Anne M. Rentz

Prevalence and Socioeconomic Impact of Upper Gastrointestinal Disorders in the United States: Results of the US Upper Gastrointestinal Study

BACKGROUND & AIMS This study examined the prevalence of upper gastrointestinal (GI) symptoms and symptom groupings and determined impact on disability days in a nationally representative US sample. METHODS A telephone survey of 21,128 adults was conducted including questions about the presence of upper GI symptoms during the past 3 months. Respondents were categorized as symptomatic (ie, reported GI symptoms once per month) or asymptomatic. The survey included questions about missed work, leisure activity, or household activity days. Symptom groupings were identified by using factor analysis, and cluster analysis was used to assign respondents into distinct groups on the basis of these symptom groupings. RESULTS The prevalence of an average of 1 or more upper GI symptoms during the past 3 months was 44.9%. The most common symptoms experienced during the past 3 months were early satiety, heartburn, and postprandial fullness. Factor analysis identified 4 symptom groupings: (1) heartburn/regurgitation; (2) nausea/vomiting; (3) bloating/abdominal pain; and (4) early satiety/loss of appetite. Five respondent clusters were identified; the largest clusters were primarily early satiety/fullness (44%) and gastroesophageal reflux disease-like symptoms (28%). Two small clusters reflected nausea and vomiting (7%) and a heterogeneous symptom profile (4%). Symptomatic respondents reported significantly more missed work, leisure, and household activity days than asymptomatic respondents (all P < .0001). CONCLUSIONS Factor analysis separated GI symptoms into groupings reflecting gastroesophageal reflux disease and dyspepsia: early satiety, postprandial fullness, and loss of appetite; bloating and abdominal pain/discomfort; and nausea and vomiting. These upper GI symptoms were associated with significant loss of work and activity days.

Development and validation of a patient‐assessed gastroparesis symptom severity measure: the Gastroparesis Cardinal Symptom Index

Background : Patient‐based symptom assessments are necessary to evaluate the effectiveness of medical treatments for gastroparesis.

Health-related quality of life associated with irritable bowel syndrome: Comparison with other chronic diseases

BACKGROUND Despite the rapidly growing body of literature on health-related quality of life (HRQoL). placing the results in a context that is meaningful to clinicians and patients is often overlooked. OBJECTIVE This study sought to quantify the impact of irritable bowel syndrome (IBS) on HRQoL by comparing the Medical Outcomes Study 36-item Short-Form Health Survey (SF-36) scores of IBS patients with normative US data and with the scores of patients having other chronic gastrointestinal (GI) and non-GI disorders. METHODS Two IBS reference groups were identified from the published literature: a largely untreated community sample of health maintenance organization (HMO) members (N = 92) and a sample of patients with IBS recruited through clinics and in the community (N = 140). SF-36 scores for these groups were compared with published US population norms (N = 2474) and with published scores for 3 other IBS samples (N = 464); a sample with other chronic GI disorders (dyspepsia [N = 126], gastroesophageal reflux disease [GERD] [N = 516]); and samples with other chronic episodic disorders (asthma [N = 375], migraine [N = 303], panic disorder [N = 73], rheumatoid arthritis [N = 693]). RESULTS The scores of patients in both IBS reference groups were significantly lower on several SF-36 domains than those of the US normative population (P < 0.003). Scores on several SF-36 scales were also significantly lower in the IBS reference groups compared with the GERD, asthma, and migraine samples (P < 0.003). Depending on the IBS sample used, scores did not differ or were higher compared with those in the sample with dyspepsia. Relative to the samples with panic disorder and rheumatoid arthritis, the IBS groups had significantly higher scores on most SF-36 domains (P < 0.003). Scores for the HMO reference group were generally higher than those for the clinic/community reference group. CONCLUSIONS Based on the results of this analysis, IBS is associated with impairment of HRQoL relative to US population norms and to populations with GERD, asthma, or migraine. HRQoL appears to be greater in patients with IBS than in those with panic disorder or rheumatoid arthritis, although the relative symptom severity in these samples was not known.

