Background/Purpose: Localized scleroderma (LS) is an autoimmune disease characterized
by inflammation and fibrosis of the skin and a wide range of extracutaneous manifestations (ECMs). The longitudinal
impact of this disease on children’s quality of life (QoL)
is not currently known. The aim of this study was to longitudinally assess
predictors of QoL impact in a cohort of pediatric LS
patients.

Methods: Subjects
who were 4-16 years old at the time of enrollment into the National Registry
for Childhood Onset Scleroderma were included in analysis.  Children’s Dermatology Life Quality
Index (CDLQI) scores at baseline and follow-up clinic visits were dichotomized;
scores of 0 or 1 indicated minimal QoL impact, and a
score of >1 was considered significant impact.

Hierarchical generalized linear
modeling was used to determine predictors of QoL
impact, first assessing univariably (alpha = 0.10)
and then including all univariably significant
variables in the final model (alpha = 0.05).  Variables included initially were gender,
age at diagnosis, time from initial visit, presence of disease flare, the
activity and damage portions of the physician-scored Localized Scleroderma
Cutaneous Assessment Tool (i.e. the modified Localized Scleroderma Skin
Severity Index (mLoSSI) and the Localized Scleroderma
Damage Index (LoSDI) respectively), presence of
facial lesion(s), use of systemic immunosuppressive medications, total number
of body sites affected, medication side effects, and number of ECMs.

Results: Eighty
subjects with a total of 616 visits were included for analysis, with median
follow-up of 29.5 months (IQR: 14.0-50.3 months) per subject.  Demographics were representative of
typical LS populations (see Table 1). 
Five univariably significant variables were
included in the final model: time from initial visit, mLoSSI,
LoSDI, gender, and number of ECMs.  Males were less likely to have QoL impact than females (OR: 0.12, p = 0.002).  Each month after the initial visit
yielded 5% lower odds of QoL impact (OR: 0.95, p < 0.001).  Two
thirds of participants reported at least one ECM, with musculoskeletal,
orofacial and neurologic ECMs being the most common.  Each additional ECM increased the
likelihood of QoL impact by 37% (OR: 1.37, p = 0.019).  mLoSSI
and LoSDI did not reach significance in the final
model.  Unit-specific and
population-average models were found to be similar; the addition of an
over-dispersion factor to the final model did not meaningfully alter the
results.

*30 visits (5%) missing disease
activity status

Conclusion: This
study is the first longitudinal assessment of predictors of QoL
impact in pediatric LS.  Female sex
and presence of ECMs significantly increase the likelihood of negative QoL impact. In addition, the odds of QoL
impact decrease with time from initial visit, suggesting that factors such as
effective immunosuppressive treatment, physical/occupational therapy, and
patient/family coping mechanisms may mitigate the QoL
impact.