Spinal muscular atrophy with respiratory distress type 1 (SMARD1) is a progressive, inherited neuromuscular disease manifesting with diaphragmatic paralysis in the first year of life. All patients need mechanical ventilation.

We describe the natural course of SMARD1, developed a scoring system, and defined prognostic values. The clinical outcome of the patients was heterogeneous, and residual enzymatic activity of the IGHMBP2 protein was associated with a more benign disease course. (Read the full article)

Children with sickle cell disease are at high risk of complications from influenza infection and have been recommended to receive annual influenza vaccine since the 1970s. Few safety studies, however, have examined the safety of influenza vaccine in this population.

This large cohort study did not find an association between influenza vaccination and hospitalization for sickle cell crises in children with sickle cell anemia. (Read the full article)

Chorioamnionitis is associated with preterm birth and an increased risk of common adverse outcomes of prematurity, including early-onset sepsis and neurodevelopmental impairment. The effect of chorioamnionitis on postnatal immune function and risk of late-onset infection is unknown.

Chorioamnionitis, which complicates many preterm births, is independently associated with a significantly reduced risk of late-onset sepsis in preterm infants. The findings suggest that chorioamnionitis may modulate the development of postnatal immunity in a clinically significant manner. (Read the full article)

Children with special health care needs (CSHCN) account for more than one-third of pediatric health care costs. Little is known regarding the impact of shared decision-making (SDM) over time on child health care expenditures and utilization.

In a national sample, we found that increasing SDM was associated with decreased health care costs and utilization for CSHCN. Results support prospective studies to determine if pediatric interventions to foster SDM reduce the financial burden of caring for CSHCN. (Read the full article)

Clinical trials in children with severe traumatic brain injury (TBI) are challenging. To date, no work has been published that permits clinical investigators to estimate the number and compositional features of sites from which to recruit children with severe TBI into clinical trials.

Children with severe TBI are infrequent. Less than 5% of all US hospitals discharged more than 78% of severe TBI cases. To maximize enrollment efficiency for future clinical trials, attention has to be paid to selecting appropriate hospital sites. (Read the full article)

The medical home is associated with beneficial outcomes in children with special health care needs and in the entire pediatric population. It is unknown if it benefits the majority of the pediatric population (ie, children without special health care needs).

This study is the first to demonstrate an association between the medical home and beneficial health care utilization, child health, and health-promoting behavior outcomes in children without special health care needs. (Read the full article)

Eating disorders are characterized by preoccupation with weight and shape, which is manifested by a refusal to maintain a normal weight. An exact determination of expected body weight (EBW) is critical for diagnosis and clinical management of these disorders.

The McLaren and Moore methods present with several limitations when calculating EBW for adolescents with eating disorders. A commonly agreed upon method for EBW calculation such as the BMI percentile method is recommended for clinical and research purposes. (Read the full article)

Previous analyses have suggested that center volume is associated with outcome in children undergoing heart surgery. There are limited data regarding factors that may mediate this volume–outcome relationship.

A multicenter analysis of 35 776 children revealed that the higher mortality observed at lower volume centers may be related to a higher rate of mortality in those with postoperative complications, rather than a higher rate of complications alone. (Read the full article)

Chronic wet cough is a common symptom well recognized by pediatricians. Protracted bacterial bronchitis is defined as more than 4 weeks of wet cough that responds to antibiotic treatment. Diagnosis of protracted bacterial bronchitis is not readily accepted by pediatricians

Children with chronic wet cough often have bronchitis, which is evident during bronchoscopy. Purulent bronchial secretions suggest the presence of bacterial infection. Children with chronic wet cough frequently have a bacterial infection of the lower airway. (Read the full article)

Newborn screening (NBS) programs may store bloodspot samples and use them for secondary purposes, such as research. Recent public controversies and lawsuits over storage and secondary uses underscore the need to engage the public on these issues.

This public engagement study identifies values underlying citizens’ acceptance of and discomfort with research from NBS samples. Well-designed methods of public education and civic discourse on the risks and benefits of storage and secondary uses of NBS samples are required. (Read the full article)

The effectiveness of intravenous immunoglobulin treatment of patients with Kawasaki disease within 9 days of illness has been established. However, the effectiveness of such treatment ≥10 days after illness onset has not yet been clarified.