Gastroparesis Cardinal Symptom Index (GCSI): Development and validation of a patient reported assessment of severity of gastroparesis symptoms

Background: Patient-rated symptom assessments are needed for evaluating the effectiveness of medical treatments and for monitoring outcomes in gastroparesis. Objective: This paper summarizes the development and psychometric evaluation of a new instrument, the Gastroparesis Cardinal Symptom Index (GCSI), for assessing severity of symptoms associated with gastroparesis. Methods: The GCSI was based on reviews of the medical literature, patient focus groups, and interviews with clinicians. A sample of 169 patients with a documented diagnosis of gastroparesis participated in the psychometric evaluation study. Patients completed the GCSI, the SF-36 Health Survey, and disability days questions at baseline and after 8 weeks. A randomly selected sub-sample of 30 subjects returned at 2 weeks to assess test–retest reliability. Clinicians rated severity of symptoms, and both clinicians and patients rated change in gastroparesis-related symptoms over the 8 week study. Results: The GCSI is based on three subscales: post-prandial fullness/early satiety (4 items); nausea/vomiting (3 items), and bloating (2 items). Internal consistency reliability was 0.84 for the GCSI total score and ranged from 0.83 to 0.85 for the subscale scores. Two week test–retest reliability was 0.76 for the total score and ranged from 0.68 to 0.81 for subscale scores. Construct validity was supported, given that we observed significant relationships between clinician assessed symptom severity and GCSI total score, significant differences between gastroparesis and dyspepsia patients (n = 760) on GCSI total (p < 0.0001) and subscale scores (p < 0.03 to p < 0.0001), moderate and significant relationships between GCSI total and SF-36 scores, and significant associations between GCSI total score and reports of restricted activity and bed disability days. Patients with greater symptom severity, as rated by clinicians, reported more symptom severity on GCSI total score. GSCI total scores were responsive to changes in overall gastroparesis symptoms as assessed by clinicians (p < 0.0001) and patients (p= 0.0004). Conclusion: The findings of this study indicate that the GCSI is a reliable and valid instrument for measuring symptom severity in patients with gastroparesis.

Development and psychometric evaluation of the patient assessment of upper gastrointestinal symptom severity index (PAGI-SYM) in patients with upper gastrointestinal disorders

Objective: Describe the development and evaluation of a new self-report instrument, the patient assessment of upper gastrointestinal disorders-symptom severity index (PAGI-SYM) in subjects with gastroesophageal reflux disease (GERD), dyspepsia, or gastroparesis. Methods: Recruited subjects with GERD (n=810), dyspepsia (n=767), or gastroparesis (n=169) from the US, France, Germany, Italy, the Netherlands, and Poland. Subjects completed the PAGI-SYM, SF-36, a disease-specific HRQL measure (PAGI-QOL), and disability day questions. Two-week reproducibility was evaluated in 277 stable subjects. We evaluated construct validity by correlating subscale scores with SF-36, PAGI-QOL, disability days, and global symptom severity scores. Results: The final 20-item PAGI-SYM has six subscales: heartburn/regurgitation, fullness/early satiety, nausea/vomiting, bloating, upper abdominal pain, and lower abdominal pain. Internal consistency reliability was good (α =0.79 0.91); test–retest reliability was acceptable (Intraclass correlation coefficients α =0.60 0.82). PAGI-SYM subscale scores correlated significantly with SF-36 scores (all p < 0.0001), PAGI-QOL scores (all p < 0.0001), disability days (p< 0.0001), and global symptom severity (p < 0.0001). Mean PAGI-SYM scores varied significantly in groups defined by disability days (all p < 0.0001), where greater symptom severity was associated with more disability days. Conclusions: Results suggest the PAGI-SYM, a brief symptom severity instrument, has good reliability and evidence supporting construct validity in subjects with GERD, dyspepsia, or gastroparesis.

