosterone production. METHODS: We undertook a quality improvement project to assess the feasibility of measuring salivary testosterone (SalT) levels in men with DMD at home. A Sal-T sampling kit was sent by post to all patients with DMD, aged 17 and older, registered at the John Walton Muscular Centre in Newcastle (n = 75). Submitted Sal-T samples were collected and submitted for analysis. RESULTS: Twenty-eight out of seventy-five patients returned samples (age range: 17-34 years). 6/28 samples were unsuitable for analysis. Overall Sal-T levels (n = 22) were significantly lower than in the healthy population (178 ± 107 v 287 ± 109 pmol/l, p = 0.0001). Sal-T was lower in those on GC compared to those off GC (144 ± 81 versus 218 ± 125 pmol/l, p = 0.05). Three patients were unable to collect a sample due to ventilator dependence. CONCLUSION: Sal T can provide information about androgen status in DMD patients at home, overcoming barriers such as mobility difficulties and challenging venepuncture. However we only obtained samples in a minority of patients suggesting that Sal-T measurement may not be appropriate or acceptable to everyone. There needs to be a more detailed exploration of the barriers to sample submission.

Police discretion in encounters with people who use drugs: operationalizing the theory of planned behavior.

BACKGROUND: Policing shapes the health risks of people who use drugs (PWUD), but little is understood about interventions that can align officer practices with PWUD health. This study deploys the Theory of Planned Behavior (TPB) to understand what influences police intentions to make discretionary referrals to treatment and harm reduction resources rather than arrest on less serious charges. METHODS: On-line surveys integrating TPB constructs and adapting an instrument measuring police intentions to make mental health treatment referrals were completed by police employees in Indiana, Massachusetts, and Missouri. They also included items about stigma towards PWUD and attitudes and beliefs about opioid addiction, treatment, and recovery. FINDINGS: Across the sites, 259 respondents perceived control over their decision to arrest for misdemeanors (69%) and confiscate items such as syringes (56%). Beliefs about others' approval of referrals to treatment, its ability to reduce future arrests, and to increase trust in police were associated with stated practices of nonarrest for drug and possession and making referrals (p ≤ .001), and nonarrest for syringe possession (p ≤ .05). Stigma a towards PWUD was negatively associated with stated practices of nonarrest (p ≤ .05). Respondents identified supervisors as having the most influence over use of discretion, seriousness of the offense as the most influential value, and attitude of the suspect as the most important situational factor. The 17 Likert scale items analyzed had a Cronbach's alpha of 0.81. CONCLUSION: The TPB offers untapped potential to better understand and modify police practices. In designing interventions to improve the health outcomes of police encounters with PWUD, further research should validate instruments that measure the relationship between these variables and discretionary intentions, and that measure role-relevant police stigma towards PWUD.

Balancing need and risk, supply and demand: Developing a tool to prioritize naloxone distribution.

Background: Opioid overdose deaths continue to rise nationally. The demand for naloxone, the opioid overdose antidote, is outpacing the supply. With increasing naloxone requests, tools to prioritize distribution are critical to ensure available supplies will reach those at highest risk of overdose. Methods: We developed a standardized "Naloxone Request Form" (NRF) and corresponding weighted prioritization algorithm to serve as decisional aid to better enable grant staff to prioritize naloxone distribution in a data-driven manner. The algorithm computed raw priority scores for each agency, which were then separated into the predetermined quintiles. Historical naloxone distribution decisions were compared with agencies' prioritization quintile. Results: Results demonstrated that the NRF and corresponding algorithm was successful at prioritizing agencies based on potential impact. Although, overall, naloxone was distributed more heavily to the agencies deemed highest priority, our algorithm identified significant shortcomings of the "first come, first served" method of distribution we had initially deployed. Conclusions: This work has laid the foundation to use this tool prospectively to allow for data-driven decision-making for naloxone distribution. Our tool is flexible and can be customized to best fit the needs of a variety of programs and locations to ensure the distribution of limited supplies of naloxone have the greatest impact.

