SummaryBackgroundKCNJ11 mutations cause permanent neonatal diabetes through pancreatic ATP-sensitive potassium channel activation. 90% of patients successfully transfer from insulin to oral sulfonylureas with excellent initial glycaemic control; however, whether this control is maintained in the long term is unclear. Sulfonylurea failure is seen in about 44% of people with type 2 diabetes after 5 years of treatment. Therefore, we did a 10-year multicentre follow-up study of a large international cohort of patients with KCNJ11 permanent neonatal diabetes to address the key questions relating to long-term efficacy and safety of sulfonylureas in these patients.MethodsIn this multicentre, international cohort study, all patients diagnosed with KCNJ11 permanent neonatal diabetes at five laboratories in Exeter (UK), Rome (Italy), Bergen (Norway), Paris (France), and Krakow (Poland), who transferred from insulin to oral sulfonylureas before Nov 30, 2006, were eligible for inclusion. Clinicians collected clinical characteristics and annual data relating to glycaemic control, sulfonylurea dose, severe hypoglycaemia, side-effects, diabetes complications, and growth. The main outcomes of interest were sulfonylurea failure, defined as permanent reintroduction of daily insulin, and metabolic control, specifically HbA1c and sulfonylurea dose. Neurological features associated with KCNJ11 permanent neonatal diabetes were also assessed. This study is registered with ClinicalTrials.gov, number NCT02624817.Findings90 patients were identified as being eligible for inclusion and 81 were enrolled in the study and provided long-term (>5·5 years cut-off) outcome data. Median follow-up duration for the whole cohort was 10·2 years (IQR 9·3–10·8). At most recent follow-up (between Dec 1, 2012, and Oct 4, 2016), 75 (93%) of 81 participants remained on sulfonylurea therapy alone. Excellent glycaemic control was maintained for patients for whom we had paired data on HbA1c and sulfonylurea at all time points (ie, pre-transfer [for HbA1c], year 1, and most recent follow-up; n=64)—median HbA1c was 8·1% (IQR 7·2–9·2; 65·0 mmol/mol [55·2–77·1]) before transfer to sulfonylureas, 5·9% (5·4–6·5; 41·0 mmol/mol [35·5–47·5]; p<0·0001 vs pre-transfer) at 1 year, and 6·4% (5·9–7·3; 46·4 mmol/mol [41·0–56·3]; p<0·0001 vs year 1) at most recent follow-up (median 10·3 years [IQR 9·2–10·9]). In the same patients, median sulfonylurea dose at 1 year was 0·30 mg/kg per day (0·14–0·53) and at most recent follow-up visit was 0·23 mg/kg per day (0·12–0·41; p=0·03). No reports of severe hypoglycaemia were recorded in 809 patient-years of follow-up for the whole cohort (n=81). 11 (14%) patients reported mild, transient side-effects, but did not need to stop sulfonylurea therapy. Seven (9%) patients had microvascular complications; these patients had been taking insulin longer than those without complications (median age at transfer to sulfonylureas 20·5 years [IQR 10·5–24·0] vs 4·1 years [1·3–10·2]; p=0·0005). Initial improvement was noted following transfer to sulfo...

Type 1 diabetes (T1D) is a chronic autoimmune disease leading to immune-mediated destruction of pancreatic beta cells, resulting in the need for insulin therapy. The incidence of T1D is increasing worldwide, thus prompting researchers to investigate novel immunomodulatory strategies to halt autoimmunity and modify disease progression. T1D is considered as a multifactorial disease, in which genetic predisposition and environmental factors interact to promote the triggering of autoimmune responses against beta cells. Over the last decades, it has become clear that vitamin D exerts anti-inflammatory and immunomodulatory effects, apart from its well-established role in the regulation of calcium homeostasis and bone metabolism. Importantly, the global incidence of vitamin D deficiency is also dramatically increasing and epidemiologic evidence suggests an involvement of vitamin D deficiency in T1D pathogenesis. Polymorphisms in genes critical for vitamin D metabolism have also been shown to modulate the risk of T1D. Moreover, several studies have investigated the role of vitamin D (in different doses and formulations) as a potential adjuvant immunomodulatory therapy in patients with new-onset and established T1D. This review aims to present the current knowledge on the immunomodulatory effects of vitamin D and summarize the clinical interventional studies investigating its use for prevention or treatment of T1D.

Pediatric cardiomyopathies, which are rare but serious disorders of the muscles of the heart, affect at least one in every 100,000 children in the USA. Approximately 40% of children with symptomatic cardiomyopathy undergo heart transplantation or die from cardiac complications within 2 years. However, a significant number of children suffering from cardiomyopathy are surviving into adulthood, making it an important chronic illness for both pediatric and adult clinicians to understand. The natural history, risk factors, prevalence and incidence of this pediatric condition were not fully understood before the 1990s. Questions regarding optimal diagnostic, prognostic and treatment methods remain. Children require long-term follow-up into adulthood in order to identify the factors associated with best clinical practice including diagnostic approaches, as well as optimal treatment approaches. In this article, we comprehensively review current research on various presentations of this disease, along with current knowledge about their causes, treatments and clinical outcomes.

