Table of Contents
- Biobehavioral Cancer Prevention and Control Training Program
- Patient-Centered Outcomes in Infants and Young Children with Cleft Lip and Palate (COO)
- Patient-Reported Outcomes Measurement Information System
-- Phase II: HIV Clinical Outcomes
- Lymphatic Malformation Function Assessment
- Symptoms and Signs, Behaviors/Events, and Quality of Life in Girls with Central Precocious Puberty (CPP) - Self and Parent Report
- CF-ePRO: Electronic Self- and Parent-Reported Patient Outcomes in Cystic Fibrosis - Talaria
- Adherence Measurement across Multiple Chronic Conditions
- Qualitative Interviews and Focus Groups with Burn Survivors: Phase I Development of A Novel Validated Patient Reported Outcome Measurement for Persons with Burn Injury
Biobehavioral Prevention and Control Training ProgramMission
The mission of the Biobehavioral Cancer Prevention and Control Training Program is to train public health researchers, health services researchers, and health policy analysts in biobehavioral and outcomes research, communications, and practice outcomes as applied to cancer prevention and control.Funder:
The National Cancer InstituteProject Period:
5 year training grantWeb Site: Program Coordinator
Patient-Centered Outcomes in Infants and Young Children with Cleft Lip and Palate (COO)Background & Significance:
Clefts of the lip with or without cleft palate (CL± P) represent one of the most common birth defects with an estimated worldwide prevalence of 1 in 700 live births leaving infants to experience significant challenges with feeding, nutrition, hearing, speech production, breathing and sleeping. Despite published guidelines, a great deal of unwarranted variation in healthcare for infants with clefts exists, nationally and worldwide, due at least in part to a shortage of validated outcome measures for assessing interventions in this population. This project will design an endpoint strategy and develop and validate a Cleft Lip and Palate Observer-Reported Outcomes instrument (COO) for evaluation of interventions in clinical trials and reduction of unwarranted treatment variation.Study Aim(s):
- Develop an endpoint strategy, including parent-reported measures for evaluation of interventions in clinical trials.
- Evaluate measurement model, reliability, and validity of parent measures in relation to existing clinical outcome assessments.
- Evaluate ability of parent measures to detect improvements associated with primary cleft repair.
- Finalize endpoint strategy and implement parent measures with guidance from field leaders.
National Institute of Dental and Craniofacial ResearchPrincipal Investigators:
Todd C. Edwards, PhD & Carrie L. Heike, MD, MSProject Period:
5 year grantE-mail the Project Manager
E-mail the Research Study Coordinator
Patient-Reported Outcomes Measurement Information System -- Phase II: HIV Clinical OutcomesBackground & Significance:
Using a nationally distributed cohort of HIV-infected individuals in routine clinical care, this project extends PROMIS validation work to those with HIV, an important, understudied patient population that includes large numbers of underserved, African-Americans, minorities, women, and persons with disabilities. In addition to key validation work for domains most useful for clinical care, this proposal addresses technical problems and barriers of routine PRO assessment in clinical care, and advances the use of PRO assessments for clinical care and applied research in clinical settings.Study Aim(s):
The goal of this project is to improve medical care processes and patient outcomes in clinical practice settings using routine collection of patient-reported outcomes (PROs) administered by computerized adaptive tests (CATs).
