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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GEOZS, IJS, IMTLJ, KILJ, KISLJ, NLZOH, NUK, OILJ, PNG, SAZU, SBCE, SBJE, UL, UM, UPCLJ, UPUK, ZRSKP
Improvements in the function, quality of life, and longevity of patients with Duchenne muscular dystrophy (DMD) have been achieved through a multidisciplinary approach to management across a range of ...health-care specialties. In part 3 of this update of the DMD care considerations, we focus on primary care, emergency management, psychosocial care, and transitions of care across the lifespan. Many primary care and emergency medicine clinicians are inexperienced at managing the complications of DMD. We provide a guide to the acute and chronic medical conditions that these first-line providers are likely to encounter. With prolonged survival, individuals with DMD face a unique set of challenges related to psychosocial issues and transitions of care. We discuss assessments and interventions that are designed to improve mental health and independence, functionality, and quality of life in critical domains of living, including health care, education, employment, interpersonal relationships, and intimacy.
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GEOZS, IJS, IMTLJ, KILJ, KISLJ, NLZOH, NUK, OILJ, PNG, SAZU, SBCE, SBJE, UL, UM, UPCLJ, UPUK, ZRSKP
As COVID-19 vaccine distribution efforts continue, public health workers can strategize about vaccine promotion in an effort to increase willingness among those who may be hesitant.
In April 2020, we ...surveyed a national probability sample of 2279 U.S. adults using an online panel recruited through address-based sampling. Households received a computer and internet access if needed to participate in the panel. Participants were invited via e-mail and answered online survey questions about their willingness to get a novel coronavirus vaccine when one became available. The survey was completed in English and Spanish. We report weighted percentages.
Most respondents were willing to get the vaccine for themselves (75%) or their children (73%). Notably, Black respondents were less willing than White respondents (47% vs. 79%, p < 0.001), while Hispanic respondents were more willing than White respondents (80% vs. 75%, p < 0.003). Females were less likely than makes (72% vs. 79%, p < 0.001). Those without insurance were less willing than the insured (47% vs. 78%, p < 0.001). Willingness to vaccinate was higher for those age 65 and older than for some younger age groups (85% for those 65 and older vs. 75% for those 50-64, p < 0.017; 72% for those 35-49, p < 0.002; 70% for those 25-34, p = NS and 75% for ages 18-24, p = NS), but other groups at increased risk because of underlying medical conditions or morbid obesity were not more willing to get vaccinated than their lower risk counterparts.
Most Americans were willing to get a COVID-19 vaccine, but several vulnerable populations reported low willingness. Public health efforts should address these gaps as national implementation efforts continue.
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DOBA, IZUM, KILJ, NUK, PILJ, PNG, SAZU, SIK, UILJ, UKNU, UL, UM, UPUK
A coordinated, multidisciplinary approach to care is essential for optimum management of the primary manifestations and secondary complications of Duchenne muscular dystrophy (DMD). Contemporary care ...has been shaped by the availability of more sensitive diagnostic techniques and the earlier use of therapeutic interventions, which have the potential to improve patients' duration and quality of life. In part 2 of this update of the DMD care considerations, we present the latest recommendations for respiratory, cardiac, bone health and osteoporosis, and orthopaedic and surgical management for boys and men with DMD. Additionally, we provide guidance on cardiac management for female carriers of a disease-causing mutation. The new care considerations acknowledge the effects of long-term glucocorticoid use on the natural history of DMD, and the need for care guidance across the lifespan as patients live longer. The management of DMD looks set to change substantially as new genetic and molecular therapies become available.
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GEOZS, IJS, IMTLJ, KILJ, KISLJ, NLZOH, NUK, OILJ, PNG, SAZU, SBCE, SBJE, UL, UM, UPCLJ, UPUK, ZRSKP
The best treatment options for binge-eating disorder are unclear.
To summarize evidence about the benefits and harms of psychological and pharmacologic therapies for adults with binge-eating ...disorder.
English-language publications in EMBASE, the Cochrane Library, Academic OneFile, CINAHL, and ClinicalTrials.gov through 18 November 2015, and in MEDLINE through 12 May 2016.
9 waitlist-controlled psychological trials and 25 placebo-controlled trials that evaluated pharmacologic (n = 19) or combination (n = 6) treatment. All were randomized trials with low or medium risk of bias.
2 reviewers independently extracted trial data, assessed risk of bias, and graded strength of evidence.