Fifteen-minute consultation: An approach to the child receiving glucocorticoids.

Glucocorticoids (GC) are used in paediatric practice for a broad range of conditions and all paediatricians will prescribe GC, in some form, during their career. A wide variety of GC formulations, doses and administration routes are used for periods of time ranging from days to years. Exposure to exogenous GC can result in hypothalamic-pituitary-adrenal axis suppression-otherwise known as adrenal suppression (AS). Patients with AS may be well most of the time but if GC therapy is reduced or stopped or if additional endogenous GC cannot be generated during illness, then an absolute or relative lack of GC can result in severe illness or death. Here, we highlight the relevance of AS to all paediatricians by providing an overview of the background and discussing the presentation and approaches to the management of this clinical entity.

Some law enforcement officers' negative attitudes toward overdose victims are exacerbated following overdose education training.

BACKGROUND: The devastating impact of the current opioid overdose crisis has led to new involvement of law enforcement officers. Training programs have focused on overdose recognition and response without targeting core attitudinal change by covering addiction or harm reduction principles. OBJECTIVES: This study examined the impact of a comprehensive overdose education and naloxone distribution (OEND) training on officers' attitudes toward overdose victims, knowledge of and competence to respond to an opioid overdose, and concerns about using naloxone. The training included the common information about overdose recognition and response, with added components covering broader content about addiction and harm reduction principles and philosophies. METHODS: A total of 787 (83% male) officers were administered surveys before and after attending a 2.5-3 hour comprehensive OEND training. Survey items measured overdose-related knowledge and attitudes, including attitudes about people who use drugs and who overdose. RESULTS: Following the training, participants' overdose-related knowledge and perceived competence to use naloxone improved. However, there were more nuanced changes in attitudes toward overdose victims: though 55.3% of officers reported more positive post-training attitudes, 31% reported more negative attitudes, and 13.7% reported no attitudinal change. Younger officers were most likely to report worsened attitudes. Improvements in attitudes toward overdose victims were associated with reductions in both naloxone-related concerns and risk compensation beliefs. CONCLUSIONS: Despite a comprehensive OEND training that addressed addiction and harm reduction and directly targeted hypothesized drivers of negative attitudes (e.g., risk compensation beliefs), some officers' attitudes worsened after the training. Randomized experiments of different training approaches would elucidate the mediators and moderators underlying these unexpected responses.

Physical Inactivity in Pulmonary Sarcoidosis.

PURPOSE: Reduced physical activity in many chronic diseases is consistently associated with increased morbidity. Little is known about physical activity in sarcoidosis. The aim of this study was to objectively assess physical activity in patients with pulmonary sarcoidosis and investigate its relationship with lung function, exercise capacity, symptom burden, and health status. METHODS: Physical activity was assessed over one week in 15 patients with pulmonary sarcoidosis and 14 age-matched healthy controls with a tri-axial accelerometer (ActivPal™) and the International Physical Activity Questionnaire (IPAQ). All participants underwent pulmonary function tests, 6-min walk test (6MWT) and completed the Fatigue Assessment Scale (FAS), Medical Research Council (MRC) Dyspnoea Scale and the King's Sarcoidosis Questionnaire (KSQ). RESULTS: Patients with sarcoidosis had significantly lower daily step counts than healthy controls; mean (SD) 5624 (1875) versus 10,429 (2942) steps (p < 0.01) and a trend towards fewer sit-to-stand transitions each day (p = 0.095). Only two patients (13%) self-reported undertaking vigorous physical activity (IPAQ) compared to half of healthy individuals (p < 0.01). Daily step count was significantly associated with 6MWT distance in sarcoidosis (r = 0.634, p = 0.01), but not with forced vital capacity (r = 0.290), fatigue (r = 0.041), dyspnoea (r = -0.466) or KSQ health status (r = 0.099-0.484). Time spent upright was associated with fatigue (r = -0.630, p = 0.012) and health status (KSQ Lung scores r = 0.524, p = 0.045), and there was a significant correlation between the number of sit-to-stand transitions and MRC dyspnoea score (r = -0.527, p = 0.044). CONCLUSION: Physical activity is significantly reduced in sarcoidosis and is associated with reduced functional exercise capacity (6MWD). Fatigue, exertional symptoms and health status were more closely associated with time spent upright and the number of bouts of physical activity, as compared to step counts. Further studies are warranted to identify the factors that determine different physical activity profiles in sarcoidosis.

