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, eye, 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 related to LS, not to treatment or other factor. TMS items were generated based upon literature review and expert group discussion. A modular design was used, with modules of cutaneous features (i.e., dyspigmentation, subcutaneous atrophy) and extent, musculoskeletal (MSK, i.e., arthritis, myositis), growth difference of body (i.e., limb girth or length difference), head/neuro (i.e., 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 and 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, Wilcoxon sign rank test, Spearman’s rho. p values < 0.05 were considered to be significant.

Results: Fourty-eight patients had TMS scores at baseline, 43 at 12 months. There were 33 females, 44 Caucasian, median age of LS onset 9.6 years, median disease duration 12.5 months. The most common LS subtype was linear scleroderma. Baseline median TMS was 7, range 0-40. Patients with ECI had higher scores than those without (p < 0.001, Table 1), and there was a trend towards higher scores in patients with disease duration > 2 years (p =0.086, Table 1). Higher scores were seen in linear scleroderma and mixed morphea subtype patients, but these differences were not significant (Table 1).

The most commonly scored TMS module was Cutaneous, followed by MSK, Growth differences, and Head/Neuro modules, which were each scored in >30% of patients at baseline (Figure 1). From 0 to 12 months, scores changed in 31 patients (72%), with change most often occurring in the Cutaneous and MSK modules (Figure 2).  Patients with linear scleroderma had a decrease in TMS scores from 0 (9, IQR 6-15) to 12 months (6, IQR 5-12, p =0.028).  Good correlation was found between TMS and PGA-D at 0 and 12 months (rho = 0.778, p < 0.001; 0.756, p < 0.001, respectively), higher 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.

Table 1: Total Morbidity Scores at the Baseline Visit of 48 jLS patients beginning methotrexate-based treatment. The number of patient in each category, their median TMS, and the range of scores is shown. P-values were calculated by t-test, p < 0.05 considered to be significant. EC: extracutaneous; IQR: interquartile range; jLS: juvenile localized scleroderma.

Figure 1: Percent of patients that were scored for each of the modules of the Total Morbidity Score at start of treatment (0 months) and after 12 months of methotrexate treatment. 48 jLS patients were assessed at 0 months, 43 at 12 months. Cutaneous refers to skin features (dyspigmentation, dermal atrophy, and skin thickening), subcutaneous tissue atrophy, and extent of these features in affected anatomic sites. Musculoskeletal (MSK) includes arthritis, joint contractures, myositis, fasciitis. Growth difference includes girth hemiatrophy, limb length differences, and breast, trunk, or buttock hemiatrophy. Head/Neuro includes hair loss, facial disfigurement, seizures, peripheral neuropathy, brain imagining abnormalities, and ocular or oral involvement. Other organ includes gastroesophageal reflux, Raynauds phenomenon, and other miscellaneous morbidities.

Figure 2: Change in TMS modules from 0 to 12 months. JLS patients were followed for 12 months after beginning methotrexate treatment. The columns show the percent of patients that had a change in their score of each TMS module from 0 to 12 months. 31 patients had a change in their TMS score, and 12 had stable scores. Cut. Extent (cutaneous extent) refers to estimate of surface area of involvement of skin and subcutaneous damage features in each affected anatomic site. Growth diff = growth difference module.

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ACR Meeting Abstracts - https://acrabstracts.org/abstract/capturing-the-range-of-disease-involvement-in-localized-scleroderma-the-total-morbidity-score-2/