Cynthia B. Whitman

Development of the NIH Patient-Reported Outcomes Measurement Information System (PROMIS) Gastrointestinal Symptom Scales

OBJECTIVES:The National Institutes of Health (NIH) Patient-Reported Outcomes Measurement Information System (PROMIS®) is a standardized set of patient-reported outcomes (PROs) that cover physical, mental, and social health. The aim of this study was to develop the NIH PROMIS gastrointestinal (GI) symptom measures.METHODS:We first conducted a systematic literature review to develop a broad conceptual model of GI symptoms. We complemented the review with 12 focus groups including 102 GI patients. We developed PROMIS items based on the literature and input from the focus groups followed by cognitive debriefing in 28 patients. We administered the items to diverse GI patients (irritable bowel syndrome (IBS), inflammatory bowel disease (IBD), systemic sclerosis (SSc), and other common GI disorders) and a census-based US general population (GP) control sample. We created scales based on confirmatory factor analyses and item response theory modeling, and evaluated the scales for reliability and validity.RESULTS:A total of 102 items were developed and administered to 865 patients with GI conditions and 1,177 GP participants. Factor analyses provided support for eight scales: gastroesophageal reflux (13 items), disrupted swallowing (7 items), diarrhea (5 items), bowel incontinence/soilage (4 items), nausea and vomiting (4 items), constipation (9 items), belly pain (6 items), and gas/bloat/flatulence (12 items). The scales correlated significantly with both generic and disease-targeted legacy instruments, and demonstrate evidence of reliability.CONCLUSIONS:Using the NIH PROMIS framework, we developed eight GI symptom scales that can now be used for clinical care and research across the full range of GI disorders.

Development of an index to define overall disease severity in IBD

Background and aim Disease activity for Crohns disease (CD) and UC is typically defined based on symptoms at a moment in time, and ignores the long-term burden of disease. The aims of this study were to select the attributes determining overall disease severity, to rank the importance of and to score these individual attributes for both CD and UC. Methods Using a modified Delphi panel, 14 members of the International Organization for the Study of Inflammatory Bowel Diseases (IOIBD) selected the most important attributes related to IBD. Eighteen IOIBD members then completed a statistical exercise (conjoint analysis) to create a relative ranking of these attributes. Adjusted utilities were developed by creating proportions for each level within an attribute. Results For CD, 15.8% of overall disease severity was attributed to the presence of mucosal lesions, 10.9% to history of a fistula, 9.7% to history of abscess and 7.4% to history of intestinal resection. For UC, 18.1% of overall disease severity was attributed to mucosal lesions, followed by 14.0% for impact on daily activities, 11.2% C reactive protein and 10.1% for prior experience with biologics. Overall disease severity indices were created on a 100-point scale by applying each attributes average importance to the adjusted utilities. Conclusions Based on specialist opinion, overall CD severity was associated more with intestinal damage, in contrast to overall UC disease severity, which was more dependent on symptoms and impact on daily life. Once validated, disease severity indices may provide a useful tool for consistent assessment of overall disease severity in patients with IBD.

Clinical Factors and the Decision to Transfuse Chronic Dialysis Patients

BACKGROUND AND OBJECTIVES Red blood cell transfusion was previously the principle therapy for anemia in CKD but became less prevalent after the introduction of erythropoiesis-stimulating agents. This study used adaptive choice-based conjoint analysis to identify preferences and predictors of transfusion decision-making in CKD. DESIGN, SETTING, PARTICIPANTS, & MEASUREMENTS A computerized adaptive choice-based conjoint survey was administered between June and August of 2012 to nephrologists, internists, and hospitalists listed in the American Medical Association Masterfile. The survey quantified the relative importance of 10 patient attributes, including hemoglobin levels, age, occult blood in stool, severity of illness, eligibility for transplant, iron indices, erythropoiesis-stimulating agents, cardiovascular disease, and functional status. Triggers of transfusions in common dialysis scenarios were studied, and based on adaptive choice-based conjoint-derived preferences, relative importance by performing multivariable regression to identify predictors of transfusion preferences was assessed. RESULTS A total of 350 providers completed the survey (n=305 nephrologists; mean age=46 years; 21% women). Of 10 attributes assessed, absolute hemoglobin level was the most important driver of transfusions, accounting for 29% of decision-making, followed by functional status (16%) and cardiovascular comorbidities (12%); 92% of providers transfused when hemoglobin was 7.5 g/dl, independent of other factors. In multivariable regression, Veterans Administration providers were more likely to transfuse at 8.0 g/dl (odds ratio, 5.9; 95% confidence interval, 1.9 to 18.4). Although transplant eligibility explained only 5% of decision-making, nephrologists were five times more likely to value it as important compared with non-nephrologists (odds ratio, 5.2; 95% confidence interval, 2.4 to 11.1). CONCLUSIONS Adaptive choice-based conjoint analysis was useful in predicting influences on transfusion decisions. Hemoglobin level, functional status, and cardiovascular comorbidities most strongly influenced transfusion decision-making, but preference variations were observed among subgroups.