Intravenous immunoglobulin treatment ≥10 days after illness onset was observed to be effective for achieving inflammation resolution. Patients who are strongly suspected to have Kawasaki disease and demonstrate ongoing inflammation should therefore be treated as soon as possible. (Read the full article)

Candidate genes involved in thrombophilia, inflammation, and preterm birth have previously been associated with cerebral palsy. Most studies to date have included small cohorts, did not allow for multiple testing, and require replication.

This study of children with cerebral palsy and their mothers did not confirm previously reported candidate gene associations. Prothrombin gene mutation was associated with hemiplegia in children born at term to mothers with a reported infection during pregnancy. (Read the full article)

Nasal continuous positive airway pressure (NCPAP) is a noninvasive ventilatory support that may reduce the need for mechanical ventilation in preterm infants with respiratory distress syndrome. Heliox, a helium-oxygen mixture, has shown positive effects, especially in obstructive diseases.

NCPAP with heliox reduces the need for mechanical ventilation in preterm infants with respiratory distress syndrome in comparison with NCPAP with medical air. (Read the full article)

Bronchiolitis is the most common cause for hospital admission in patients aged <1 year. Hypoxia is a common reason for admission. Despite a multitude of studies looking at various treatment strategies, no clear benefit has been found.

With oxygen therapy being the main therapeutic option, home oxygen offers a novel way to manage bronchiolitis. This study shows that home oxygen is a safe and effective way to decrease hospital admissions in a select group of patients. (Read the full article)

Neglect cases in Child Protective Services often receive home-based interventions, but their success in preventing maltreatment recidivism has been elusive. Structured, behavioral skills models, such as SafeCare, are promising but have not been tested in full-scale implementation trials.

This cluster trial experiment demonstrates significant maltreatment recidivism reduction due to implementing the SafeCare model in a fully scaled-up statewide system. The findings support adopting the SafeCare model for these types of services. (Read the full article)

Community-acquired pneumonia (CAP) is a common pediatric illness caused by Streptococcus pneumoniae. New pediatric Infectious Diseases Society of America CAP guidelines are now available recommending ampicillin as empirical treatment of children hospitalized with uncomplicated CAP.

This study found that a CAP guideline led to an increase in the narrow-spectrum antibiotic ampicillin. Additionally, an increase in the use of amoxicillin at discharge was observed. Furthermore, change in therapy did not lead to increased adverse outcomes. (Read the full article)

Studies in the Netherlands show that pubertal blockade at Tanner 2/3 prevents unwanted sex characteristics and improves psychological functioning. Endocrine Society guidelines (2009) recommend pubertal suppression for adolescents with gender identity disorder until approximately age 16.

This is the first study of a US cohort of children and adolescents with gender identity disorder. Patients were referred for medical treatment to a pediatric center that supports a multidisciplinary Gender Management Service. (Read the full article)

General measures discourage all self-referrals to the emergency department. For adults, self-referral to the emergency department has been associated with nonsevere disease, whereas severity of illness of self-referred children is still unknown.

One in four parents properly judged and acted on their febrile child’s illness by presentation to the emergency department on their own initiative. Measures to discourage parents from self-referral may potentially result in delayed or missed diagnoses. (Read the full article)

Active termination of life in newborns with myelomeningocele because of assumed suffering in these newborns has been extensively discussed. However, the level of discomfort and pain in these newborns has never been substantially assessed.

This is the first study presenting quantitative data on discomfort and pain in newborns with myelomeningocele. Therefore, it can be of guidance in the choice of treatment: either active treatment or palliative care in the context of end-of-life decisions. (Read the full article)

Children with special health care needs present clinically with varied functional difficulties across an array of health conditions. Little attention has been given to the interaction of these descriptors at a population level, thereby not addressing the complexity of functional difficulties and their impact on the health of CSHCN.