The case for a medication first approach to the treatment of opioid use disorder.

Background: The opioid addiction and overdose crisis continues to ravage communities across the U.S. Maintenance pharmacotherapy using buprenorphine or methadone is the most effective intervention for Opioid Use Disorder (OUD), yet few have immediate and sustained access to these medications. Objectives: To address lack of medication access for people with OUD, the Missouri Department of Mental Health began implementing a Medication First (Med First) treatment approach in its publicly-funded system of comprehensive substance use disorder treatment programs. Methods: This Perspective describes the four principles of Med First, which are based on evidence-based guidelines. It draws conceptual comparisons between the Housing First approach to chronic homelessness and the Med First approach to pharmacotherapy for OUD, and compares state certification standards for substance use disorder (SUD) treatment (the traditional approach) to Med First guidelines for OUD treatment. Finally, the Perspective details how Med First principles have been practically implemented. Results: Med First principles emphasize timely access to maintenance pharmacotherapy without requiring psychosocial services or discontinuation for any reason other than harm to the client. Early results regarding medication utilization and treatment retention are promising. Feedback from providers has been largely favorable, though clinical- and system-level obstacles to effective OUD treatment remain. Conclusion: Like the Housing First model, Medication First is designed to decrease human suffering and activate the strengths and capacities of people in need. It draws on decades of research and facilitates partnerships between psychosocial and medical treatment providers to offer effective and life-saving care to persons with OUD.

Marital Processes Linking Gender Role Attitudes and Marital Satisfaction Among Mexican-Origin Couples: Application of an Actor-Partner Interdependence Mediation Model.

Informed by dyadic approaches and culturally informed, ecological perspectives of marriage, we applied an actor-partner interdependence mediation model (APIMeM) in a sample of 120 Mexican-origin couples to examine (a) the associations linking Mexican immigrant husbands' and wives' gender role attitudes to marital satisfaction directly and indirectly through marital processes (i.e., warmth and negativity) and (b) whether the associations between spouses' gender role attitudes and marital processes were moderated by wives' employment. Although previous research has identified spouses' gender role attitudes as potential predictors of spouses' marital satisfaction, no study has examined these links in a dyadic model that elucidates how gender role attitudes may operate through processes to shape marital satisfaction and conditions under which associations may differ. We found that when spouses reported less sex-typed attitudes, their partners reported feeling more connected to them and more satisfied with the marriage, regardless of whether wives were employed. Our results suggest that marital satisfaction was highest for those Mexican-origin couples in which marital partners were less sex-typed in their attitudes about marital roles to the extent that partners' attitudinal role flexibility promoted spouses' feelings of warmth and connection to their partner.

Sobre la base de enfoques diádicos y perspectivas de matrimonio ecológicas y culturalmente fundamentadas, aplicamos un modelo de mediación e interdependencia actor-pareja en una muestra de 120 parejas de origen mexicano para examinar (a) las asociaciones que conectan las actitudes hacia los roles de género de los esposos y las esposas inmigrantes mexicanos con la satisfacción conyugal directamente e indirectamente mediante procesos conyugales (p. ej.: calidez y negatividad) y (b) si las asociaciones entre las actitudes hacia los roles de género de los cónyuges y los procesos conyugales estuvieron moderados por el empleo de las esposas. Aunque en investigaciones anteriores se han reconocido las actitudes hacia los roles de género de los cónyuges como posibles predictores de su satisfacción conyugal, ningún estudio ha analizado estas conexiones en un modelo diádico que aclare cómo las actitudes hacia los roles de género pueden funcionar mediante procesos para determinar la satisfacción conyugal y las condiciones en las cuales las asociaciones pueden diferir. Descubrimos que cuando los cónyuges informaron menos actitudes consideradas adecuadas para cada género, sus parejas informaron sentirse más conectadas con ellos y más satisfechas con el matrimonio, independientemente de si las esposas estaban empleadas o no. Nuestros resultados sugieren que la satisfacción conyugal fue mayor para las parejas de origen mexicano en las cuales los cónyuges tuvieron actitudes menos típicas de cada género con respecto a los roles conyugales hasta el punto de que la flexibilidad actitudinal hacia los roles de los integrantes de la pareja fomentó sentimientos de calidez y conexión con su pareja.

