Rosen R.C.,New England Research Institutes, Inc. |
Allen K.R.,New England Research Institutes, Inc. |
Ni X.,Lilly United States LLC |
Araujo A.B.,New England Research Institutes, Inc.
European Urology | Year: 2011
Background: Despite widespread adoption of the six-item erectile function (EF) domain of the International Index of Erectile Function (IIEF) as a clinical trial end point, there are currently no objective data on what constitutes a minimal clinically important difference (MCID) in the EF domain. Objective: Estimate the MCID for the IIEF EF domain. Design, setting, and participants: Anchor-based MCIDs were estimated using data from 17 randomized, double-blind, placebo-controlled, parallel-group clinical trials of the phosphodiesterase type 5 inhibitor (PDE5-I) tadalafil for 3345 patients treated for 12 wk. Measurements: The anchor for the MCID is the minimal improvement measure calculated using change from baseline to 12 wk on IIEF question 7: "Over the past 4 weeks, when you attempted sexual intercourse how often was it satisfactory for you?" MCIDs were developed using analysis of variance (ANOVA)- and receiver operating characteristic (ROC)-based methods in a subset of studies (n = 11) by comparing patients with and without minimal improvement (n = 863). MCIDs were validated in the remaining six studies (n = 377). Results and limitations: The ROC-based MCID for the EF domain was 4, with estimated sensitivity and specificity of 0.74 and 0.73, respectively. MCIDs varied significantly (p < 0.0001) according to baseline ED severity (mild: 2; moderate: 5; severe: 7). MCIDs consistently distinguished between patients in the validation sample classified as no change or minimally improved overall and by geographic region, ED etiology, and age group. MCIDs did not differ by age group, geographic region, or ED etiology. Current analyses were based on 17 clinical trials of tadalafil. Results need to be replicated in studies using other PDE5-Is or in nonpharmacologic intervention studies. Conclusions: The contextualization of treatment-related changes in terms of clinically relevant improvement is essential to understanding treatment efficacy, to interpreting results across studies, and to managing patients effectively. This analysis provides, for the first time, anchor-based estimates of MCIDs in the EF domain score of the IIEF. © 2011 European Association of Urology.
Chiu G.R.,New England Research Institutes, Inc.
Violence and victims | Year: 2013
Abuse is associated with a wide variety of health problems, yet comprehensive population-based data are scant. Existing literature focuses on a single type of abuse, population, or lifestage. Using a racially/ethnically diverse community-based sample, we document the prevalence of physical, emotional, and sexual abuse by lifestage and gender, assess variation in abuse by sociodemographics; establish overlap of abuses; and examine childhood abuse relationships with abuse in adulthood. Prevalence of abuse ranges from 15% to 27%; women report more adulthood emotional abuse and lifetime sexual abuse than men; reports of abuse can vary by race/ethnicity and poverty status, particularly in women; there is overlap between types of abuse; and a history of childhood abuse is associated with a greater risk of abuse as an adult.
Trachtenberg F.L.,New England Research Institutes, Inc.
Quality of life research : an international journal of quality of life aspects of treatment, care and rehabilitation | Year: 2014
Thalassemia, a chronic blood disease, necessitates life-long adherence to blood transfusions and chelation therapy to reduce iron overload. We examine stability of health-related quality of life (HRQOL) in thalassemia and adherence to chelation therapy over time, especially after changes in chelator choice. Thalassemia Longitudinal Cohort participants in the USA, UK, and Canada completed the SF-36v2 (ages 14+) and the PF-28 CHQ (parents of children <14 years). Chelation adherence was defined as self-reported percent of doses administered in the last 4 weeks. Two hundred and fifty-eight adults/adolescents (mean 29.7 years) and 133 children (mean 8.5 years) completed a mean of 2.8-years follow-up. Children made few chelator changes, whereas a mean of 2.2 changes was observed among the 37% of adults/adolescents who made chelator changes, mainly due to patient preference or medical necessity. Physical HRQOL improved among those with lower iron burden (better health status) at baseline who made a single change in chelator, but declined among participants with multiple changes and/or high iron burden (worse health status). Mental health improved among participants with lower iron burden, but iron overload was negatively associated with social functioning. Adherence did not significantly change over follow-up except for an increase after a change from deferoxamine (DFO) infusion to oral deferasirox (p = 0.03). Predictors of lower adherence for adults/adolescents at follow-up included side effects, smoking, younger age, problems preparing DFO, increased number of days per week DFO prescribed, and lower physical quality of life . Strategies to balance medical needs with family, work, and personal life may assist in adherence.