The data demonstrate the relationships among functional difficulties and health conditions, which then improve our understanding of CSHCN and their needs. Functional difficulties contribute significantly to outcomes, such as emergency room visits, parental work patterns, and limitations in daily activities, and have implications for practice, training, policy, and research. (Read the full article)

Rare diseases in childhood can be debilitating and require lifelong care. Since 1983, the Orphan Drug Act incentives have stimulated the development and significantly improved the availability of treatment products for patients with rare diseases.

We report an increasing pediatric orphan product designations and approvals from 2000 to 2009. The trend indicates that the Orphan Drug Act has continued to address this important unmet need. (Read the full article)

Few studies have examined the reliability of clinical findings in pediatric appendicitis. Clinical prediction rules are most useful if the included variables are reliable across practice settings and practitioners.

Among children who present with possible appendicitis, the interrater reliability varied considerably for patient history and physical examination variables. Those variables with the highest degree of reliability may be best suited for inclusion in appendicitis clinical prediction rules. (Read the full article)

Strategies to assist patients in achieving optimal chronic disease self-management are critical. The complex family and regimen issues surrounding pediatric type 1 diabetes management suggest the need to integrate such strategies into routine clinical care.

This study demonstrates the efficacy of a practical, low-intensity behavioral intervention delivered during routine care for improving glycemic outcomes. Findings indicate that the approach may offer a potential model for integrating medical and behavioral sciences to improve health care. (Read the full article)

Previous studies have identified barriers to providing optimal pediatric palliative care, including general communication issues between clinicians and family members. However, there is a paucity of data regarding the barriers specifically relating to advance care discussions.

This study identifies significant barriers to advance care discussions for children with life-threatening conditions. Clinicians perceive parental issues as the most common impediments to these discussions. Furthermore, providers believe that advance care discussions happen too late in the course of illness. (Read the full article)

Postpartum vitamin A supplementation is a strategy to combat vitamin A deficiency and seems to reduce maternal/infant morbidity and mortality. However, controversies exist regarding which dose has a greater efficacy, 200 000 IU (WHO protocol) or 400 000 IU (IVACG protocol).

In this study, postpartum maternal supplementation with 400 000 IU of vitamin A did not provide any additional beneficial effect in reducing infant morbidity compared with the standard dose of 200 000 IU. (Read the full article)

Early administration of oral corticosteroids is essential for children presenting to emergency departments with moderate to severe acute asthma exacerbations, because subsequent admission need is directly related to time to receipt of systemic steroids, yet delays to administration remain long.

A medical directive allowing nurse initiation of oral corticosteroids before physician assessment was associated with improved quality and efficiency of care provided in the pediatric emergency department by ensuring implementation of evidence-based practice. (Read the full article)

Survival takes precedence for adolescent patients with cancer and their families. Patients may not discuss their treatments’ potential to damage their reproductive capacity, which has significant psychological late effects in survivorship.

Strong reproductive concerns of adolescents with cancer may not be captured on current health-related quality of life instruments and may be neglected by parents’ unawareness. Parent-proxy reports of adolescent reproductive concerns are not suitable for capturing specific emotions and feelings. (Read the full article)

Insulin is commonly used to treat neonatal hyperglycemia, but there are few data to support its use. Tight glycemic control with insulin improves outcome in diabetic patients, but it is not known whether it is effective in hyperglycemic preterm infants.

Tight glycemic control with insulin in hyperglycemic preterm neonates decreases the rate of linear growth despite increased weight and occipitofrontal head circumference gain and increases the risk of hypoglycemia. Insulin may not be a safe and effective treatment in hyperglycemic preterm neonates. (Read the full article)

The survival of extremely low birth weight infants in resource-limited situations is dismal due to limited neonatal intensive care facilities. Nasal continuous positive airway pressure resulted in an increased survival of these infants, but many still require back-up ventilation.

Nasal continuous positive airway pressure and intubation, surfactant, and extubation practiced in a neonatal high care ward with limited resources and limited back-up ventilatory facilities can improve the survival of extremely low birth weight infants. (Read the full article)

Cross-sectional studies have shown an increased risk of mental health difficulties in children with developmental coordination disorder. However, there has been limited longitudinal research in this area controlling for confounding factors and assessing the role of potential mediators.