E-mail the Project Manager
Lymphatic Malformation Function AssessmentBackground & Significance:
Lymphatic malformations (LM) are masses of abnormal lymphatic channels that most commonly involve the head and neck, occurring 1/2- 4,000 live births. Development of an evidence base for LM can be aided through standardized questions directed to the patient and/or their caregiver that measure how treatment affects daily functions, such as breathing, speaking and eating. There is no other way to measure the impact these malformations have on function before or after treatment, as the only current measure of treatment success is lesion ablation. This is inadequate, as the majority of LMs cause continued morbidity and are never completely "cured." Getting the patient or caregiver perspective on what is important is a critical adjunct to clinical assessment.Study Aim(s):
The purpose of this project is the establishment of a standardized patient, parent, and clinician-reported assessment system for use in assessing treatment outcomes and for decision-making regarding treatment.E-mail the Project Manager
Symptoms and Signs, Behaviors/Events, and Quality of Life in Girls with Central Precocious Puberty (CPP) - Self and Parent ReportBackground & Significance:
Central Precocious Puberty (CPP) is characterized by early pubertal changes, acceleration of growth velocity and rapid bone maturation that often result in reduced adult height. It occurs much more commonly in girls, in whom it is usually idiopathic. The main reasons for treating CPP are to prevent compromised adult height and to avoid psychosocial or behavioral problems. The complex complement of characteristics that are associated with CPP requires a measure that cuts across all aspects of life. This is a protocol for the development of self- and parent-report instruments for assessing sign/symptoms, behaviors, and quality of life (QoL) of girls ages 8 to 13 years old with Central Precocious Puberty.Study Aim(s):
Phase 1: Concept Elicitation and Item Development --
1. Develop youth self-report of symptoms, behaviors/events, and quality of life specific to Central Precocious Puberty (CPP) by conducting qualitative interviews with girls ages 11-13 years.
2. Develop parent-report of observable signs and behaviors/events specific to CPP by conducting focus groups and interviews with parents of girls ages 6-10 years.
3. Assess item acceptability, clarity, and readability of the youth and parent instruments via cognitive interviews with youth and parents.
Phase 2: Assessment of Cross-Sectional Measurement Properties --
4. Evaluate the cross-sectional measurement properties of the new CPP Instruments in youth and parents, including measurement model, internal consistency, test-retest reliability, and construct validity.
CF-ePRO: Electronic Self- and Parent-Reported Patient Outcomes in Cystic Fibrosis - TalariaBackground & Significance:
Studies of rare diseases are often limited to small sample sizes, which can be under-powered to detect significant treatment effects unless repeated measurements are taken. More frequent assessments yield more statistical power, but increasing assessment frequency presents logistical challenges, particularly with patient reported outcomes (PROs). We're examining the reliability and acceptability of prompting web-based PRO assessments for Cystic Fibrosis (CF).Study Aim(s):
The aim of this study is to conduct Phase I feasibility and usability work that will provide critical preliminary data for further development of an electronic parent-report system. In-depth qualitative interviews will be conducted with primary caregivers of 30 children with CF=6 years, in order to elicit the respiratory and gastrointestinal signs caregivers of infants and young children with CF can observe (see, hear, touch) when their child is sick, as well as on a day-to-day basis.E-mail the PI's
Adherence Measurement across Multiple Chronic ConditionsBackground & Significance:
Adherence to treatment is crucial for many chronic disease patients. Detection of inadequate adherence and suboptimal adherence patterns prior to the development of poor outcomes remains one of the most important and elusive goals in the treatment of many patients with chronic diseases. There is a key unmet need for adherence assessments that can be successfully integrated into clinical care settings.Study Aim(s):
Aim 1 and 2. Develop a medication adherence behavior item pool and a medication adherence barrier item pool for multiple chronic disease patient populations.
Aim 3. Evaluate validity of adherence measures and develop a cross-walk approach that facilitates direct comparisons of adherence estimates from different time-frames and follow-up periods.
Aim 4. Integrate the adherence behavior and adherence barrier item pools into clinical care settings.
Qualitative Interviews and Focus Groups with Burn Survivors: Phase I Development of A Novel Validated Patient Reported Outcome Measurement for Persons with Burn InjuryBackground & Significance:
Advances in critical care and surgical management have significantly improved survival following burn injury. Survival alone therefore is no longer a sufficient outcome measure following burn injury. Accordingly, there is a shift in emphasis in burn care towards optimizing the quality of life of those who survive injury. Currently, there are few patient reported outcome instruments (PROs) that have been validated in the burn population, and the existing measures have significant limitations that limit their utility.Study Aim(s):
The purpose of this study is to use utilize qualitative research methodology to perform Phase I development of a new burn-specific PRO. We will conduct qualitative interviews and focus groups with burn survivors, researchers and care providers, to generate a bank of concepts that are relevant and important to burn survivors. Second, the concept bank can be used to generate items for a new burn specific PRO which we will plan to do with subsequent funding. This PRO will be critical to the evaluation of novel treatments and therapies as well as in the evaluation of surgical decision making.E-mail the Project Manager