The Role of Online Social Support in Supporting and Educating Parents of Young Children With Special Health Care Needs in the United States: A Scoping Review.

BACKGROUND: When parents of young children with special health care needs (CSHCN) receive their child's diagnosis, they encounter information they may not understand, emotions they may not know how to cope with, and questions about their child's immediate and long-term future that frequently lack answers. The challenge of health care providers is how to prepare parents for caring for their CSHCN, for coping with any resulting challenges, and for accessing the systems and services that can assist them. OBJECTIVE: The purpose of this work was to review evidence of the information and support needs of parents of young CSHCN and to determine whether online social support can serve as an avenue for learning and empowerment for these parents. METHODS: A scoping review identified the challenges, coping mechanisms, and support needs among parents of CSHCN, and the reach and effectiveness of digital technologies with these families and health care providers. We also conducted interviews with professionals serving parents of CSHCN. RESULTS: The literature review and interviews suggested that parents best learn the information they need, and cope with the emotional challenges of raising a CSHCN, with support from other parents of CSHCN, and that young parents in recent years have most often been finding this parent-to-parent support through digital media, particularly social media, consistent with the theory of online social support. Evidence also shows that social media, particularly Facebook, is used by nearly all women aged 18-29 years across racial and socioeconomic lines in the United States. CONCLUSIONS: Parents of young CSHCN experience significant stress but gain understanding, receive support, and develop the ability to care for and be advocates for their child through parent-to-parent emotional and informational social support. Online social support is most effective with young adults of childbearing age, with social media and apps being the most useful within the theoretical framework of social support. This opens new opportunities to effectively educate and support parents of young CSHCN. Providers seeking to inform, educate, and support families of CSHCN should develop strategies to help parents find and use social support through digital resources to facilitate their emotional adjustment and practical abilities to care for and access services for their child.

Imatinib mesylate for plexiform neurofibromas in patients with neurofibromatosis type 1: a phase 2 trial.

BACKGROUND: Plexiform neurofibromas are slow-growing chemoradiotherapy-resistant tumours arising in patients with neurofibromatosis type 1 (NF1). Currently, there are no viable therapeutic options for patients with plexiform neurofibromas that cannot be surgically removed because of their proximity to vital body structures. We undertook an open-label phase 2 trial to test whether treatment with imatinib mesylate can decrease the volume burden of clinically significant plexiform neurofibromas in patients with NF1. METHODS: Eligible patients had to be aged 3-65 years, and to have NF1 and a clinically significant plexiform neurofibroma. Patients were treated with daily oral imatinib mesylate at 220 mg/m(2) twice a day for children and 400 mg twice a day for adults for 6 months. The primary endpoint was a 20% or more reduction in plexiform size by sequential volumetric MRI imaging. Clinical data were analysed on an intention-to-treat basis; a secondary analysis was also done for those patients able to take imatinib mesylate for 6 months. This trial is registered with ClinicalTrials.gov, number NCT01673009. FINDINGS: Six of 36 patients (17%, 95% CI 6-33), enrolled on an intention-to-treat basis, had an objective response to imatinib mesylate, with a 20% or more decrease in tumour volume. Of the 23 patients who received imatinib mesylate for at least 6 months, six (26%, 95% CI 10-48) had a 20% or more decrease in volume of one or more plexiform tumours. The most common adverse events were skin rash (five patients) and oedema with weight gain (six). More serious adverse events included reversible grade 3 neutropenia (two), grade 4 hyperglycaemia (one), and grade 4 increases in aminotransferase concentrations (one). INTERPRETATION: Imatinib mesylate could be used to treat plexiform neurofibromas in patients with NF1. A multi-institutional clinical trial is warranted to confirm these results. FUNDING: Novartis Pharmaceuticals, the Indiana University Simon Cancer Centre, and the Indiana University Herman B Wells Center for Pediatric Research.

Inclusion of Children in Clinical Research: The Role of Advocacy and a Personal Journey.

Many groups have historically been excluded from clinical research. It has required vigorous, long-term advocacy efforts for better inclusion of women and children across racial and ethnic groups. To understand who is included in clinical research, data are required. A personal journey of advocacy requiring the National Institutes of Health to report inclusion in clinical studies by age was ultimately accomplished by federal legislation.

Corticosteroids increase protein breakdown and loss in newly diagnosed pediatric Crohn disease.

