Clinical trial NCT00280137: Health-related Quality of Life Measure in Pediatric Lupus
Official title: Health-related Quality of Life Measure in Pediatric Lupus
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Systemic Lupus Erythematosus
Quality of Life
Keywords: Quality of Life
Pediatric Systemic Lupus Erythematosus
Lead sponsor: Rutgers, The State University of New Jersey
Sponsor class: OTHER
Collaborators: name: Hospital for Special Surgery, New York
name: St. Barnabas Medical Center
name: Hackensack Meridian Health
name: University of Chicago
name: The Cleveland Clinic
name: Children's Hospital Los Angeles
name: Legacy Health System
name: University Hospitals Cleveland Medical Center
name: Tufts Medical Center
name: University of Mississippi Medical Center
First posted: 2006-01-20
Enrollment: 31
Sex: ALL
Minimum age (years): 2
Maximum age (years): 18
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: The population will consist of outpatients during clinics visits and inpatients during hospitalizations.

Brief summary: To examine the psychometric properties of a brief quality of life (QOL) instrument for use in pediatric systemic lupus erythematosus (SLE). The purpose of this prospective study is primarily to determine the validity and reliability of a new health-related quality of life (HRQOL) measure in children with systemic lupus erythematous (SLE). We wish to secondarily examine concordance between child- and parent-reports of the HRQOL measure and identify factors associated with poorer HRQOL in them.

Earlier studies have shown that SLE significantly impacts QOL in adults. At present, there is no disease-specific instrument for measuring HRQOL in children with SLE. In response to these concerns, we developed the "Simple Measure of Impact of Lupus Erythematosus in Youngsters© (SMILEY©). Establishing the validity and reliability of SMILEY©, examining child-parent agreement and identifying factors associated with poorer HRQOL will enable us to measure the impact of SLE in children, and formulate appropriate interventions for this sensitive population. We plan the following specific aims:

1. to determine construct validity and reliability of SMILEY© child and parent versions in children with SLE using gold standards (Pediatric Quality of Life inventory - PedsQL generic and rheumatology modules, Childhood Health Assessment Questionnaire -CHAQ)
2. to determine responsiveness of SMILEY©
3. to examine level of agreement between child- and parent-reports of SMILEY© in children with SLE
4. to identify medical (steroid use, use of disease modifying agents such as cytoxan, cellcept, thalidomide, or cyclosporine, disease duration, disease activity and disease damage etc.) and psychosocial (self-concept, socioeconomic status) factors that affect HRQOL (as measured by child- and parent-reports of SMILEY© and PedsQL generic and rheumatology modules) and physical function 5) to translate, adapt and validate SMILEY in different languages

Detailed description: Pediatric lupus is a chronic multisystem rheumatic disease, associated with significant medical and psychosocial implications. Frequent physician visits for routine, urgent or emergent care, limitation of activities, medication side effects, change in body image, fear of the future, and missing school are all disruptive to the patients and parents and impact all spheres of their lives. There is no lupus-specific questionnaire designed to measure the well-being of children with this disease. We developed a brief questionnaire, "Simple Measure of Impact of Lupus Erythematosus in Youngsters© (SMILEY©) with both child and parent versions. We conducted further research about how children/parents feel about having/their children having lupus and used those responses to modify the preliminary SMILEY©. After several iterations, the SMILEY with 26 questions was developed with parallel child- and parent-versions with responses in the form of 5 faces-scale denoting different expressions, in order to ensure easy comprehension across different ages and cultures.

During their visit, information about their lupus will be collected from the children and parents, and they will be asked to complete the SMILEY© and questionnaires measuring quality of life, physical function, self-esteem and behavior. They will be given an additional copy of SMILEY©, which they will be asked to complete within 10 days of their initial evaluation and return by mail (self-addressed, stamped envelope will be provided). Then both children and legal guardians will again be asked to complete questionnaires including SMILEY© during their subsequent visits at least every 3-6 month intervals or earlier if there has been a change in disease activity as determined by the physician. Medication use, disease activity and disease severity assessments will be made by the physician with initial and subsequent evaluations. We will determine the psychometrics properties of SMILEY and responsiveness to change in disease activity.

