Asthma and Atopic Dermatitis Validation of PROMIS Pediatric Instruments (AAD-PEPR) is our response to the NIH RFA AR-15-014, titled: Validation of Pediatric Patient Reported Outcomes in Chronic Diseases (PEPR) Consortium (U19). Our project combines a series of research and development projects targeted at two of the most common chronic diseases affecting children: asthma and atopic dermatitis (AD, or eczema). Together these diagnoses affect almost 25% of children under age 18. Nevertheless, there are significant knowledge gaps about the impact of these diseases and their treatments on the quality of life (QOL) of these children. The Patient Reported Outcomes Measurement Information System (PROMIS) offers the unprecedented opportunity to understand QOL from the patient and caregiver (parent) perspective on a wide range of common symptoms and functional abilities across physical, mental and social health. The multidimensional effects of these chronic diseases on the child and family have not been well-quantified. Over the course of the last 12 years, PROMIS investigators have created pediatric self-report measures covering 26 domains, with 21 of those domains having instruments formatted for parent proxy reporting. While almost all of these measures have been validated, calibrated and normed in the general population, there have only been limited efforts to validate these measures in clinical samples. Our proposed work will address this gap using three existing asthma cohorts and a large cohort of pediatric patients with skin conditions with a primary focus on AD. We will examine the ability of PROMIS instruments to detect meaningful and clinically significant change in disease status and estimate minimally important differences for PROMIS measures in children with asthma and AD. Our review of AD outcomes revealed that one prominent outcome not currently addressed by PROMIS is pruritus (itch), the most frequent and bothersome symptom associated with AD. Our local patient and parent advisory committee has stressed the importance of measuring this symptom and its QOL effects. Itch can have profound effects upon sleep, mood and functioning, and is associated with psychological, financial, and social burden to patients and families. By leveraging the sample for the proposed AD validation, we propose to develop an itch item bank to complement the currently available, more generic pediatric PROMIS tools, thereby capturing the full spectrum of patient-reported outcomes (PROs) and their impact in children with chronic pruritic skin disorders. Given our experience as the PROMIS Statistical Coordinating Center for PROMIS I and the PROMIS Statistical and Technical Centers for PROMIS II, we will provide coordination and oversight for Consortium-wide data management activities, including timely submission of any new content changes or additions of PROMIS measures to the PROMIS Health Organization (PHO) for domain framework and standards review, and to the Person Centered Assessment Resource (PCAR), the entity authorized to distribute PROMIS and sustain its scientific quality and credibility.
The pediatric version of the Patient Reported Outcomes Measurement Information System (PROMIS) offers the unprecedented opportunity to understand Quality of Life from the patient and caregiver (parent) perspective on a wide range of common symptoms and functional abilities across physical, mental and social health. Our work is directed to confirm the capacity of these measures to detect meaningful and clinically significant change in disease status in asthma and pediatric skin conditions with a primary focus on Atopic Dermatitis (also known as eczema). The results of this study will enable researchers to better assess the effects of new treatments, and for clinicians to better understand the needs of their patients and the impact of the treatments that are prescribed to those patients.
| Phipatanakul, Wanda; Koutrakis, Petros; Coull, Brent A et al. (2017) The School Inner-City Asthma Intervention Study: Design, rationale, methods, and lessons learned. Contemp Clin Trials 60:14-23 |