Objective This cross sectional study examined the relationship between parental health literacy (HL), diabetes related numeracy, and parental perceived diabetes self-efficacy on glycemic control in a sample of young children with Type 1 DM. Methods Seventy primary caregivers of children (age 3–9 years) with Type 1 DM were recruited and surveyed at diabetes outpatient clinic visits. Patients’ medical histories were obtained by medical chart review. Results Parental diabetes related numeracy (r = −.52, p <.01), but not reading skills (r = −.25, p = NS) were inversely correlated with the child’s glycemic control (HbA1c). Parental perceived diabetes self-efficacy was also negatively correlated to their child’s HbA1c (r = −.47, p <.01). When numeracy and parental perceived diabetes self-efficacy were included as predictors of HbA1c, the model was significant (F = 12.93, p<.01) with both numeracy (β = − .46, p<.01) and parental perceived diabetes self-efficacy (β = − .36, p=.01) as significant predictors of HbA1c. Conclusions Data from this study highlight the importance of considering the role of parental numeracy, in health outcomes for children with Type 1 DM. Practice Implications: Practitioners should assess parental health literacy and consider intervention when needed.

Objective This study evaluated the rate of Hispanic children who have grandparents involved in caretaking and whether grandparents’ involvement has a negative impact on feeding practices, children's physical activity, and body mass index (BMI). Method One hundred and ninety-nine children and their parents were recruited at an elementary school. Parents completed a questionnaire regarding their children's grandparents’ involvement as caretakers and the feeding and physical activity practices of that grandparent when with the child. Children's height and weight were measured and zBMI scores were calculated. Results Forty-three percent of parents reported that there was a grandparent involved in their child's caretaking. Grandparents served a protective role on zBMI for youth of Hispanic descent, except for the Cuban subgroup. There was no relationship between grandparent involvement and feeding and physical activity behaviors. Conclusions In some cases grandparents may serve a protective function for childhood obesity. These results highlight the need for future research on grandparents and children's health, especially among Hispanic subgroups.

Purpose-The purpose of this study is to investigate parent reports of the diabetes care support their children receive in school, their concerns about diabetes management in school, and their knowledge of federal laws that protect children with diabetes. In addition, the study explores ethnic and socioeconomic status differences in diabetes management in school.Methods-An ethnically heterogeneous sample of 309 parents of children with diabetes was recruited from a community-based and a university-based diabetes outpatient clinic. Parents completed a survey assessing supports their child's school provides for diabetes care, worries about diabetes care in school, and awareness of federal laws that pertain to children with diabetes.Results-Many children did not have a written care plan or a nurse at school, but significantly more white children had these supports than Hispanic or black children. Most children were not allowed to check blood glucose levels or administer insulin in class. Most parents were worried about hyperglycemia and hypoglycemia in school, and most were not at all or only a little confident in the school's ability to care for diabetes. Most parents were not aware of federal laws, but high-income and white parents were more likely to be aware.Conclusions-According to parents in the current study, children receive inadequate diabetes management support in schools. Minority children are less likely to receive supports than white children. Parents are worried about diabetes management in school, but most do not have the knowledge of federal laws necessary to protect their children.Diabetes mellitus is one of the most common chronic conditions of childhood. 1 Type 1 diabetes is the most common diagnosis, with 1 in every 400 to 600 individuals younger than 20 years affected. 2 Recent studies suggest that the incidence of type 1 diabetes may be increasing in the United States and worldwide. 3,4 Although type 2 diabetes is less common among school-age children than type 1 diabetes, the incidence of type 2 diabetes has increased in the past several years in concordance with the unprecedented rates of obesity among youth. 5 The increasing prevalence of type 2 diabetes among overweight children and adolescents is especially dramatic among minorities. 4,6 In total, an estimated 18 700 children younger than 20 years are newly diagnosed with diabetes mellitus each year in the United States. 4 Diabetes self-care is complex and requires significant lifestyle adaptations that can be difficult for all people with diabetes; however, these changes are especially complicated for children and adolescents. Frequent monitoring of blood glucose levels is required, and constant access to glucose meters is a necessity. Food intake, particularly carbohydrates, must be carefully Balancing the array of diabetes self-care behaviors can be frustrating. 7 For children and adolescents, who spend approximately half of their waking hours at school, these frustrations can be compounded by difficulties getting the support...

Present findings indicate that insulin pump therapy does not have negative implications for HRQOL. They also suggest that interventions aiming to improve HRQOL in this population should target child, parent, and family adjustment and not focus solely on disease-related outcomes.

The distinction between long-term and short-term complications and complications occurring to ones' self or someone else with diabetes was supported. Assessment of perceived risks for short-term complications is important for this age group and should be addressed in interventions to improve adherence.