Bones and muscular dystrophies: what do we know?

PURPOSE OF REVIEW: Muscle and bone are intrinsically linked, and therefore, it is not surprising that many muscular dystrophies are associated with impaired bone health and increased risk of osteoporosis. Osteoporotic fracture is an important and preventable cause of morbidity and mortality. This article will firstly review the general causes of impaired bone health in muscular dystrophies and then focus on the evidence available for the diagnosis and treatment of osteoporosis in specific conditions. RECENT FINDINGS: With the exception of DMD, there is a paucity of data regarding bone health in muscular dystrophies. However, it appears that in common with all types of muscular dystrophies that cause a significant level of muscle weakness and disability there is an increased risk of falls, fractures and decreased vitamin D levels. A better understanding of the extent of the impaired bone health and underlying causes could help to identify potential new therapeutic agents and aid clinical care. SUMMARY: It would be prudent for clinicians to assess fracture risk in their muscular dystrophy patients and if appropriate, arrange surveillance and recommend vitamin D supplementation. Additionally, fracture should be considered in any patient presenting with new-onset bone pain.

Preterm birth and the timing of puberty: a systematic review.

BACKGROUND: An estimated 11% of births occur preterm, and survival is improving. Early studies suggested an association between preterm birth and earlier puberty. Given the adverse outcomes associated with early puberty this could have significant public health implications. The objective of this review was to assess the timing of puberty after preterm birth. METHODS: Pubmed, Embase, Popline, Global Health and Global Health Library were searched using terms relating to "premature birth", "menarche", "puberty" and "follow up studies". Inclusion criteria were a population consisting of pubertal or post-pubertal adolescents and adults; studies which defined preterm delivery in participants and compared outcomes to those after term delivery; and a quantitative assessment of pubertal onset. Assessment of risk of bias was conducted using principles from the Critical Appraisal Study Process. RESULTS: Our search identified 1051 studies, of which 16 met the inclusion criteria. In females, 8 studies found no association between preterm birth and the timing of menarche. Five studies found earlier onset in preterm infants, 1 found later onset, and 1 showed both earlier and later menarche, depending on birth weight. The range of effect of studies showing earlier menarche was - 0.94 to -0.07 years in the preterm group, with a median of - 0.3 years. In males, 2 studies showed earlier onset of puberty in the preterm group, 5 showed no difference, and 1 showed later onset. Most studies did not present outcomes in the form of a mean with standard deviation, precluding a meta-analysis. There was insufficient data to address potential confounding factors. CONCLUSIONS: The published evidence does not suggest that being born preterm leads to a significant acceleration in the onset of puberty. This should prove reassuring for public health purposes, and for clinicians counseling parents of infants born preterm.

MicroRNA-155 regulates monocyte chemokine and chemokine receptor expression in Rheumatoid Arthritis.