Children with "probable" developmental coordination disorder at 7 years had a significantly increased risk mental health difficulties at 10 years. Protective factors for self-reported depression included high IQ, high self-esteem, good social communication skills, and the absence of bullying. (Read the full article)

The presence of malodorous urine is often mentioned as one of the clinical manifestations of urinary tract infection (UTI) in young children, yet the few studies looking at this symptom are contradictory.

Our study demonstrates that malodorous urine as reported by parents increases the likelihood of UTI among young children evaluated for suspected UTI. However, this association is not strong enough to definitely rule in or out a diagnosis of UTI. (Read the full article)

Autism is widely considered a heterogeneous disorder in terms of etiology and phenotype. Although autism is usually a lifelong disorder, little is known about the rate or timing of how children develop regarding their communication and social functioning.

Utilizing annual evaluations for a large population of children with autism, we describe the 6 most common trajectories from diagnosis through age 14 years. Trajectories revealed considerable variation, and high socioeconomic status children were more likely to experience rapid improvement. (Read the full article)

Complementary and alternative medicine (CAM) use for pediatric asthma is increasing. It is well known that effective asthma management depends on patient adherence to treatment. The authors of previous cross-sectional studies have linked CAM use with decreased adherence to conventional asthma treatment regimens.

This longitudinal data set was unique, allowing us to focus on patients who initiated CAM and to follow subsequent asthma medication adherence. We found that CAM use was not associated with adherence, suggesting that patients may practice CAM alongside conventional therapies. (Read the full article)

Trisomy 13 and trisomy 18, common chromosomal abnormalities, are generally considered fatal within the first year after birth, although some children live longer. Little is known, however, about the inpatient medical courses of these infants and children.

Evaluation of nationally representative hospitalization data demonstrates that a significant number of children with trisomy 13 and trisomy 18 live beyond 1 year of age and that the care they receive includes both medical and surgical treatments. (Read the full article)

Rural Alaska children have high rates of hospitalization with lower respiratory tract infections from a variety of pathogens. Past studies of risk factors for respiratory syncytial virus infection associated medically high-risk status, household crowding, and infant feeding practices with hospitalization.

This study reveals the importance of medically high-risk status and infant feeding practices as important factors in respiratory hospitalization. In addition, we identified woodstove use and the absence of 2 or more sinks in household as risk factors for hospitalization. (Read the full article)

Asthma disparities exist, with Latino children of Caribbean descent at risk for poor disease control. Controller medications reduce symptoms; however, medication adherence remains suboptimal. Identifying what factors predict poor medication adherence in at-risk groups could identify important treatment targets.

This study is the first to assess objective rates of medication use among children with asthma in Puerto Rico. Findings suggest that interventions incorporating family resources and addressing parental beliefs about medications may be of benefit across cultural groups. (Read the full article)

Dimenhydrinate, an antihistaminic agent, is a widely used drug in Canada and Europe. It limits stimulation of the vomiting center via the vestibular system. Multiple studies have shown its effectiveness in the treatment of vertigo and postoperative nausea and vomiting.

Dimenhydrinate, when given orally, did not significantly decrease the frequency of vomiting in children with acute gastroenteritis compared with placebo. The reported adverse effect proportions were similar for the dimenhydrinate and placebo groups. (Read the full article)

Some researchers have suggested that individuals with Down syndrome (DS) are protected from atherosclerotic disease; however, recent data from 2 large cohort studies of individuals with DS are significant for increased mortality from ischemic heart disease and cerebrovascular disease.

This study compares lipid profiles among children with DS and their siblings, highlighting the presence of a less favorable lipid profile in this high-risk population. (Read the full article)

Previous research has identified low rates of employment and postsecondary education for youth with autism, but generalizability has been limited by small samples.

Using national data, the authors of this study found that youth with autism are at high risk for no postsecondary education or employment, especially in the first 2 years after high school. Findings highlight the need for improved transition planning. (Read the full article)

Previous research on injuries related to bottle, pacifier, and sippy cup use has largely focused on case reports of infant injuries or fatalities attributed to pacifiers or pacifier parts causing asphyxiation or to bottle warming causing burns.

