Cincinnati Children’s Hospital Medical Center


Grant Number: 1U01AR057940-01
Project Title: Enhancing PROMIS in Pediatric Pain, Rheumatology, and Rehabilitation Research
PI Name, Title and Contact Information: Esi Morgan DeWitt, MD, MSCE, Principal Investigator Assistant Professor, Division of Rheumatology, Division of Health Policy and Clinical Effectiveness, Cincinnati Children’s Hospital Medical Center
Other Key Personnel: Susmita Kashikar-Zuck, PhD, Co-Investigator
Michael Seid, PhD, Co-Investigator
Jilda Vargus-Adams, MD, MSc, Co-Investigator
C. Jeffrey Jacobson Jr, PhD, Co-Investigator
Bin Huang, PhD, Co-Investigator
Kenneth Goldschneider, MD, Co-Investigator
Carlton Dampier, MD, CPI, Co-Investigator
David Sherry, MD, Co-Investigator
Darren A. DeWalt, MD, MPH, Study Consultant
Kevin P. Weinfurt, PhD, Study Consultant
Laura Schanberg, MD, Study Consultant
Gary A. Walco, PhD, Study Consultant
Web Site: www.chop.edu

Abstract:

The Patient Reported Outcomes Measurement Information System (PROMIS) has created pediatric item banks for patient reported outcomes in several health domains (physical function, pain, fatigue, emotional distress, social role). To bring the work of the first phase of PROMIS to fruition will entail demonstrating validity and responsiveness to change in childhood chronic illness populations. The utility of PROMIS as an assessment tool in clinical trials will be increased by determination of minimal important differences (MIDs) in the context of legacy scales, clinical, and statistical benchmarks as well as from patient and provider perspectives. PROMIS has improved on existing instruments in some health domains, but not all important domains have been addressed. For example, PROMIS has created a pediatric pain interference item bank. However, other dimensions of pain, such as intensity, quality, and behavior are also important clinical research outcomes that are not currently included in pediatric PROMIS. Understanding therapeutic effects on these other aspects of pain are important to fully evaluate pain outcomes for children.

The goal of this project are

  1. validate and test longitudinally for responsiveness the current PROMIS pediatrics item banks in pediatric populations with musculoskeletal disorders;
  2. to create, test, and validate new pediatric pain domain items according to PROMIS item bank development procedures in patient populations with conditions that may experience chronic or recurrent pain. The enhancements we propose will help PROMIS better meet the needs of pediatrics researchers and patients. We will accomplish these goals by pursuing two specific aims:

AIM 1: To evaluate the validity and responsiveness of the PROMIS pediatric item banks with longitudinal assessments in children with juvenile idiopathic arthritis, cerebral palsy, and chronic musculoskeletal pain. In each study population content validity will be assessed with individual interviews, and construct validity analyzed with co-administration of legacy scales and by test of responsiveness to change. Longitudinal assessments will be performed to capture clinical transition points expected to result in health status changes measurable with patient reported outcomes. The end product of this aim will be the determination of MIDs to enhance the interpretability and acceptance of the PROMIS measures for clinical research.

AIM 2: To enhance PROMIS pediatric pain assessment with new item bank creation and testing. In recognition of the multiple facets of the pain experience, PROMIS pediatric pain items and banks will be developed in the areas of pain intensity, pain quality, and pain behavior. The item creation will follow the established PROMIS qualitative item review process. Content validity will be established through key informant interviews with patients with chronic and recurrent painful conditions including sickle cell disease, musculoskeletal pain syndromes, and juvenile idiopathic arthritis. Construct validity will be assessed by correlation with legacy scales, validated measures currently administered in clinical research studies.

Patient populations/disease groups:

AIM 1a: To evaluate the validity and responsiveness of the PROMIS pediatric item banks with longitudinal assessments in children with Juvenile Idiopathic Arthritis (JIA).

Study Population: Children aged 8 to 18 years with a physician diagnosis of any subtype of JIA. 15–20 patients will be recruited into the individual interview content validation effort. 120 patients will be recruited into the longitudinal testing component.

Study Sites: Cincinnati Children's Hospital Medical Center (CCHMC) Division of Pediatric Rheumatology.

AIM 1b: To evaluate the validity and responsiveness of the PROMIS pediatric item banks parent-proxy and self-report measures with longitudinal assessments in children with Cerebral Palsy.

Study Population: Children aged 5 to 18 years with CP and their parent (or guardian) will be recruited into the study from the CP Multi–specialty clinic. Up to 15–20 parents and up to 15–20 children will be recruited into the individual interview content validation effort. 120 parents and 60 children will be recruited into the longitudinal testing component. PROMIS Parent–Proxy measures will be administered for children ages 5–7, and due to the high prevalence of cognitive impairment in CP, will be used for children ages 8–18 who are unable to self–report. For children with CP ages 8–18 who do meet inclusion criteria and are able to self–report, parent (or guardian) and child dyads will both complete the assessments.

Study Site: CCHMC Cerebral Palsy Multi–specialty Clinic, Division of Pediatric Rehabilitation.

AIM 1c: To evaluate the validity and responsiveness of the PROMIS Pediatrics Item Banks with longitudinal assessments in children with Chronic Musculoskeletal Pain.

Study Population: 140 patients with chronic musculoskeletal pain – regional or widespread – participating in two structured longitudinal intervention treatment programs. Half (70) of the sample will participate in a program consisting of a six session coping skills training protocol, physical therapy and counseling. The other half of the sample will participate in a program of daily intense physical and occupational therapy, weekly music therapy and counseling with a psychologist.

Study Sites: CCHMC and Children's Hospital of Philadelphia.

AIM 2: To enhance PROMIS pediatric pain assessment with new item bank creation and testing.

Study Population/Disease groups: Total 450 children ages 8 – 18 years with a chronic painful condition, including chronic musculoskeletal pain and Sickle Cell Disease.

Study Sites: CCHMC, Children's Hospital of Philadelphia, and Emory University School of Medicine (Sickle Cell Disease).

For questions concerning Cincinnati Children's Hospital Medical Center’s role in the PROMIS network, please contact Esi Morgan DeWitt, MD, MSCE by e-mail at Esi.Morgan-DeWitt@cchmc.org. For more information about the PROMIS network, go to http://www.nihpromis.org.