The link between health-related quality of life and clinical symptoms among children with attention-deficit hyperactivity disorder

ABSTRACT. Clinical research on attention-deficit hyperactivity disorder (ADHD) has begun to integrate measures of health-related quality of life (HRQL) as part of the overall assessment of treatment outcomes. This study examines the association between HRQL and measures of clinical symptoms of ADHD. Data were gathered from 297 children and adolescents in an 8-week, randomized, double-blind, placebo-controlled, clinical trial of atomoxetine treatment for ADHD. HRQL was assessed with the Child Health Questionnaire 50-item Parent Form. ADHD symptoms were assessed with the ADHD Rating Scale-IV; Parent Version and Clinical Global Impressions-ADHD-Severity. Associations between HRQL and clinical symptoms were assessed with correlations, analyses of variance with post hoc comparisons, and t tests. The Child Health Questionnaire 50-item Parent Form scales assessing psychosocial domains of HRQL were significantly negatively correlated with clinical measures. Improvement in clinical symptoms was associated with corresponding improvement in psychosocial aspects of HRQL. The findings suggest that HRQL instruments can add important information to efficacy measures in clinical trials of ADHD treatment.

Psychometric validation of the child health questionnaire (CHQ) in a sample of children and adolescents with attention-deficit/hyperactivity disorder.

Measuring health-related quality of life (HRQL) in children with attention-deficit/hyperactivity disorder (ADHD) in clinical trials can complement efficacy measures, offering a complete picture of the impact of disease and treatment on overall well-being. The purpose of the current study was to psychometrically validate a multidimensional, generic HRQL instrument completed by parents of children with ADHD. A total of 921 children and adolescents with ADHD participated in a clinical study of atomoxetine. Subjects’ parents completed the 50-item Child Health Questionnaire-Parent Version (CHQ-PF50) at baseline and week 10. ADHD symptoms and behavioral problems were assessed using parent and clinician rating scales. Internal consistency reliability at baseline was 0.88 for the CHQ-PF50 Psychosocial Summary Score, ranging from 0.53 to 0.91 for subscale scores. Construct validity was supported by statistically significant correlations between CHQ-PF50 psychosocial scores and parent and clinician symptom ratings. Several methods were used to determine clinically meaningful differences in patient outcomes: inter- and intra-patient mean CHQ-PF50 change scores were tested and standard error of measurement (SEM) and effect sizes were calculated. Study results indicate the CHQ-PF50 is reliable, valid, and responsive to change in ADHD symptoms, suggesting it may provide a means of assessing HRQL outcomes associated with treatments for ADHD.

Cross-cultural development and validation of a patient self-administered questionnaire to assess quality of life in upper gastrointestinal disorders: The PAGI-QOL?'

AbstractSummarize the Patient Assessment of Upper GastroIntestinal Disorders-Quality of Life (PAGI-QOL©) development and provide results on its reliability and validity from the international psychometric validation in dyspepsia, GastroEsophageal Reflux Disease (GERD), and gastroparesis. Methods: Subjects completed the pilot PAGI-QOL at baseline and 8 weeks; and a subsample also at 2 weeks. Other assessments were: Patient Assessment of Upper GastroIntestinal Disorders-Symptom Severity Index, SF-36, number of disability days. Results: 1736 patients completed the PAGI-QOL at baseline. The questionnaire was reduced, producing a 30-item final version covering five domains: Daily Activities, Clothing, Diet and Food Habits, Relationship (REL), and Psychological Well-Being and Distress. Internal consistency was excellent (Cronbach’s α range: 0.83–0.96). Test–retest reproducibility was good: intraclass correlations coefficients were over 0.70 except for the REL scale (0.61). Concurrent validity between the PAGI-QOL total score and all SF-36 subscale scores was good with moderate (0.52) to strong (0.72) correlations. PAGI-QOL scores showed excellent discriminant properties: patients who had spent some days in bed, had missed some days at work, and were kept from usual activities had much lower PAGI-QOL scores than those who did not (p < 0.0001). Conclusion: The PAGI-QOL is a valid and reliable instrument assessing quality of life in patients with dyspepsia, GERD, or gastroparesis.