Therapist-led cognitive behavioral therapy, lisdexamfetamine, and second-generation antidepressants (SGAs) decreased binge-eating frequency and increased binge-eating abstinence (relative risk, 4.95 95% CI, 3.06 to 8.00, 2.61 CI, 2.04 to 3.33, and 1.67 CI, 1.24 to 2.26, respectively). Lisdexamfetamine (mean difference MD, -6.50 CI, -8.82 to -4.18) and SGAs (MD, -3.84 CI, -6.55 to -1.13) reduced binge-eating-related obsessions and compulsions, and SGAs reduced symptoms of depression (MD, -1.97 CI, -3.67 to -0.28). Headache, gastrointestinal upset, sleep disturbance, and sympathetic nervous system arousal occurred more frequently with lisdexamfetamine than placebo (relative risk range, 1.63 to 4.28). Other forms of cognitive behavioral therapy and topiramate also increased abstinence and reduced binge-eating frequency and related psychopathology. Topiramate reduced weight and increased sympathetic nervous system arousal, and lisdexamfetamine reduced weight and appetite.
Most study participants were overweight or obese white women aged 20 to 40 years. Many treatments were examined only in single studies. Outcomes were measured inconsistently across trials and rarely assessed beyond end of treatment.
Cognitive behavioral therapy, lisdexamfetamine, SGAs, and topiramate reduced binge eating and related psychopathology, and lisdexamfetamine and topiramate reduced weight in adults with binge-eating disorder.
Agency for Healthcare Research and Quality.
Objective To examine the predictive ability of stage of hypoxic-ischemic encephalopathy (HIE) for death or moderate/severe disability at 18 months among neonates undergoing hypothermia. Study design ...Stage of encephalopathy was evaluated at <6 hours of age, during study intervention, and at discharge among 204 participants in the National Institute of Child Health and Human Development Neonatal Research Network Trial of whole body hypothermia for HIE. HIE was examined as a predictor of outcome by regression models. Results Moderate and severe HIE occurred at <6 hours of age among 68% and 32% of 101 hypothermia group infants and 60% and 40% of 103 control group infants, respectively. At 24 and 48 hours of study intervention, infants in the hypothermia group had less severe HIE than infants in the control group. Persistence of severe HIE at 72 hours increased the risk of death or disability after controlling for treatment group. The discharge exam improved the predictive value of stage of HIE at <6 hours for death/disability. Conclusions On serial neurologic examinations, improvement in stage of HIE was associated with cooling. Persistence of severe HIE at 72 hours and an abnormal neurologic exam at discharge were associated with a greater risk of death or disability.
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GEOZS, IJS, IMTLJ, KILJ, KISLJ, NUK, OILJ, PNG, SAZU, SBCE, SBJE, UL, UM, UPCLJ, UPUK
Objective To evaluate the relationship between abnormal feeding patterns and language performance on the Bayley Scales of Infant Development-Third Edition at 18-22 months adjusted age among a cohort ...of extremely premature infants. Study design This is a descriptive analysis of 1477 preterm infants born ≤26 weeks gestation or enrolled in a clinical trial between January 1, 2006 and March 18, 2008 at a National Institute of Child Health and Human Development Neonatal Research Network center who completed the 18-month neurodevelopmental follow-up assessment. At 18-22 months adjusted age, a comprehensive neurodevelopmental evaluation was performed by certified examiners including the Receptive and Expressive Language Subscales of the Bayley Scales of Infant Development-Third Edition and a standardized adjusted age feeding behaviors and nutritional intake. Data were analyzed using bivariate and multilevel linear and logistic regression modeling. Results Abnormal feeding behaviors were reported in 193 (13%) of these infants at 18-22 months adjusted age. Abnormal feeding patterns, days of mechanical ventilation, hearing impairment, and Gross Motor Functional Classification System level ≥2 each independently predicted lower composite language scores. Conclusions At 18 months adjusted age, premature infants with a history of feeding difficulties are more likely to have language delay. Neuromotor impairment and days of mechanical ventilation are both important risk factors associated with these outcomes.
