Scleroderma Clinical Trial Consortium Gastrointestinal Research Articles

Construct Validity of the Patient‐Reported Outcomes Measurement Information System Gastrointestinal Symptom Scales in Systemic Sclerosis

Gastrointestinal (GI) involvement is common in patients with systemic sclerosis (SSc; scleroderma). The Patient-Reported Outcomes Measurement Information System (PROMIS) GI symptom item bank captures upper and lower GI symptoms (reflux, disrupted swallowing, nausea/vomiting, belly pain, gas/bloating/flatulence, diarrhea, constipation, and fecal incontinence). The objective of this study was to evaluate the construct validity of the PROMIS GI bank in SSc. A total of 167 patients with SSc were administered the PROMIS GI bank and the University of California, Los Angeles, Scleroderma Clinical Trials Consortium Gastrointestinal Scale (GIT 2.0) instrument. GIT 2.0 is a multi-item instrument that measures SSc-associated GI symptoms. Product-moment correlations and a multitrait-multimethod analysis of the PROMIS GI scales with the GIT 2.0 symptom scales were used to evaluate convergent and discriminant validity. Patients with SSc GI involvement had PROMIS GI scale scores 0.2-0.7 SD worse than the US general population. Correlations among scales measuring the same domains for the PROMIS GI and GIT 2.0 measures were large, ranging from 0.61 to 0.87 (average r = 0.77). The average correlation between different symptom scales was 0.22, supporting discriminant validity. This study provides support for the construct validity of the PROMIS GI scales in SSc. Future research is needed to assess the responsiveness to change of these scales in patients with SSc.

The Impact of Medical Nutrition Therapy for Patients with Advanced Systemic Sclerosis (MNT PASS)

Methods: Eighteen patients with SSc with unintentional weight loss and GI symptoms were consented and recruited. Nutrition symptomology (abridged Patient Generated Subjective Global Assessment), GI symptoms (UCLA Scleroderma Clinical Trial Consortium Gastrointestinal Tracker), health-related quality of life (CDC HRQOL), anthropometrics, diet (24-hour recall) and body composition (dual energy x-ray absorptiometry) were assessed at baseline and 6 weeks following MNT that emphasized greater caloric intake, improved protein distribution and texture alterations. Descriptive statistics (frequencies, means standard deviations), T-tests and c2 tests were conducted.

FRI0223 Development and Validation of Patient-Reported Outcomes Measurement Information System (PROMIS®) Gastrointestinal (GI) Symptom Scales in Systemic Sclerosis

BackgroundThe National Institutes of Health PROMIS® roadmap initiative is a cooperative research program designed to develop, evaluate, and standardize item banks to measure patient-reported outcomes (PROs) across different medical conditions...

OP0131 Prevalence and Predictors of Small Intestinal Bacterial Overgrowth in Systemic Sclerosis

BackgroundGastro-Intestinal (GI) involvement is a well-known complication of Systemic Sclerosis (SSc). Small intestinal bacterial overgrowth (SIBO) is part of such involvement. It can compromise the patient quality of life and...

Nutritional status and gastrointestinal symptoms in systemic sclerosis patients

Gastrointestinal manifestations in systemic sclerosis (SSc) can influence the nutritional status of patients. Our objective was to examine whether nutritional status was associated with symptoms captured by the University of California Los Angeles Scleroderma Clinical Trials Consortium Gastrointestinal Tract Questionnaire (GIT 2.0). A series of 24 University of Utah SSc Center patients were assessed using the MUST, SGA, and GIT 2.0. A single evaluator administered the nutrition assessment and gastrointestinal symptom questionnaire. Nine patients were assessed at moderate to high risk of malnutrition using the Malnutrition Universal Screening Tool (MUST) and 12 patients with moderate to severe malnutrition using Subjective Global Assessment (SGA). Neither MUST nor SGA status was associated with duration of disease. Soilage, social function and emotional subscores were associated with SGA nutritional status. Clinically significant differences in Total GIT 2.0 score, reflux, distention/bloating, soilage, diarrhea, social function and emotional well-being were observed across levels of nutritional status. Clinically significant differences in gastrointestinal tract symptoms were observed across levels of nutritional status in patients with varying severity of SSc. These two clinically utilized tools, the SGA and the GIT 2.0, appear to be complementary in the evaluation of SSc patients.

Effect of Gastrointestinal Manifestations on Quality of Life in 87 Consecutive Patients with Systemic Sclerosis

To assess the effect of gastrointestinal (GI) manifestation on the quality of life in patients with systemic sclerosis (SSc). The University of California, Los Angeles Scleroderma Clinical Trial Consortium Gastrointestinal Tract 2 questionnaire was completed by 87 consecutive patients with SSc attending the scleroderma clinic at a single center. Their clinical features and current therapies were recorded; 100 patients with rheumatologic disorders other than SSc were used as controls. Individual scores were compared between SSc and controls, and between SSc subgroups. Of 87 patients, 76 (90%) were women. Median age was 55 years and disease duration 105 months. Thirty-three (38%) had diffuse and 54 (62%) had limited SSc. Patients with SSc had a higher score than controls in all domains (p < 0.05). Numbers of patients who responded positively to individual questionnaire components are as follows: any GI symptom 86 (99%), reflux 77 (89%), distension 73 (84%), soilage 19 (22%), diarrhea 44 (51%), constipation 51 (59%), well-being 43 (49%), and social 43 (49%). There was no difference between the scores of patients with diffuse and limited disease subtypes. The use of calcium channel blockers did not significantly increase the constipation score (p = 0.99). Patients who responded positively to the reflux, distension, diarrhea, and constipation domains had lower scores in the well-being and social domains. GI manifestations, especially fecal incontinence (affecting 22% of patients), have a negative influence on the quality of life of patients with SSc. There was no difference between SSc disease subtypes.

