Initial Evaluation of the Pediatric PROMIS Health Domains In Children and Adolescents with Sickle Cell Disease (SCD).
Authors: Dampier Carlton , Thornburg Courtney , Greenway Anthea Louise , Faircloth Dana , Gross Heather , Thissen David , DeWalt Darren 
Published: October 2019

Abstract
Abstract 3829
The Patient-Reported Outcomes Measurement Information System (PROMIS) project was developed to advance the science and application of patient-reported outcomes (PRO) associated with chronic diseases, and is supported as a trans-NIH initiative as part of the NIH Roadmap for Medical Research. One main PROMIS goal is to develop a set of item banks and computerized adaptive tests (CAT) that could be used by the clinical research community for clinical trials or health outcome studies. The PROMIS pediatric project is developing self-report PRO for youths aged 8–17 years across several health domains that are important across a variety of illnesses, including physical function, pain, fatigue, emotional distress, and social function. In addition to validation in a large sample of healthy children, these measures are being validated in a variety of pediatric chronic illnesses, including sickle cell disease (SCD). We hypothesized that the pediatric PROMIS measure would be sensitive to changes in health status of children with SCD. A one-time cross-sectional study was conducted of the pediatric PROMIS item banks in children with SCD. A convenience sample of SCD patients aged 8–17 years followed at 2 large Sickle Cell programs (Emory University and Duke University) was recruited from routine clinic visits. Individuals were eligible to participate if they could speak and read English and were able to complete a computer administered survey. Children completed the pediatric PROMIS survey over the internet using a computer adapted testing (CAT) protocol on the PROMIS Assessment Center secure website. Children were supervised by the research staff to ensure that the responses were self-reported. Parents also completed a short demographics questionnaire, and medical related information was verified from medical records. A total of 236 subjects (mean age 12.49 ± 2.82, 49.8% female) participated in the study (Emory 170, Duke 66) over an 8 month period from May 2009 to January 2010. Most participants had an SS genotype (76.5%), while 16.7% had SC, 4.7% had Sickle B+thalassemia, and 1.3% had Sickle B0 thalassemia. At the time of the study 19.1% were receiving chronic transfusions and 45.5% were taking hydroxyurea. In the prior 6 months, participants reported a mean (SD) of 4.93±9.08 home-managed painful episodes; 1.21±2.91 ED managed painful episodes; and 0.72±1.71 hospitalizations. Parents reported daily analgesic usage by 35%, and 17.1% with hip or joint problems. Scores on the pain interference domain increased with increasing age (r = .175, p=.007). Correlations between age and all other domains were not significant. Physical function dexterity and mobility, depression, pain interference, tired, and low energy domain scores were reduced in children with a reported hip or joint problem compared to for those without hip or joint problems (all p<.05). The occurrence of pain at home requiring treatment in the previous 7 days was associated with declines in functioning in the following domains: depression, anxiety, pain interference, fatigue, low energy, dexterity, and mobility (all p<0.01). Similarly, the number of pain episodes managed at home in the past 6 months was positively correlated with the following domains: pain interference (r = .21, p < .002), depression (r=-.6, p=.01), tired (r=-.26, p<.001), low energy (r=.17, p=.03), and mobility (r=-.23, p=.004). Initial evaluation of the the pediatric PROMIS item banks indicate that self-reported health domains vary with overall clinical severity and frequency of recent clinical events. Current longitudinal studies with these measures at the Emory Sickle Cell Consortium are designed to determine their sensitivity to specific clinical events (hospitalized painful episodes) and related minimally significant differences. These results suggest that the PROMIS pediatric measures should be considered for use in future pediatric SCD clinical trials. Supported by NIH U01-AR052181.

Disclosures:
Dampier: Anthera Pharmaceuticals Inc: Chair DSMB; Glycomimetics Inc: Chair DSMB.