Autoimmune diseases offer unique opportunities to measure disease burden and response to immunomodulation with hematopoietic cell transplantation (HCT). To study baseline impairments in health-related quality of life (HR-QOL), we analyzed correlates of HR-QOL indices among patients with diffuse systemic sclerosis (SSc) participating in the SCOT trial. Subjects with poor prognosis SSc were enrolled in the ongoing multicenter trial comparing immunosuppression with 12 monthly infusions of CY (750mg/m2) vs. immunoablation followed by CD34-selected autologous HCT. Entry criteria are detailed at the study website (www.sclerodermatrial.org). Validated HR-QOL indices included the short form 36 (SF-36) physical component summary (PCS) and mental component summary (MCS), health assessment questionnaire-disability index (HAQ-DI), and the UCLA gastrointestinal instrument (GIT 2.0). We assessed Pearson correlations between HR-QOL indices and objective disease measures: coefficients > / = 0.32 (representing R2 > / = 10%) were considered meaningful associations. 61 subjects were evaluated before treatment: 40 were female; mean age was 46 and disease duration was 3.8 yr; mean (SD) % predicted FVC was 74.6 (16.2) and DLCO was 50.5 (8.2). Mean modified Rodnan Skin Score (mRSS) was 30.9 (9.5). mRSS measures dermal thickening ranging from 0 (none) to 51 (total body sclerosis). Baseline mean PCS was 27.4 which is 2.3 SD below the US general population. The 61 patients had moderate functional disability (mean HAQ-DI 1.4) while the mean total GIT (measured in 21 pts) was 0.76, where 0.4 signifies moderate severity. For comparison, subjects with diffuse SSc enrolled in the Scleroderma Lung Study (comparing CY with placebo, Tashkin NEJM 2006) had a higher (better) baseline PCS (32.1) and lower (better) HAQ-DI (1.02) (Khanna Arthritis Rheum 2005). The table presents Pearson's correlations between HR-QOL indices and mRSS, FVC and DLCO. Correlations (r) with magnitudes of > / = 0.32 are bolded.Table 1Baseline Pearson's Correlation Coefficients (r) for HR-QOL Measures versus Dermal and Pulmonary MeasuresVariablesmRSS1Lower scores denote better HR-QOL or lower intensity of symptoms.FVC2Higher scores denote better HR-QOL or lower intensity.DLCO2Higher scores denote better HR-QOL or lower intensity.SHAQHAQ-DI1Lower scores denote better HR-QOL or lower intensity of symptoms.0.58*Correlations significant at p<0.05. %R 2 >10% (r>0.32) are bolded.0.02-0.41*Correlations significant at p<0.05. %R 2 >10% (r>0.32) are bolded.Pain VAS1Lower scores denote better HR-QOL or lower intensity of symptoms.0.050.050.00SF-36SF-36 PCS 2Higher scores denote better HR-QOL or lower intensity.-0.47*Correlations significant at p<0.05. %R 2 >10% (r>0.32) are bolded.0.020.25SF-36 MCS2Higher scores denote better HR-QOL or lower intensity.-0.030.080.13SF-36 Physical functioning2Higher scores denote better HR-QOL or lower intensity.-0.47*Correlations significant at p<0.05. %R 2 >10% (r>0.32) are bolded.0.100.45*Correlations significant at p<0.05. %R 2 >10% (r>0.32) are bolded.SF-36 Role limitations2Higher scores denote better HR-QOL or lower intensity.-0.40*Correlations significant at p<0.05. %R 2 >10% (r>0.32) are bolded.-0.010.31*Correlations significant at p<0.05. %R 2 >10% (r>0.32) are bolded.SF-36 Pain 2Higher scores denote better HR-QOL or lower intensity.-0.200.100.08SF-36 General health 2Higher scores denote better HR-QOL or lower intensity.-0.15-0.070.07SF-36 Vitality2Higher scores denote better HR-QOL or lower intensity.-0.120.110.06SF-36 Social functioning 2Higher scores denote better HR-QOL or lower intensity.-0.170.000.07SF-36 Role emotional 2Higher scores denote better HR-QOL or lower intensity.-0.150.100.25SF-36 Mental health2Higher scores denote better HR-QOL or lower intensity.-0.040.010.17UCLA GIT 2.0Reflux scale1Lower scores denote better HR-QOL or lower intensity of symptoms.-0.34*Correlations significant at p<0.05. %R 2 >10% (r>0.32) are bolded.0.320.24Distention scale1Lower scores denote better HR-QOL or lower intensity of symptoms.-0.47*Correlations significant at p<0.05. %R 2 >10% (r>0.32) are bolded.0.270.41Diarrhea1Lower scores denote better HR-QOL or lower intensity of symptoms.0.09-0.13-0.23Constipation1Lower scores denote better HR-QOL or lower intensity of symptoms.0.08-0.11-0.30Emotional well-being1Lower scores denote better HR-QOL or lower intensity of symptoms.-0.13-0.050.07Social functioning1Lower scores denote better HR-QOL or lower intensity of symptoms.0.070.33-0.23GIT Total Score-0.290.240.171 Lower scores denote better HR-QOL or lower intensity of symptoms.2 Higher scores denote better HR-QOL or lower intensity.∗ Correlations significant at p<0.05. %R 2 >10% (r>0.32) are bolded. Open table in a new tab Patients in the SCOT study had marked baseline impairments in physical health. Objective disease measures of dermal and pulmonary involvement correlated with symptom burden. Longitudinal analysis (12 test repetitions over 72 mo) at study completion will offer unique rigor in HCT trials, provide a rich basis for comparisons with immune and mechanistic studies, and characterize alterations in HR-QOL indices across the two treatments of SSc.

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