Measures of systemic sclerosis (scleroderma): Health Assessment Questionnaire (HAQ) and Scleroderma HAQ (SHAQ), Physician‐ and Patient‐Rated Global Assessments, Symptom Burden Index (SBI), University of California, Los Angeles, Scleroderma Clinical Trials Consortium Gastrointestinal Scale (UCLA SCTC GIT) 2.0, Baseline Dyspnea Index (BDI) and Transition Dyspnea Index (TDI) (Mahler's Index), Cambridge Pulmonary Hypertension Outcome Review (CAMPHOR), and Raynaud's Condition Score (RCS)

Measures of systemic sclerosis (scleroderma): Health Assessment Questionnaire (HAQ) and Scleroderma HAQ (SHAQ), Physician‐ and Patient‐Rated Global Assessments, Symptom Burden Index (SBI), University of California, Los Angeles, Scleroderma Clinical Trials Consortium Gastrointestinal Scale (UCLA SCTC GIT) 2.0, Baseline Dyspnea Index (BDI) and Transition Dyspnea Index (TDI) (Mahler's Index), Cambridge Pulmonary Hypertension Outcome Review (CAMPHOR), and Raynaud's Condition Score (RCS)

Minimally Important Differences of the UCLA Scleroderma Clinical Trial Consortium Gastrointestinal Tract Instrument

To provide minimally important difference (MID) estimates for the UCLA Scleroderma Clinical Trial Consortium Gastrointestinal Tract 2.0 (UCLA SCTC GIT 2.0) in a longitudinal observational cohort. We administered the UCLA SCTC GIT 2.0 to 115 patients with systemic sclerosis (SSc) at 2 timepoints 6 months apart. The UCLA SCTC GIT 2.0 has 7 multi-item scales: Reflux, Distension/Bloating, Diarrhea, Fecal Soilage, Constipation, Emotional Well-being, and Social Functioning and a total GIT score. All scales are scored from 0 [better health-related quality of life (HRQOL)] to 3 (worse HRQOL) except the diarrhea and constipation scales (ranges 0-2 and 0-2.5, respectively). Patients also rated their overall and upper and lower GIT involvement during the second visit using a response scale with options "much better; somewhat better; almost the same; somewhat worse; or much worse." The minimally changed group was defined by those reporting they were somewhat better or somewhat worse compared to first visit. Study participants were 84% female and 81% white with a mean disease duration of 6.9 years. The MID estimates for improvement ranged from 0.07 for the Social Functioning scale to 0.36 for the Emotional Well-being scale. For worsening, the MID estimates ranged from 0.06 for the Fecal Soilage scale to 0.21 for the Social Functioning scale. We provide MID estimates for the UCLA SCTC GIT 2.0 scales. This information can aid in interpreting scale scores in future randomized controlled trials and observational studies.

Prevalence and correlates of sleep disturbance in systemic sclerosis--results from the UCLA scleroderma quality of life study

Rheumatologic disorders are associated with sleep disturbances. This study examines sleep disturbance correlates in patients with SSc. Participants are 180 SSc patients in an observational study. At baseline, patients completed the Medical Outcomes Study Sleep measure (MOS-Sleep scale). In addition, patients were administered other patient-reported outcome (PRO) measures including the 36-item short form (SF-36), HAQ disability index (HAQ-DI), Functional Assessment of Chronic Illness Therapy-Fatigue (FACIT-Fatigue), Center for Epidemiologic Studies Depression (CESD) scale and a University of California at Los Angeles Scleroderma Clinical Trial Consortium Gastrointestinal Tract Questionnaire (UCLA SCTC GIT 2.0). Descriptive statistics were assessed for six scales of MOS-Sleep and the 9-item sleep problem index (SLP-9; a composite index). We computed Spearman's rank-order correlations between the MOS-Sleep scales and the HAQ-DI, FACIT-Fatigue, CESD, SSc-SCTC GIT 2.0 and SF-36 scales. In addition, we developed a regression model to assess predictors of SLP-9 scores. Covariates included demographics, physician variables of disease severity and patient-reported variables of worsening symptoms and the PRO measures. SSc patients reported a mean (s.d.) of 7.1 (1.73) h of sleep a night. Patients reported worse scores on four of six scales (except for snoring and sleep quantity) compared with the US general population (P < 0.001). SLP-9 was correlated with worsening pain and dyspnoea over the past 1 month, reflux scale of the UCLA SCTC GIT 2.0, CESD and FACIT-Fatigue (ρ 0.26-0.56). In the stepwise multivariate regression model, the CESD, worsening dyspnoea and reflux scale were significantly associated with SLP-9 index. Sleep disturbances are common in SSc and are associated with worsening dyspnoea, depressed mood and severity of reflux symptoms.

Low-Dose Naltrexone for Pruritus in Systemic Sclerosis

Pruritus is a common symptom in systemic sclerosis (SSc), an autoimmune disease which causes fibrosis and vasculopathy in skin, lung, and gastrointestinal tract (GIT). Unfortunately, pruritus has limited treatment options in this disease. Pilot trials of low-dose naltrexone hydrochloride (LDN) for pruritus, pain, and quality of life (QOL) in other GIT diseases have been successful. In this case series we report three patients that had significant improvement in pruritus and total GIT symptoms as measured by the 10-point faces scale and the University of California Los Angeles Scleroderma Clinical Trials Consortium Gastrointestinal Tract 2.0 (UCLA SCTC GIT 2.0) questionnaire. This small case series suggests LDN may be an effective, highly tolerable, and inexpensive treatment for pruritus and GIT symptoms in SSc.