Capturing the Range of Disease Involvement in Localized Scleroderma: The Total Morbidity Score

Background/Purpose: Localized scleroderma (LS) is a chronic inflammatory and fibrosing disease that causes both cutaneous and extracutaneous (EC) damage. EC involvement (ECI) is common in juvenile LS (jLS), and includes joints, bones, oral cavity, and nervous system. A measure that captures the full spectrum of morbidity would improve disease assessment. The CARRA LS group generated a measure, the Total Morbidity score (TMS), for this purpose.  We report on its performance in a prospective cohort of jLS patients beginning methotrexate (MTX) treatment.

Methods: The TMS specifies that items should only be scored if considered secondary to LS, not to treatment or other factor. TMS items were generated based upon literature review and LS expert group discussion. A modular design was used with multiple items scored within each module: cutaneous (i.e., dyspigmentation, subcutaneous atrophy) including extent, musculoskeletal (MSK, i.e., arthritis, contractures), growth difference of body (i.e., limb girth or length difference), head/neuro (i.e., scalp hair loss, facial hemiatrophy, brain involvement), other organ (i.e., vasculopathy).  The weighting of scored items was determined by surveys, 1000Minds software, and iterative cycles of testing using case scenarios followed by discussion.   We evaluated the TMS in a prospective cohort of jLS patients followed for 12 months after starting MTX treatment (jLS consensus treatment plan pilot study). Clinical assessments included skin scores, physician global assessment of disease damage (PGA-D), and ECI scoring. Data was analyzed by descriptive statistics, with correlations evaluated by Spearman’s rho. p values < 0.05 were considered to be significant.

Results: At baseline, 48 patients were scored. There were 33 females, 44 Caucasian, median age of LS onset 9.6 years, and disease duration 12.5 months. The most common LS subtype was linear scleroderma. Baseline median TMS was 7, range 0-40; the 12 months TMS was 6, range 0-35. Patients with ECI had higher scores than those without, p < 0.001, with a trend towards higher TMS in those with disease duration > 2 years, p =0.086 (Table 1). While higher scores were seen in linear scleroderma and mixed morphea subtype patients, differences were not significant.  

Over 95% of patients had cutaneous damage at 0 and 12 months (Figure 1).  At baseline, about 1/3 had MSK, growth differences, and/or head/neuro morbidity, with decline of MSK morbidity at 12 months. Over time, scores declined in 22, increased in 10, and were stable in 12 patients, with change most often occurring in cutaneous and MSK modules (Figure 2).   Good correlation was found between TMS and PGA-D at 0 and 12 months (Spearman’s rho 0.778, p < 0.001; 0.756, p < 0.001, respectively). This correlation was better than that found for the localized scleroderma skin damage index (0.629 at 0 months, 0.713 at 12 months).

Conclusion: The Total Morbidity Score was developed to capture the range of, and quantify, LS morbidity. Scores were found to reflect a range of damage levels and morbidities in jLS patients, and vary over time. The TMS showed good correlation with PGA-D. Our findings suggest the TMS could aid tracking of clinical status and response. More study  is needed to further assess its performance.

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