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GEOZS, IJS, IMTLJ, KILJ, KISLJ, NUK, OILJ, PNG, SAZU, SBCE, SBJE, UL, UM, UPCLJ, UPUK
Objectives To compare 18- to 22-month cognitive scores and neurodevelopmental impairment (NDI) in 2 time periods using the National Institute of Child Health and Human Development's Neonatal Research ...Network assessment of extremely low birth weight infants with the Bayley Scales of Infant Development, Second Edition (Bayley II) in 2006-2007 (period 1) and using the Bayley Scales of Infant and Toddler Development, Third Edition (Bayley III), with separate cognitive and language scores, in 2008-2011 (period 2). Study design Scores were compared with bivariate analysis, and regression analyses were run to identify differences in NDI rates. Results Mean Bayley III cognitive scores were 11 points higher than mean Bayley II cognitive scores. The NDI rate was reduced by 70% (from 43% in period 1 to 13% in period 2; P < .0001). Multivariate analyses revealed that Bayley III contributed to a decreased risk of NDI by 5 definitions: cognitive score <70 and <85, cognitive or language score <70; cognitive or motor score <70, and cognitive, language, or motor score <70 ( P < .001). Conclusion Whether the Bayley III is overestimating cognitive performance or whether it is a more valid assessment of emerging cognitive skills than the Bayley II is uncertain. Because the Bayley III identifies significantly fewer children with disability, it is recommended that all extremely low birth weight infants be offered early intervention services at the time of discharge from the neonatal intensive care unit, and that Bayley scores be interpreted with caution.
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GEOZS, IJS, IMTLJ, KILJ, KISLJ, NUK, OILJ, PNG, SAZU, SBCE, SBJE, UL, UM, UPCLJ, UPUK
Globally, socioeconomic status (SES) is an important health determinant across a range of health conditions and diseases. However, measuring SES within low- and middle-income countries (LMICs) can be ...particularly challenging given the variation and diversity of LMIC populations. The current study investigates whether maternal SES as assessed by the newly developed Global Network-SES Index is associated with pregnancy outcomes (stillbirths, perinatal mortality, and neonatal mortality) in six LMICs: Democratic Republic of the Congo, Guatemala, India, Kenya, Pakistan, and Zambia. The analysis included data from 87,923 women enrolled in the Maternal and Newborn Health Registry of the NICHD-funded Global Network for Women's and Children's Health Research. Generalized estimating equations models were computed for each outcome by SES level (high, moderate, or low) and controlling for site, maternal age, parity, years of schooling, body mass index, and facility birth, including sampling cluster as a random effect. Women with low SES had significantly higher risks for stillbirth (p < 0.001), perinatal mortality (p = 0.001), and neonatal mortality (p = 0.005) than women with high SES. In addition, those with moderate SES had significantly higher risks of stillbirth (p = 0.003) and perinatal mortality (p = 0.008) in comparison to those with high SES. The SES categories were associated with pregnancy outcomes, supporting the validity of the index as a non-income-based measure of SES for use in studies of pregnancy outcomes in LMICs.
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DOBA, IZUM, KILJ, NUK, PILJ, PNG, SAZU, SIK, UILJ, UKNU, UL, UM, UPUK
To compare medical and developmental outcomes over the first 2 years of life in extremely preterm infants with bronchopulmonary dysplasia (BPD) who were discharged on supplemental oxygen via nasal ...cannula with outcomes of infants with a similar severity of respiratory illness who were discharged breathing in room air.
We performed a propensity score-matched cohort study. Eligible infants were born at <27 weeks' gestation, were receiving supplemental oxygen or respiratory support at 36 weeks' postmenstrual age, and were assessed at 18 to 26 months' corrected age. Study outcomes included growth, resource use, and neurodevelopment between discharge and follow-up. Outcomes were compared by using multivariable models adjusted for center and age at follow-up.
A total of 1039 infants discharged on supplemental oxygen were propensity score matched 1:1 to infants discharged breathing in room air. Infants on oxygen had a marginal improvement in weight
score (adjusted mean difference 0.11; 95% confidence interval CI 0.00 to 0.22), with a significantly improved weight-for-length
score (adjusted mean difference 0.13; 95% CI 0.06 to 0.20) at 22 to 26 months' corrected age. Infants on oxygen were more likely to be rehospitalized for respiratory illness (adjusted relative risk 1.33; 95% CI 1.16 to 1.53) and more likely to use respiratory medications and equipment. Rates of neurodevelopmental impairment were similar between the groups.
In this matched cohort of infants with BPD, postdischarge oxygen was associated with marginally improved growth and increased resource use but no difference in neurodevelopmental outcomes. Ongoing and future trials are critical to assess the efficacy and safety of postdischarge supplemental oxygen for infants with BPD.
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