Both national and international sites will be included in the study. SMILEY in addition will be translated and adapated to different languages and subsequently validated. This study will provide valuable information about the impact of lupus on their overall well-being. Being a brief, valid, reliable and easy to administer instrument, SMILEY© would be suitable for use across different age groups, languages and cultures. SMILEY© will be used as an important clinical outcome tool in both clinical and research arenas, thus enabling us to formulate appropriate interventions.

Eligibility criteria: Inclusion Criteria:

* (1) Willing to participate
* (2) Have a child with SLE that meets eligibility criteria for the study

Exclusion Criteria:

* (1) Subjects who are not well enough to complete the questionnaires
* (2) Physical or mental disabilities which would seriously affect the individual's ability to understand the informed consent or study questionnaires
* (3) Refusal to participate

Primary outcomes:
measure: Health Related Quality of Life - scores of the SMILEY scale for child and parent reports
timeFrame: At different points of the study
Secondary outcomes:
Other outcomes:
Locations:
city: Los Angeles
country: United States
facility: Childrens Hospital Los Angeles
state: California
city: Chicago
country: United States
facility: La Rabida Children's Hospital - The University of Chicago
state: Illinois
city: Boston
country: United States
facility: New England Medical Center -Tufts
state: Massachusetts
city: Jackson
country: United States
facility: University of Mississippi Medical Center
state: Mississippi
city: Hackensack
country: United States
facility: Hackensack University Medical Center
state: New Jersey
city: Livingston
country: United States
facility: St. Barnabas Medical Center
state: New Jersey
city: New Brunswick
country: United States
facility: University of Medicine and Dentistry - Robert Wood Johnson University Hospital
state: New Jersey
city: New York
country: United States
facility: Hospital for Special Surgery
state: New York
city: Cleveland
country: United States
facility: The CLeveland CLinic
state: Ohio
city: Cleveland
country: United States
facility: University Hospital Case Medical Center
state: Ohio
city: Portland
country: United States
facility: Legacy Health System
state: Oregon
Study officials:
affiliation: UMDNJ/RWJUH Department of Pediatrics
name: Lakshmi N Moorthy, MD, MS
role: PRINCIPAL_INVESTIGATOR
References:
citation: Moorthy LN, Robbins L, Harrison MJ, Peterson MG, Cox N, Onel KB, Lehman TJ. Quality of life in paediatric lupus. Lupus. 2004;13(4):234-40. doi: 10.1191/0961203304lu1003oa.
citation: Bombardier C, Gladman DD, Urowitz MB, Caron D, Chang CH. Derivation of the SLEDAI. A disease activity index for lupus patients. The Committee on Prognosis Studies in SLE. Arthritis Rheum. 1992 Jun;35(6):630-40. doi: 10.1002/art.1780350606.
citation: Stoll T, Stucki G, Malik J, Pyke S, Isenberg DA. Association of the Systemic Lupus International Collaborating Clinics/American College of Rheumatology Damage Index with measures of disease activity and health status in patients with systemic lupus erythematosus. J Rheumatol. 1997 Feb;24(2):309-13.
citation: Varni JW, Seid M, Kurtin PS. PedsQL 4.0: reliability and validity of the Pediatric Quality of Life Inventory version 4.0 generic core scales in healthy and patient populations. Med Care. 2001 Aug;39(8):800-12. doi: 10.1097/00005650-200108000-00006.
citation: Varni JW, Seid M, Rode CA. The PedsQL: measurement model for the pediatric quality of life inventory. Med Care. 1999 Feb;37(2):126-39. doi: 10.1097/00005650-199902000-00003.
citation: Singh G, Athreya BH, Fries JF, Goldsmith DP. Measurement of health status in children with juvenile rheumatoid arthritis. Arthritis Rheum. 1994 Dec;37(12):1761-9. doi: 10.1002/art.1780371209.