Reliability and validity of the National Eye Institute Visual Function Questionnaire-25 in patients with age-related macular degeneration.

PURPOSE To evaluate the psychometric characteristics of the National Eye Institute Visual Function Questionnaire-25 (NEI VFQ-25) in patients with age-related macular degeneration (AMD) who participated in two clinical trials. METHODS A secondary analysis of data from two randomized clinical trials was performed. NEI VFQ-25 data were collected from 1134 of 1146 patients with subfoveal choroidal neovascularization due to AMD with minimally classic or occult with no classic types and predominantly classic type. The NEI VFQ-25 was administered at baseline and months 1, 2, 3, 6, 9, and 12, and the SF-36 Health survey was administered at baseline and months 6 and 12. Visual acuity assessments were completed monthly throughout the studies. Internal consistency reliability and construct validity were examined. RESULTS The average age was 77 years (SD = 7.5; range, 52-96) with 59% women. At baseline, internal consistency reliability was 0.96 for the NEI VFQ-25 total score and ranged from 0.62 (ocular pain) to 0.91 (near activities) for the subscales. NEI VFQ-25 total and subscale scores correlated significantly with SF-36 scores (P < 0.05), and total, near activities, distance activities, and dependency scores correlated significantly with best corrected visual acuity (BCVA) in the better (P < 0.0001) and worse seeing eye (P < 0.0001). Mean NEI VFQ-25 total and subscale scores, except for ocular pain and general health, varied by BCVA group (P < 0.001), with higher impairment scores seen in the lower visual acuity groups. CONCLUSIONS The NEI VFQ-25 demonstrated good reliability and construct validity as a measure of vision-related functioning outcomes in patients with AMD.

Responsiveness and interpretation of a symptom severity index specific to upper gastrointestinal disorders

BACKGROUND AND AIMS Determining clinically meaningful change of patient-reported outcome measures is important for evaluating effectiveness of treatments for gastrointestinal (GI) diseases. This study evaluates responsiveness of the Patient Assessment of Gastrointestinal Disorders-Symptom Severity Index (PAGI-SYM) in gastroesophageal reflux disease (GERD) and dyspepsia. METHODS The PAGI-SYM was based on a review of the published literature and interviews with patients and clinicians. Items were developed to be linguistically and culturally appropriate for multicountry studies. The PAGI-SYM includes 6 subscales: heartburn/regurgitation, fullness/early satiety, nausea/vomiting, bloating, upper abdominal pain, and lower abdominal pain. Subjects with GERD (n = 810) or dyspepsia (n = 767) participated in this multicountry, observational study. All subjects completed the PAGI-SYM, a global symptom relief questionnaire, and a measure of patient-rated change in GI-related symptoms, the Overall Treatment Effect (OTE) scale. Responsiveness was evaluated at 8 weeks by comparing groups by disease, symptom relief, and OTE (improved, stable, and worsened). RESULTS Subjects reporting symptom relief reported significantly lower (better) PAGI-SYM scores than those reporting no symptom relief ( P < 0.0001 to P < 0.0005). Subjects with improvements in overall GI symptoms exhibited significant decreases in PAGI-SYM subscale scores compared with those who remained the same or worsened (all P values < 0.0001). Effect sizes ranged from 0.21-1.28, and standard errors of measurement ranged from 0.29-0.63, depending on subscale and disease sample. CONCLUSIONS The PAGI-SYM is a brief symptom severity instrument that measures common GI symptoms. Results suggest that the PAGI-SYM is responsive and sensitive to change in clinical status in subjects with GERD or dyspepsia.