This study is the first to use a nationally representative sample to investigate the range of injuries requiring emergency department visits associated with bottles, pacifiers, and sippy cups among children aged <3 years. (Read the full article)

A single inhalation of laninamivir octanoate has previously been shown to be as effective as repeated doses of zanamivir in vitro and in vivo, but it is not known whether this is also the case for children.

Median time to fever resolution was not significantly different between laninamivir octanoate and zanamivir for pediatric patients with influenza. The severity of influenza symptoms and the frequency of complications were similar in the 2 groups. (Read the full article)

Survival of children born with Down syndrome has been improving, but few studies have used population-based data to examine the influence of fetal and maternal characteristics on survival.

This study examined predictors of survival for children born with Down syndrome using population-based data from the UK Northern Congenital Abnormality Survey and shows that year of birth, gestational age, birth weight, and presence of additional anomalies influence survival status. (Read the full article)

The hypothesis that maternal prenatal folic acid lowers risk for childhood malignancy in offspring is supported by experimental and epidemiologic evidence, including 2 Canadian ecologic studies that showed inverse associations for some cancer types in the very young.

Examining Surveillance Epidemiology and End Results Program data, a decrease in the incidence of some childhood cancers (Wilms tumor, primitive neuroectodermal tumors) was observed in those <5 years after mandatory US folic acid fortification, with stronger effects detected in infants. (Read the full article)

Observation studies and short-term prospective intervention studies have shown that physical activity positively affects the accrual of bone mass and size during growth; however, fracture risk has not been evaluated.

This study reports the long-term results of a prospective intervention with increased physical activity at a population-based level and for the first time evaluated the clinical relevant end point, fracture risk. (Read the full article)

Children with specific language impairment are at risk for social difficulties. However, whether this occurs adaptively as a result of language impairment or occurs as a result of an underlying deficit in social cognition remains unclear.

We used eye tracking to explore how children with specific language impairment view social scenes. The overall gaze behavior resembled that of typically developing children. Significant attention to the speaker’s mouth may result in receiving less social-emotional information from the eyes. (Read the full article)

The causes of Perthes disease are unknown. There is considerable evidence that the disease has a vascular mechanism, although the nature of this is unknown. There is some suggestion that affected individuals may have a heightened cardiovascular risk in adulthood.

Children with Perthes disease have reduced vascular caliber, which is independent of body height, and abnormal functional vascular measures. These findings may be important in the mechanism of disease and may have implications on long-term vascular morbidity. (Read the full article)

Regular use of an integrated personal health record (PHR) may lead to improved outcomes through improved care coordination, communication, and patient empowerment. A limited number of studies have examined integrated PHR use for children.

Parents of children with chronic disease appear willing to use an integrated PHR to address health care needs for their child. PHRs may lead to improved health care and outcomes by enabling more coordinated care for children with chronic disease. (Read the full article)

Male circumcision reduces risk of HIV acquisition in men by 60% and is associated with other health benefits. Compared with adult circumcision, infant male circumcision is safer, less expensive, and represents a cost-saving intervention for HIV prevention in many settings.

IMC is little known in East Africa and is not routinely practiced. This is the first study to assess acceptability and uptake of IMC in East Africa among parents who were actually offered the procedure. (Read the full article)

Chronic fatigue syndrome (CFS) is a disabling illness. Some children with chronic disabling fatigue are housebound. Some primary care physicians find making the diagnosis of CFS difficult.

Chronic disabling fatigue is more common in 13-year-olds than previously realized with a population prevalence of ~2.0%. Chronic disabling fatigue in teenagers is more common in those with higher levels of early family adversity. (Read the full article)

Neonatal hyperbilirubinemia may progress to bilirubin encephalopathy. Findings from previous studies on long-term development of neonatal hyperbilirubinemia are conflicting.

Using Ages and Stages Questionnaire, we observed no association between bilirubin exposure and overall development in 1- to 5-year-old children who in the neonatal period had total serum bilirubin level ≥25 mg/dL and no or only minor neurologic symptoms. (Read the full article)