Agency: Department of Health and Human Services | Branch: National Institutes of Health | Program: SBIR | Phase: Phase II | Award Amount: 1.30M | Year: 2015
DESCRIPTION provided by applicant andquot The PHS Syphilis Study Guatemala Willowbrook radiation experiments andquot Despite decades of work to improve the safety of research subjects these words bring to mind images of fear and the mistreatment of potential research participants including children Excellence in research is essential to our health and quality of life yet while many potential participants recognize the need for clinical studies they avoid participating Recruitment for pediatric clinical trials presents unique challenges including a lak of information for parents faced with a decision about whether to allow a child to participate Despite best efforts of the National Institutes of Health to help improve inform and standardize clinical trial recruitment these efforts are targeted to parents who are asked to involve their chid in a clinical trial Misperceptions fears and myths about the well known but not representative mistakes misconduct or abuse from the earliest days of clinical research persist regardless of these efforts With demonstrated need for information about clinical research the importance of clinical research to reduce social justice and disparities and the vast gap between treatments used with children but never tested in children no national broadcast program yet exists which has tackled the complex and important issue of clinical studies particularly in pediatric populations The goal of this Phase Il SBIR application is to develop the one hour original narrative documentary style film entitled No More Hand Me Downs Clinical Research and Children To accomplish this goal we will complete four major Aims Aim To meet with clinical experts and identify families from related trials for the complete film Aim Develop the minute broadcast film Aim Conduct an evaluation of the film in preparation for national broadcast Aim Conduct national broadcast and outreach operations in collaboration with WebMD PUBLIC HEALTH RELEVANCE Despite decades of work to improve the safety of research subjects fear about mistreatment of potential research participants remains a concern for many Mistrust as a result of early well publicized abuses continues to outweigh the many rules and requirements now in place to ensure high ethical standards These fears are particularly entrenched with minority and underserved populations such as children Excellence in research is essential to our health and quality of life yet while many potential participants recognize the need for clinical studies they avoid participating
Agency: Department of Health and Human Services | Branch: | Program: SBIR | Phase: Phase I | Award Amount: 150.00K | Year: 2014
PROJECT SUMMARY/ABSTRACT Type 1 diabetes (T1DM) is the most common chronic illness in children, affecting 1 in 400 with evidence that its prevalence is increasing worldwide. Parents of children with T1DM are responsible for a labor-intensive and complicated daily regimen that has been described as an overwhelming experience, requiring constant vigilance. The prevalence of anxiety symptoms for parents of children with T1DM range from 21-59%; depressive symptoms from 10-74%; psychological distress from 29-33%; and symptoms of Post Traumatic Stress Disorder (PTSD) from 19-24%. Parental psychological distress has negative health implications for the parent, the overall functioning of the family, the psychological adjustment of their child with T1DM, and diabetes management. Combined with parental stress, children (as they reach adolescence) have an increasing need for autonomy in T1DM management, creating additional concerns about good metabolic control. Most health care visits are thus focused on the adolesce
Agency: Department of Health and Human Services | Branch: National Institutes of Health | Program: SBIR | Phase: Phase II | Award Amount: 514.33K | Year: 2016
DESCRIPTION provided by applicant Type diabetes T D is one of the most common chronic illnesses in children affecting in with evidence that its prevalence is increasing worldwide The prevalence of T D in adolescents is approximately percent White percent Hispanic and percent Black Parents of children with T D are responsible for a labor intensive and complicated daily regimen that has been described as an overwhelming experience requiring constant vigilance Intensive management of T D requires frequent blood glucose monitoring multiple insulin injections or use of an insulin pump frequent alterations in insulin dose to match changing diet and activity patterns and regular visits to health care providers This attention to their childandapos s health manifests in elevated rates of parental perception of stress and increased risk for depression and anxiety The prevalence of anxiety symptoms for parents of children with T D range from percent depressive symptoms from percent psychological distress from percent and posttraumatic stress symptoms from percent Parental psychological distress has negative health implications for the parent the overall functioning of the family the psychological adjustment of their child with T D diabetes management and child metabolic control The goal of this application is to complete the psycho educational web based program for parents of adolescents with T D Type Teamwork which will help to decrease parental perceived stress and distress promote parental adjustment to the developmental transitions in adolescence support adolescent autonomy and transfer of diabetes responsibility from parent to