Background/Purpose: Pediatric localized scleroderma (LS) is an autoimmune disease of the skin and underlying tissue leading to sclerosis and complications such as joint contractures.  The impact of this disease on children and their parents has not been well described.  Most published studies on LS outcomes have centered on physician based cutaneous assessment.  Patient based outcomes are a critical component of research in disease management and clinical trials.  The Childhood Dermatology Life Quality Index (CDLQI) is a quality of life (QOL) measure designed for pediatric skin conditions. The aim of this study is to explore the relationships between physician and patient scored measures of disease outcomes in pediatric localized scleroderma as compared to the CDLQI. 

Methods: Patients included in this cohort were those with completed CDLQIs enrolled in the National Registry for Childhood Onset Scleroderma from 2007-2013.  The following measures were extracted from the database for each patient; the modified Localized Scleroderma Skin Severity Index (mLoSSI), the Localized Scleroderma Damage Index (LoSDI), Physician Global Assessment of Activity, Damage and Severity (PGA-A, PGA-D, and PGA-S), CDLQI and patient and parent Global Assessment of Disease Impact (GA-Pt, GA-Par).  Relationships between the physician and patient reported clinical measures were examined using Spearman’s correlations (p<0.05).

Results: Sixty-eight subjects were identified.  The majority were female (72%) and Caucasian (94%), and were representative of the major LS subtypes in pediatric onset disease.  The median age of onset was 7.8 years old (IQR 4.4-10.3) and age at first clinic visit was 11.2 years old (IQR 8.1-14.7).  Fifty-six patients had follow-up visits to include in the analyses.  The relationships between the clinical outcomes and QOL measures at the initial and follow-up visits were similar. The median CDLQI decreased from 3 (IQR 1-7) at initial visit to 2 (IQR 1-4) at follow-up visits. The impact of disease on patient and parent decreased accordingly, with median GA-Pt 37.5 (IQR 8.8-64.3) at baseline and 9 (IQR 2-23.5) at follow-up, and median GA-Par 43 (IQR 9-67) at initial visit and 12 (5.0-33.8) at follow-up visit.  CDLQI moderately and significantly correlated with the GA-par and GA-patient (rho = 0.584, 0.591, respectively).  The GA-pt and GA-par were moderately and significantly related to each other (rho = 0.615).  The clinician-scored outcome measures did not show a strong correlation to the CDLQI.  At the initial visit, the LoSDI was weakly though significantly correlated (rho=0.256), and at the follow-up visits the PGA-A was weakly though significantly correlated (rho=0.284) to the CDLQI.

Conclusion: At this time, clinical outcomes in LS are based primarily on physician scored measures.  However, the CDLQI did not correlate to the physician scored measures in this sample, but it did correlate to the patient’s and parents’ overall perception of disease impact.  This study suggests that patient and parent global assessments are measuring the same underlying construct as the CDLQI but further study will be required to better clarify the nature of this construct and the reasons for the disconnect between the CDLQI and physician scored disease measures.

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ACR Meeting Abstracts - https://acrabstracts.org/abstract/relationships-between-physician-and-patient-scored-clinical-outcome-measures-in-pediatric-localized-scleroderma/