Rigorous and consistent evaluation of diagnostic tests in children: another unmet need

It is time for the pediatric community, along with the FDA and the device industry, to renew efforts to better provide appropriately evaluated devices and diagnostic tests for children. This will require seriously considering incentives and/or funding for pediatric trials, improving insurance coverage to reimburse for device use, and creative approaches to the evaluation process in children. In the meantime, the FDA should work to better communicate the device and diagnostic test regulatory process to the public and physicians, and make clear to pediatricians to what extent specific diagnostic tests and devices have and have not been evaluated in children.This work was supported in part by the Indiana Clinical and Translational Sciences Institute funded, and in part by Award Number UL1TR002529 from the National Institutes of Health, National Center for Advancing Translational Sciences, and Clinical and Translational Sciences Award

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. [J Med Internet Res 2016;18(12):e333

Project development teams: a novel mechanism for accelerating translational research

The trend in conducting successful biomedical research is shifting from individual academic labs to coordinated collaborative research teams. Teams of experienced investigators with a wide variety of expertise are now critical for developing and maintaining a successful, productive research program. However, assembling a team whose members have the right expertise requires a great deal of time and many resources. To assist investigators seeking such resources, the Indiana Clinical and Translational Sciences Institute (Indiana CTSI) created the Project Development Teams (PDTs) program to support translational research on and across the Indiana University-Purdue University Indianapolis, Indiana University, Purdue University, and University of Notre Dame campuses. PDTs are multidisciplinary committees of seasoned researchers who assist investigators, at any stage of research, in transforming ideas/hypotheses into well-designed translational research projects. The teams help investigators capitalize on Indiana CTSI resources by providing investigators with, as needed, mentoring and career development; protocol development; pilot funding; institutional review board, regulatory, and/or nursing support; intellectual property support; access to institutional technology; and assistance with biostatistics, bioethics, recruiting participants, data mining, engaging community health, and collaborating with other investigators.Indiana CTSI leaders have analyzed metrics, collected since the inception of the PDT program in 2008 from both investigators and team members, and found evidence strongly suggesting that the highly responsive teams have become an important one-stop venue for facilitating productive interactions between basic and clinical scientists across four campuses, have aided in advancing the careers of junior faculty, and have helped investigators successfully obtain external funds

Acute Effects of Enteral Nutrition on Protein Turnover in Adolescents with Crohn Disease

ABSTRACT: Adults with inactive Crohn disease have been shown to have normal rates of protein turnover when compared with healthy adults. It is not known whether this is true for adolescents with inactive Crohn disease, when rate of protein synthesis must be greater than that of breakdown for normal development. The objective of this study was to determine whether enteral nutrition acutely suppresses proteolysis and increases protein synthesis in adolescents with inactive Crohn disease. Six adolescents (five males/one female; mean age, 15.8 Ϯ 1.9 y; range, 13.2-17.6 y; mean bone age, 14.6 Ϯ 1.8 y; range, 12.5-17 y) participated. Leucine (Leu) and phenylalanine (Phe) kinetics were measured using stable isotopes under fasted and fed conditions during a single study visit. In response to enteral nutrition, the endogenous rates of appearance (R a ) of Leu and Phe (reflecting proteolysis) decreased significantly by 40%. The percentages of splanchnic uptake of Leu and Phe were 35 Ϯ 10% and 13 Ϯ 12%, respectively. Under fed conditions, utilization of Phe for protein synthesis increased significantly. We conclude that in clinically stable adolescents with Crohn disease, enteral nutrition promotes anabolism by suppressing proteolysis and increasing protein synthesis. Rates of suppression of proteolysis were similar to those reported previously in normal children

Utilizing a reviewer database to facilitate integration of an investigator-focused translational research and career development program across the state of Indiana

OBJECTIVES/SPECIFIC AIMS: The Indiana CTSI is investigating innovative approaches to integrate resources that will enrich scientific investigators. Our goals are to enhance the availability and communication among CTSI resources, for example internal funding, and to expand existing mentorship. METHODS/STUDY POPULATION: Developed a reviewer database that serves to streamline reviewer identification, decrease reviewer fatigue, and promote collaboration among disciplines. We started with a pool of NIH-funded investigators from across the Indiana CTSI core institutions and merged this list with previous CTSI reviewers and internal funding awardees. To expand this list, names and expertise from new faculty hires were added. RESULTS/ANTICIPATED RESULTS: Though this tool is relatively new, we have already observed an increase in junior faculty awareness and engagement with the CTSI. This database allows for increased opportunities of junior faculty to serve as reviewers and to refine grant writing skills and provides a platform for networking and collaborating across disciplines. It also allows for increased integration of programs with a shared reviewer database and promotes grant review standardization. DISCUSSION/SIGNIFICANCE OF IMPACT: Our database utilization seeks to decrease the time for junior faculty to obtain their first extramural grant, to enhance promotion and tenure packages, strengthen integration among CTSI programs, increase interactions between clinical and basic science investigators, and promote team science

Pharmacotherapy and Pregnancy: Highlights from the Third International Conference for Individualized Pharmacotherapy in Pregnancy

To address provider struggles to provide evidence-based, rational drug therapy to pregnant women, this third Conference was convened to highlight the current progress and research in the field. Speakers from academic centers, industry, and governmental institutions spoke about: the Food and Drug Administration’s role in pregnancy pharmacology and the new labeling initiative; drug registries in pregnancy; the pharmacist’s role in medication use in pregnancy; therapeutic areas such as preterm labor, gestational diabetes, nausea and vomiting in pregnancy, and hypertension; breast-feeding and medications; ethical challenges for consent in pregnancy drug studies; the potential for cord blood banks; and concerns about the fetus when studying drugs in pregnancy. The Conference highlighted several areas of collaboration within the current Obstetrics Pharmacology Research Units Network and hoped to educate providers, researchers, and agencies with the common goal to improve the ability to safely and effectively use individualized pharmacotherapy in pregnancy

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 welldesigned 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