adolescent decrease family conflict and maintain metabolic control during adolescence We propose to complete two major Aims in Phase II Specific Aim Develop the Type Teamwork program for use on computer tablet or smartphone based on the activities completed in Phase I Six content themes have been identified as important to parents and providers Feasibility assessment has provided support of these content themes as well as substantive recommendations to ensure content and interactivity is engaging and meets the needs of parents of children with T D as well as health care providers We will use an iterative process of development and evaluation collaborating with our technology team parent advisors and clinical consultants to ensure development of a quality product and to submit a peer reviewed manuscript for publication of the results Specific Aim Evaluate the Type Teamwork program through a modest randomized clinical trial We will determine the effect of the program on parent psychosocial outcomes stress depression and anxiety adolescent autonomy parent adolescent responsibility for T D management family conflict and adolescent metabolic control Results will be presented at diabetes meetings published in top tier journals to provide clinical and scientific evidence of the newly developed program and widely disseminated per the commercialization and marketing plan PUBLIC HEALTH RELEVANCE Type Diabetes T DM is a common chronic illness in children which presents difficult and often stressful management concerns for parents As children approach adolescence this burden increases with the desire for independence and self management No tool exists that addresses in a user friendly easy to access and socio culturally appropriate way the psychosocial needs of parents as they move through this transition This program targets the parents to help them at the very point where this transition i occurring
Agency: Department of Health and Human Services | Branch: | Program: SBIR | Phase: Phase I | Award Amount: 138.89K | Year: 2011
DESCRIPTION (provided by applicant): Developmental delays are common in early childhood, affecting at least 10 percent of children and are markers for later developmental conditions. It is well established that the tremendous adaptability of the brain in the first three years of life means that early treatment of delays leads to improved outcomes, whereas later intervention is less effective. In order to provide treatment to improve children's outcomes, early identification of delays and sensory impairmentsduring the first three years of life are critical. There is a tremendous need for and interest in early identification of children at risk for development problems, as is evidenced by the vast attention to this topic by the leading organizations for pediatric health (AAP recommendations, NICHD's Early Identification and Interventions for Infants and Young Children at Risk for Mental Retardation, and AHRQ Special Emphasis Notice for research on diagnostic errors). A consistent concern is that there is theneed for screening strategies in diverse audiences, and for translating effective, evidence-based screening approaches for use in primary care practices. There are a number of different screening tools available, but no consensus on which screens are bestused in practice. However, screening is being increasingly adopted by practitioners and there is a high level of discomfort translating the results to parents. Thus, there is a need for educational materials that brings together information to assist primary care providers in choosing the best tools; provides guidance on implementation of screening in practice; and communicates the purpose and results of these screens in practice. This resource will inform providers about the importance and challenges of conducting developmental screening; assist providers in understanding and communicating those results to parents, and identifying and acknowledging cultural differences in screening strategies and delivery of results. The specific aims are: Aim 1: To develop a clinical content outline for a CME/CE program that will focus on two major areas: 1) Understanding and appropriate implementation of development screeners in primary care. 2) Improving communication of the purpose and results of developmental screeningtests with parents. Aim 2: Develop clinical content/interactivity for a prototype module of the Web-based CME/CE course. Aim 3: Develop a unique approach to learning on the web through video-based storytelling vignettes. This online CME program will speakthrough real world stories of pediatricians and parents who have dealt with such issues (in their own words). The course will illustrate techniques for communicating results to parents and seeking appropriate next steps for care. Because capturing highquality video is a core cost component of such a program, we also propose to use the video to create a web site for parents. Aim 4: Conduct a set of qualitative interviews to inform development and evaluate the course. PUBLIC HEALTH RELEVANCE: In this Phase I program we will develop a prototype of a CME/CE Web-based course to assist health care providers understand the purpose and approach to developmental screening, and more effectively communicate results with parents. This program will bring together existing research and provide an easily accessible source for pediatricians on screening. By providing innovative content via an established medium, we aim, through this product, to reach primary care providers facing this important issue to improve outcomes through early identification and intervention.
