Associations of Mental Health Measures and Retention in a Community-Based Perinatal Care Recovery Support Program for Women of Childbearing Age With Substance Use Disorder.

Objective: This research was undertaken to evaluate factors related to program retention among participants in a community-based peer recovery program for women of childbearing age with a history of substance use disorder. Methods: In all, 184 women of childbearing age with a history of substance use disorder were enrolled in a community-based peer recovery program. Half of the participants were pregnant or postpartum. The outcome of interest was retention in the program as measured at 2 and 6 months. Participants were paired with a peer recovery coach (PRC). PRCs were women with a personal history of substance use disorder who assisted with healthcare system navigation, facilitated access to local resources, and provided advice and emotional support. All PRCs were also licensed perinatal community health workers. Independent variables included gestational status, depression, anxiety, type and frequency of substance use, childhood trauma, abuse, readiness for treatment, and attachment patterns. Results: Anxiety was found to be a key factor associated with retention. Moderate anxiety was associated with higher rates of retention compared to normal to mild anxiety. Severe anxiety was associated with lower rates of retention compared to normal to mild anxiety. Attrition was highest in the first 2 months. Conclusions: Early integration with mental health services to address severe anxiety symptoms could potentially improve retention in substance use disorder recovery programs, thereby improving outcomes. More research is needed regarding severe anxiety and care-avoidant behaviors, particularly among women of childbearing age.

Recommendations for the design of therapeutic trials for neonatal seizures.

Although seizures have a higher incidence in neonates than any other age group and are associated with significant mortality and neurodevelopmental disability, treatment is largely guided by physician preference and tradition, due to a lack of data from well-designed clinical trials. There is increasing interest in conducting trials of novel drugs to treat neonatal seizures, but the unique characteristics of this disorder and patient population require special consideration with regard to trial design. The Critical Path Institute formed a global working group of experts and key stakeholders from academia, the pharmaceutical industry, regulatory agencies, neonatal nurse associations, and patient advocacy groups to develop consensus recommendations for design of clinical trials to treat neonatal seizures. The broad expertise and perspectives of this group were invaluable in developing recommendations addressing: (1) use of neonate-specific adaptive trial designs, (2) inclusion/exclusion criteria, (3) stratification and randomization, (4) statistical analysis, (5) safety monitoring, and (6) definitions of important outcomes. The guidelines are based on available literature and expert consensus, pharmacokinetic analyses, ethical considerations, and parental concerns. These recommendations will ultimately facilitate development of a Master Protocol and design of efficient and successful drug trials to improve the treatment and outcome for this highly vulnerable population.

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.

The Concordance of Electronic Health Record Diagnoses and Substance use Self-Reports Among Reproductive Aged Women Enrolled in a Community-Based Addiction Reduction Program.

Substance use disorders among reproductive aged women are a major public health issue. There is little work investigating the validity and reliability of electronic health record (EHR) data for measuring substance use in this population. This study examined the concordance of self-reported substance use with clinical diagnoses of substance use, substance abuse and substance use disorder in EHR data. Reproductive age women enrolled in the Community-Based Addiction Reduction (CARE) program were interviewed by peer recovery coaches (PRC) at enrollment. That survey data was linked with EHR data (n = 102). Concordance between self-reported substance use and clinical diagnoses in the EHR was examined for opioids, cannabis/THC, and cocaine. Cohen's kappa, sensitivity, and specificity were calculated. The survey captured a higher number of women who use substances compared to the EHR. The concordance of self-report with EHR diagnosis varied by substance and was higher for opioids (17.6%) relative to cannabis/THC (8.8%), and cocaine (3.0%). Additionally, opioids had higher sensitivity (46.2%) and lower specificity (76.2%) relative to cannabis/THC and cocaine. Survey data collected by PRCs captured more substance use than EHRs, suggesting that EHRs underestimate substance use prevalence. The higher sensitivity and lower specificity of opioids was due to a larger number of women who had a diagnosis of opioid use in the EHR who did not self-report opioid use in the self-report survey relative to cannabis/THC and cocaine. Opioid self-report and diagnosis may be influenced by research setting, question wording, or receipt of medication for opioid use disorder.

Distinguishing between translational science and translational research in CTSA pilot studies: A collaborative project across 12 CTSA hubs.

Introduction: The institutions (i.e., hubs) making up the National Institutes of Health (NIH)-funded network of Clinical and Translational Science Awards (CTSAs) share a mission to turn observations into interventions to improve public health. Recently, the focus of the CTSAs has turned increasingly from translational research (TR) to translational science (TS). The current NIH Funding Opportunity Announcement (PAR-21-293) for CTSAs stipulates that pilot studies funded through the CTSAs must be "focused on understanding a scientific or operational principle underlying a step of the translational process with the goal of developing generalizable solutions to accelerate translational research." This new directive places Pilot Program administrators in the position of arbiters with the task of distinguishing between TR and TS projects. The purpose of this study was to explore the utility of a set of TS principles set forth by NCATS for distinguishing between TR and TS. Methods: Twelve CTSA hubs collaborated to generate a list of Translational Science Principles questions. Twenty-nine Pilot Program administrators used these questions to evaluate 26 CTSA-funded pilot studies. Results: Factor analysis yielded three factors: Generalizability/Efficiency, Disruptive Innovation, and Team Science. The Generalizability/Efficiency factor explained the largest amount of variance in the questions and was significantly able to distinguish between projects that were verified as TS or TR (t = 6.92, p < .001) by an expert panel. Conclusions: The seven questions in this factor may be useful for informing deliberations regarding whether a study addresses a question that aligns with NCATS' vision of TS.

Evaluation of adequacy of protein and energy.

Growth assessment is the most common measure of nutritional adequacy in pediatrics, especially when evaluating nutrition of preterm neonates. The American Academy of Pediatrics defines postnatal nutrient intake to promote growth as one that "approximates the rate of growth...for a normal fetus of the same post-menstrual age." It is known that in the fetus, fat and lean body mass are accreted progressively as gestation progresses, whereas postnatal growth and observed accretion of fat and lean body mass differ. This review discusses anthropometric measures used to assess growth, biochemical markers used to monitor nutritional sufficiency, and the effect of growth trajectory in preterm infants on health outcomes later in life.

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.