OBJECTIVE: To test the hypothesis that miR-155 regulates monocyte migratory potential via modulation of chemokine and chemokine receptor expression in RA, and thereby is associated with disease activity. METHODS: The miR-155 copy-numbers in monocytes from peripheral blood (PB) of healthy (n = 22), RA (n = 24) and RA SF (n = 11) were assessed by real time-PCR using synthetic miR-155 as a quantitative standard. To evaluate the functional impact of miR-155, human monocytes were transfected with control or miR-155 mimic, and the effect on transcript levels, and production of chemokines was evaluated by Taqman low-density arrays and multiplex assays. A comparative study evaluated constitutive chemokine receptor expression in miR-155-/- and wild-type murine (CD115 + Ly6C + Ly6G-) monocytes. RESULTS: Compared with healthy monocytes, the miR-155 copy-number was higher in RA, peripheral blood (PB) and SF monocytes (PB P < 0.01, and SF P < 0.0001). The miR-155 copy-number in RA PB monocytes was higher in ACPA-positive compared with ACPA-negative patients (P = 0.033) and correlated (95% CI) with DAS28 (ESR), R = 0.728 (0.460, 0.874), and with tender, R = 0.631 (0.306, 0.824) and swollen, R = 0.503 (0.125, 0.753) joint counts. Enforced-expression of miR-155 in RA monocytes stimulated the production of CCL3, CCL4, CCL5 and CCL8; upregulated CCR7 expression; and downregulated CCR2. Conversely, miR155-/- monocytes showed downregulated CCR7 and upregulated CCR2 expression. CONCLUSION: Given the observed correlations with disease activity, these data provide strong evidence that miR-155 can contribute to RA pathogenesis by regulating chemokine production and pro-inflammatory chemokine receptor expression, thereby promoting inflammatory cell recruitment and retention in the RA synovium.

Prophylactic oral bisphosphonate therapy in duchenne muscular dystrophy.

INTRODUCTION: We assessed prophylactic use of bisphosphonate (BP) in Duchenne muscular dystrophy (DMD) patients on glucocorticoid (GC) therapy. METHODS: Fifty-two DMD patients on daily GC were offered BP (oral risedronate). Patients were reviewed for tolerability, side effects, bone pain, and fracture frequency. Bone mineral density (BMD) was determined by annual dual energy X-ray absorptiometry. BP-treated patients were compared with 15 BP-naïve patients (untreated cohort). RESULTS: Side effects occurred in 9 patients. Thirty-six patients continued BP therapy for over 12 months (mean, 3.6 years). Five treated patients reported bone pain. Three treated patients suffered a vertebral fracture, significantly less than in the untreated cohort (5/15). Lumbar spine adjusted BMD Z-scores remained unchanged in treated patients and were significantly greater than in the untreated cohort. CONCLUSIONS: Prophylactic oral risedronate therapy was tolerated by most DMD patients. It appears to maintain BMD and may reduce fracture rate in DMD patients on GC. Muscle Nerve 54: 79-85, 2016.

Testosterone Treatment of Pubertal Delay in Duchenne Muscular Dystrophy.

BACKGROUND: The outlook for adolescents with Duchenne muscular dystrophy (DMD) has improved greatly as a result of corticosteroid use, but treatment will compromise growth and delay puberty. Whether exogenous testosterone can promote growth, development, and skeletal health is unclear. METHODS: We collected data retrospectively on growth and pubertal response in 14 adolescents with DMD who were treated with testosterone between 2008 and 2014. RESULTS: A total of 14 boys were treated at a median age of 14.5 years. Eight have finished treatment after a mean age of 3.1 years and the feedback from families was generally positive. The mean testicular volume pretreatment was 2.4 and 3.9 mL posttreatment. The mean baseline testosterone concentrations were < 1.0 and 5.4 nmol/L postintervention. Median height velocity increased from 0.45 cm/y before treatment to 3.6 cm/y after the treatment. The mean height gain was 14.2 cm. CONCLUSIONS: A broad range of testosterone preparations was used. Testosterone was generally well-liked, but side effects were experienced by some patients and the pubertal growth increment appears to be compromised. Few subjects had adult endogenous testosterone levels posttreatment. Controlled studies are required to determine the most appropriate treatment regimen and the precise impact of testosterone on key outcomes, such as muscle function and bone integrity. Clinicians will then be better placed to advise families about likely benefits and risks.