Children with Crohn disease have altered growth and body composition. Previous studies have demonstrated decreased protein breakdown after either corticosteroid or anti-TNF-α therapy. The aim of this study was to evaluate whole body protein metabolism during corticosteroid therapy in children with newly diagnosed Crohn disease. Children with suspected Crohn disease and children with abdominal symptoms not consistent with Crohn disease underwent outpatient metabolic assessment. Patients diagnosed with Crohn disease and prescribed corticosteroid therapy returned in 2 wk for repeat metabolic assessment. Using the stable isotopes [d5] phenylalanine, [1-(13)C] leucine, and [(15)N(2)] urea, protein kinetics were determined in the fasting state. Thirty-one children (18 controls and 13 newly diagnosed with Crohn disease) completed the study. There were no significant differences in protein breakdown or loss between patients with Crohn disease at diagnosis and controls. After corticosteroid therapy in patients with Crohn disease, the rates of appearance of phenylalanine (32%) and leucine (26%) increased significantly, reflecting increased protein breakdown, and the rate of appearance of urea also increased significantly (273%), reflecting increased protein loss. Whole body protein breakdown and loss increased significantly after 2 wk of corticosteroid therapy in children with newly diagnosed Crohn disease, which may have profound effects on body composition.

Effects of recombinant human growth hormone on protein turnover in the fasting and fed state in adolescents with Crohn disease.

The primary purpose of this study was to test whether recombinant human growth hormone (rhGH) supplementation would enhance protein synthesis and accretion of lean body mass. Eight adolescents (six males and two females; 17.2 +/- 2.6 years; age range, 13.7-21.2 years) participated in a randomized double-blind placebo-controlled cross-over trial of rhGH. We employed stable isotopes to measure proteolysis and protein synthesis during fasting and fed conditions during two 6-month treatment conditions. We also measured bone mineral density (BMD), markers of bone turnover, and body composition. Whole-body proteolysis, phenylalanine catabolism, and protein synthesis did not differ during treatment with rhGH vs. placebo. Enteral nutrition suppressed proteolysis and increased protein synthesis similarly during placebo and rhGH treatments. We conclude that rhGH is unlikely to provide sufficient metabolic benefit to warrant its use as an adjunct treatment in clinically stable adolescents with Crohn disease. A high prevalence of vitamin D deficiency and suboptimal BMD existed, which deserves further investigation and clinical attention.

Women's health care providers' attitudes toward research in pregnancy.

OBJECTIVE: To evaluate women's health care providers' attitudes towards research in pregnancy. STUDY DESIGN: An online, anonymous survey given to health care providers and personnel directly involved in the care of pregnant women at 3 hospitals. The survey ascertained research involvement and attitudes toward both research in general and research on pregnant women. RESULTS: One hundred thirty-one people responded to the survey (27%). The majority of health care providers supported research, with 83.2% encouraging pregnant women to participate in a research study. However, there was a consistent difference between their attitudes toward encouraging others to participate while pregnant and whether or not they would consent themselves or have their partner consent to participate in research while pregnant. CONCLUSION: While women's health care workers are generally supportive of research in pregnancy, a discrepancy exists between what they would encourage others to do and what they would do. In order to promote recruitment of pregnant women into research studies, education of all health care workers involved in pregnant women's care should be a major focus.

Energy expenditure in extremely low birth weight infants near time of hospital discharge.

OBJECTIVE: To test the hypothesis that total energy expenditure is significantly higher in extremely low birth weight (ELBW) infants compared with healthy term infants near the time of discharge. STUDY DESIGN: This study was designed to determine total energy expenditure and body composition in a group of ELBW infants nearing discharge receiving full-volume enteral feedings of fortified breast milk or postdischarge formula (Neosure) (n = 10; mean birth weight, 0.8 +/- 0.1 kg; mean gestational age, 26 +/- 0.8 weeks; mean age at study, 68 +/- 9 days; mean postconceptional age, 36 +/- 1 weeks) and compare them with healthy term newborns all receiving breast milk (n = 14; mean birth weight, 3.5 +/- 0.5 kg; mean gestational age, 39.0 +/- 1.4 weeks; mean age at study, 2.3 +/- 1 days). Body composition and total energy expenditure were measured using the doubly labeled water method over a 7-day period. RESULTS: Mean total energy expenditure was significantly higher in the ELBW infants compared with the term infants (89 +/- 22 kcal/kg/day vs 58 +/- 19 kcal/kg/day; P

Carbohydrate and lipid metabolism following infliximab therapy in pediatric Crohn's disease.

Improvements in insulin resistance after anti-TNF-alpha therapy have been reported in inflammatory conditions, although no changes were noted in adult patients with Crohn's disease. There is no information concerning insulin resistance and substrate metabolism in children with Crohn's disease after anti-TNF-alpha therapy. Our aim was to describe changes in carbohydrate and lipid metabolism in children with active Crohn's disease after their initial dose of infliximab. Children with active Crohn's disease underwent measurement of plasma insulin and glucose just before and 2 wk after their initial infusion of infliximab, an anti-TNF-alpha antibody. In addition, resting energy expenditure, with determination of both carbohydrate and lipid oxidation rates, was determined. Measurements were conducted in both fasting and parenterally fed states. Despite no changes in resting energy expenditure, a significant reduction (p < 0.05) in RQ (5%) and carbohydrate oxidation rate (24%), with a corresponding increase in lipid oxidation rate (42%) was found during parenteral nutrition infusion. No differences in plasma insulin, glucose, and insulin resistance were noted when comparing pre- and postinfliximab measurements.