Agency: Department of Health and Human Services | Branch: | Program: SBIR | Phase: Phase II | Award Amount: 629.67K | Year: 2010
DESCRIPTION (provided by applicant): Widely publicized cases such as Terri Schiavo have raised awareness of the importance of advance care planning. However, while promoted through federal law and regulation, emphasis is placed on the completion of an advance directive (AD) document rather than on the informed decision making and communication essential to ensuring that end of life care preferences are honored. In fact, there is little evidence that having an AD alone results in improved or preferred care at the end of life. Consequently, the advance directive (or living will) as a public policy instrument has been called a failed social policy. More recently, attention has been redirected to the process of advance care planning. In response, we propose an innovative interactive decision aid to help individuals not only to make informed decisions about preferred treatment at end of life but also to communicate these preferences and decisions to their health care providers and families. The program will use video to provide visual depiction of several advance stage health states and options for end of life care treatment and to model effective communication with health care providers and family about preferences for care. Template documents for completing an AD and health care proxy will be provided for completion immediately or at a later date. The program is presented on a tablet-PC, with the capability to be web-based allowing the inclusion of these documents in an electronic medical record. No other available program offers all of these features on a portable platform to maximize and facilitate use across a range of settings The Specific Aims of Phase I were completed successfully. The Specific Aims of Phase II are to: 1) produce the final two dramatic videos regarding patient communication with family and a health care proxy; 2) produce digital videos for additional health states and potential treatment options; 3) finalize web design and complete programming of text screens and integration of video and audio narration; 4) evaluation of the program's effectiveness; and 5) finalization and implementation of the commercialization plan. PUBLIC HEALTH RELEVANCE: Advance care planning is more than signing a document. It is a process. This interactive decision aide will help individuals not only make informed decisions about preferred treatment at end of life but also to communicate these preferences and decisions to their health care providers and families. The program will use video to present several advance stage health states and options for end of life care treatment and to model communication with health care providers and family about preferences for care.
Agency: Department of Health and Human Services | Branch: | Program: SBIR | Phase: Phase I | Award Amount: 99.80K | Year: 2010
Agency: Department of Health and Human Services | Branch: | Program: SBIR | Phase: Phase II | Award Amount: 982.49K | Year: 2012
Screening and recruitment of children into multi-center clinical trials poses unique challenges not seen in the adult population. Screening tools, recruitment strategies, registries to provide baseline data on which to base future clinical trials and waysto document follow-up are needed. Validated materials and tools applicable to diverse populations and for use in community practices, which often do not have the resources of larger hospitals or academic institutions, are particularly relevant. These toolsshould apply to a wide range of pediatric heart, lung, blood, and sleep disorders studies and would be developed to improve clinical trial performance. The ultimate goal is to make trial recruitment and management more efficient and effective via tools orstrategies offered to the public as an open-source product. Tools are not limited to, but can include: Interactive gaming strategies for older children and adolescents that have the potential to stimulate interest in scientific research areas related to heart, lung, blood and sleep disorders. This is an innovative and proven way to engage users (particularly youth) and deliver essential health messages, such as the importance of clinical research in improving health, what is involved when one joinsa study, and to highlight the role of National Heart, Lung, and Blood Institute (NHLBI) in biomedical research. Infrastructure development for a registry in congenital heart disease such as case report forms, development of a web-based portal, and needs assessment for sustainable operation (manpower, maintenance, IT support, etc.). Activities for back end operations could include assessment of existing information systems, development of software to facilitate interoperability of current systems, development of software that can extract data from current electronic medical records and/or electronic monitoring systems, and development of a flexible database platform that can draw data from varied sources. Reminder tools for clinical trial participants,educational resources (pamphlets, public websites), novel and improved consent tools (forms, videos, web-based), and clinical trial costs assessment tools that can be easily used by investigators or sponsors. Approaches to facilitating adoption of evidence-based guidelines. Phase I research focused on development of a new technology or strategy, to provide initial testing of the prototype and demonstrate how it will improve pediatric trial recruitment or trial management. Phase II studies will further refine the technology or strategy and test its effectiveness for incorporation into the clinical research setting.