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Browsing by Author "Denne, Scott C."
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Item Imatinib mesylate for plexiform neurofibromas in patients with neurofibromatosis type 1: a phase 2 trial(Elsevier, 2012-12) Robertson, Kent A.; Nalepa, Grzegorz; Yang, Feng-Chun; Bowers, Daniel C.; Ho, Chang Y.; Hutchins, Gary D.; Croop, James M.; Vik, Terry A.; Denne, Scott C.; Parada, Luis F.; Hingtgen, Cynthia M.; Walsh, Laurence E.; Yu, Menggang; Pradhan, Kamnesh R.; Edwards-Brown, Mary K.; Cohen, Mervyn D.; Fletcher, James W.; Travers, Jeffrey B.; Staser, Karl W.; Lee, Melissa W.; Sherman, Marcie R.; Davis, Cynthia J.; Miller, Lucy C.; Ingram, David A.; Clapp, D. Wade; Pediatrics, School of MedicineBACKGROUND: 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.Item Improving Care for Adolescents with Substance Use Disorder: More than Screening(Springer Nature, 2021) Adams, Zachary W.; Denne, Scott C.; Pediatric Policy Council; Psychiatry, School of MedicineItem Project development teams: a novel mechanism for accelerating translational research(Wolters Kluwer, 2015-01) Sajdyk, Tammy J.; Sors, Thomas G.; Hunt, Joe D.; Murray, Mary E.; Deford, Melanie E.; Shekhar, Anantha; Denne, Scott C.; Department of Pediatrics, IU School of MedicineThe 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.Item Rigorous and consistent evaluation of diagnostic tests in children: another unmet need(Springer Nature, 2020-09-06) Caldwell, Christine S.; Denne, Scott C.; Pediatrics, School of MedicineIt 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.Item 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(JMIR Publications, 2016) DeHoff, Beth A.; Staten, Lisa K.; Rodgers, Rylin Christine; Denne, Scott C.; Department of Social and Behavioral Sciences, Richard M. Fairbanks School of Public HealthBackground: 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]Item Screening for postpartum depression: obligation and opportunity for pediatricians to improve the lives of children(Nature, 2019) Chambers, Joanna E.; Denne, Scott C.; Psychiatry, School of MedicineItem Utilizing a reviewer database to facilitate integration of an investigator-focused translational research and career development program across the state of Indiana(Cambridge University Press, 2018-06) Coffee, R. L., Jr.; Driscol, Julie; Saydyk, Tammy J.; Shekhar, Anantha; Denne, Scott C.; Hunt, Joe D.; Medicine, School of MedicineOBJECTIVES/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.