Effects of infliximab and parenteral nutrition on albumin and fibrinogen synthesis rates in pediatric Crohn disease.

OBJECTIVES: Tumor necrosis factor-alpha (TNF-alpha) may play a significant role in growth disturbance in pediatric Crohn disease. The aim of this study was to determine the effects of anti-TNF-alpha therapy on albumin and fibrinogen synthesis during both fasting and parenteral nutrition infusion in pediatric patients with active Crohn disease. PATIENTS AND METHODS: Children with active Crohn disease scheduled for their initial dose of infliximab underwent assessment immediately before and 2 weeks following infliximab infusion. Using the stable isotope [d5] phenylalanine, rates of fractional and absolute albumin and fibrinogen synthesis were calculated. Measurements were made in both the fasting and parenterally fed states. RESULTS: Fifteen children (mean age 14.9 +/- 0.3) completed the study. The mean serum albumin changed from 3.59 +/- 0.08 to 3.66 +/- 0.04 g/dL, and the mean fibrinogen level decreased from 230 +/- 17 to 187 +/- 8 mg/dL (P < 0.05) following infliximab therapy. During fasting, there were no changes in albumin and fibrinogen synthesis rates following infliximab. During parenteral nutrition infusion, the fractional albumin synthesis rate changed from 11.8% to 15.1%/day (P = 0.06), and the absolute albumin synthesis rate increased from 192 to 248 mg x kg(-1) x day(-1) (P < 0.05), whereas no changes in fibrinogen synthesis rates were observed. Synthesis rates of albumin and fibrinogen were increased during parenteral nutrition infusion compared with the fasting state. CONCLUSIONS: Following infliximab therapy, during parenteral nutrition infusion, albumin synthesis increased significantly. Conversely, serum fibrinogen levels decreased following infliximab therapy in the absence of significant change in synthesis rates.

Regulation of proteolysis and optimal protein accretion in extremely premature newborns.

Growth outcomes for extremely premature infants remain poor, and improving growth in this population will require a better understanding of how to limit proteolysis and promote protein accretion. Extremely premature infants exhibit high rates of proteolysis that are unrestrained by physiologic increases in insulin, intravenous amino acids, and full parenteral nutrition. Imbalances in current amino acid solutions may be in part responsible for the inability of parenteral nutrition to reduce proteolysis in preterm infants. However, amino acids in parenteral nutrition are effective for increasing protein synthesis in extremely preterm infants, which leads to improved protein balance. Current evidence suggests that early administration of 3 g amino acids kg(-1)d(-1) to extremely premature infants is safe and effective. Enteral nutrition may be more effective than parenteral nutrition in limiting proteolysis and producing protein accretion in preterm infants, but the protein content of current preterm formulas may be inadequate for supporting optimal growth in this population. Important areas of future research include determining whether altered intravenous amino acid solutions can better effect reductions in proteolysis, investigating the effect of enteral nutrition on proteolysis and protein accretion, and conducting a large randomized controlled trial of formula with a higher protein content.

Protein and energy metabolism response to the initial dose of infliximab in children with Crohn's disease.

BACKGROUND: Tumor necrosis factor-alpha (TNF-alpha) may contribute to the alterations in protein and energy metabolism present in children with Crohn's disease (CD), who frequently suffer from growth disturbance. We hypothesized that anti-TNF-alpha therapy would reduce protein losses, due to decreased proteolysis and increased protein synthesis, and that anti-TNF-alpha therapy would decrease resting energy expenditure. METHODS: Children with active CD underwent metabolic assessment immediately before and 2 weeks following initial infliximab infusion. Using the stable isotopes [d5] phenylalanine and [1-13C] leucine, 2 independent measures of protein metabolism were determined during fasting and in response to parenteral nutrition. Energy expenditure, determined by indirect calorimetry, was measured in fasting and parenterally fed states. RESULTS: Fifteen children completed the study. Following infliximab therapy, significant reductions in proteolysis (P < 0.05) were noted in the fasting state (8%-11%) and during parenteral nutrition infusion (10%-12%). Phenylalanine utilization for protein synthesis decreased significantly (8%-13%) following infliximab (P < 0.05). Protein balance was not significantly altered. No significant changes in energy expenditure were observed following infliximab in fasting or parenterally fed states. Supplementation with parenteral nutrition resulted in significantly decreased proteolysis (8%-21%; P < 0.05), increased protein synthesis (37%-45%; P < 0.01), and improved protein balance (P < 0.01) compared to the fasting state. CONCLUSIONS: Following the initial infliximab infusion in children with CD, proteolysis and protein synthesis were significantly reduced in the fasting and parenterally fed states. Supplementation with parenteral nutrition resulted in significant improvements in protein metabolism compared to the fasting state both before and after infliximab therapy.