Abstract
P18 Evaluating the feasibility of conducting comparative effectiveness studies in juvenile Localized Scleroderma (jLS)
Pediatric rheumatology online journal, Vol.14(S1)
07/12/2016
DOI: 10.1186/s12969-016-0098-0
PMCID: PMC4943514
PMID: 27409414
Abstract
Aim: To evaluate the feasibility of conducting comparative effectiveness studies in jLS and identify potential confounders for evaluating treatment response. JLS is a rare chronic disease that often causes major morbidity for the growing child including hemiatrophy, arthropathy, and seizures.
Methods
We conducted a prospective observational cohort study of juvenile localized scleroderma subjects who were beginning treatment with one of 3 standardized regimens (consensus treatment plans [CTPs]). The CTPs were: methotrexate alone, methotrexate with intravenous pulse methylprednisolonge, and methotrexate with oral corticosteroids. Choice of CTP was up to the subject’s physician. Subjects were evaluated with standardized activity and damage assessment forms at 6 visits over 1 year. The majority of the data was entered into the web-based legacy CARRA registry; some paper study forms were collected to aid with evaluating clinical assessment tools and additional patient and parent health related quality of life measures. The primary outcome was to assess the feasibility of enrolling patients. Secondary aims were to explore the performance of developed clinical tools for evaluating clinical state and treatment response, and assess the value of other HRQOL questions. Data was analyzed by descriptive statistics, correlation analyses, factor analysis, linear regression, and Bayesian methods.
Results
Physicians at 10 CARRA sites enrolled between 1 and 9 subjects (median 5.5) into all 3 CTPs. The target enrollment of 50 subjects was reached, with enrollment finishing 26 months after study initiation. The actual enrollment period was 16 months, with initiation of enrollment at each site delayed for 10-17 months by time required for IRB and contract approval. The average rate of subject accrual was 3.1 subjects/month during the active enrollment period. There were no significant differences between subjects in the different CTPs for age, gender, race, or ethnicity; most were white, non-hispanic girls with a median age of 13 years, and median disease duration of 13 months. Most were new to treatment with systemic immunosuppressants. At baseline, over 70 % of subjects had extracutaneous involvement, with 32 % having joint involvement and 46 % having a growth difference. Significant differences were found between subjects in the different CTPs for disease duration, prior treatment with systemic immunosuppressants, LS subtype, and some types of extracutaneous involvement.
Conclusions
We achieved our primary outcome of showing the feasibility of conducting comparative effectiveness studies in jLS, a rare disease. We reached our target enrollment and enrolled subjects into all 3 CTPs. We identified a much higher rate of extracutaneous and severe morbidity than has previously been reported, possibly related to detailed prospective data collection. Identifying potential confounders will help determine appropriate sample size for conducting full-scale jLS comparative effectiveness studies.
Details
- Title: Subtitle
- P18 Evaluating the feasibility of conducting comparative effectiveness studies in juvenile Localized Scleroderma (jLS)
- Creators
- Suzanne C LiSandy D Hong - University of Iowa, Stead Family Department of PediatricsKathryn S TorokPolly J Ferguson - University of Iowa, Stead Family Department of PediatricsC. Egla RabinovichMara L BeckerFatma DedeogluMaria F IbarraRob C FuhbriggeKatie G StewartElena PopeRonald M LaxerThomas G MasonGloria C HigginsXiaohu LiMarilynn G PunaroGeorge TomlinsonEleanor PullenayegumJohn MatelskiLaura SchanbergBrian M Feldman
- Resource Type
- Abstract
- Publication Details
- Pediatric rheumatology online journal, Vol.14(S1)
- DOI
- 10.1186/s12969-016-0098-0
- PMID
- 27409414
- PMCID
- PMC4943514
- ISSN
- 1546-0096
- Language
- English
- Date published
- 07/12/2016
- Academic Unit
- Stead Family Department of Pediatrics; Iowa Neuroscience Institute; Rheumatology, Allergy, and Immunology
- Record Identifier
- 9984129302002771
Metrics
17 Record Views