Computer-Generated Vs. Physician-Documented History of Present Illness (HPI): Results of a Blinded Comparison

Objectives:Healthcare delivery now mandates shorter visits with higher documentation requirements, undermining the patient–provider interaction. To improve clinic visit efficiency, we developed a patient–provider portal that systematically collects patient symptoms using a computer algorithm called Automated Evaluation of Gastrointestinal Symptoms (AEGIS). AEGIS also automatically “translates” the patient report into a full narrative history of present illness (HPI). We aimed to compare the quality of computer-generated vs. physician-documented HPIs.Methods:We performed a cross-sectional study with a paired sample design among individuals visiting outpatient adult gastrointestinal (GI) clinics for evaluation of active GI symptoms. Participants first underwent usual care and then subsequently completed AEGIS. Each individual thereby had both a physician-documented and a computer-generated HPI. Forty-eight blinded physicians assessed HPI quality across six domains using 5-point scales: (i) overall impression, (ii) thoroughness, (iii) usefulness, (iv) organization, (v) succinctness, and (vi) comprehensibility. We compared HPI scores within patient using a repeated measures model.Results:Seventy-five patients had both computer-generated and physician-documented HPIs. The mean overall impression score for computer-generated HPIs was higher than physician HPIs (3.68 vs. 2.80; P<0.001), even after adjusting for physician and visit type, location, mode of transcription, and demographics. Computer-generated HPIs were also judged more complete (3.70 vs. 2.73; P<0.001), more useful (3.82 vs. 3.04; P<0.001), better organized (3.66 vs. 2.80; P<0.001), more succinct (3.55 vs. 3.17; P<0.001), and more comprehensible (3.66 vs. 2.97; P<0.001).Conclusions:Computer-generated HPIs were of higher overall quality, better organized, and more succinct, comprehensible, complete, and useful compared with HPIs written by physicians during usual care in GI clinics.

Development of an Online Library of Patient-Reported Outcome Measures in Gastroenterology: The GI-PRO Database

OBJECTIVES:Because gastrointestinal (GI) illnesses can cause physical, emotional, and social distress, patient-reported outcomes (PROs) are used to guide clinical decision making, conduct research, and seek drug approval. It is important to develop a mechanism for identifying, categorizing, and evaluating the over 100 GI PROs that exist. Here we describe a new, National Institutes of Health (NIH)-supported, online PRO clearinghouse—the GI-PRO database.METHODS:Using a protocol developed by the NIH Patient-Reported Outcome Measurement Information System (PROMIS®), we performed a systematic review to identify English-language GI PROs. We abstracted PRO items and developed an online searchable item database. We categorized symptoms into content “bins” to evaluate a framework for GI symptom reporting. Finally, we assigned a score for the methodological quality of each PRO represented in the published literature (0–20 range; higher indicates better).RESULTS:We reviewed 15,697 titles (κ>0.6 for title and abstract selection), from which we identified 126 PROs. Review of the PROs revealed eight GI symptom “bins”: (i) abdominal pain, (ii) bloat/gas, (iii) diarrhea, (iv) constipation, (v) bowel incontinence/soilage, (vi) heartburn/reflux, (vii) swallowing, and (viii) nausea/vomiting. In addition to these symptoms, the PROs covered four psychosocial domains: (i) behaviors, (ii) cognitions, (iii) emotions, and (iv) psychosocial impact. The quality scores were generally low (mean 8.88±4.19; 0 (min)–20 (max). In addition, 51% did not include patient input in developing the PRO, and 41% provided no information on score interpretation.CONCLUSIONS:GI PROs cover a wide range of biopsychosocial symptoms. Although plentiful, GI PROs are limited by low methodological quality. Our online PRO library (www.researchcore.org/gipro/) can help in selecting PROs for clinical and research purposes.

Development and validation of a disease-targeted quality of life instrument for chronic diverticular disease: the DV-QOL.

BackgroundColonic diverticular disease is typically conceived as acute diverticulitis attacks surrounded by periods of clinical silence. However, evolving data indicate that many patients have persistent symptoms and diminished health-related quality of life (HRQOL) long after acute attacks. We developed a disease-targeted HRQOL measure for symptomatic uncomplicated diverticular disease (SUDD)—the diverticulitis quality of life (DV-QOL) instrument.MethodsWe conducted a systematic literature review to craft a conceptual model of SUDD HRQOL. This was complemented by three focus groups including 45 SUDD patients. We developed items based on our literature search, focus groups, and cognitive debriefings. We administered the items to SUDD patients with persistent symptoms following a confirmed diverticulitis event. We created scales based on factor analysis and evaluated the scales for reliability and validity.ResultsConcept elicitation revealed a range of illness experiences attributed to SUDD. Coding of 20,490 transcribed words yielded a 52-code network with four primary, condition-related concepts: (1) physical symptoms (e.g., bloating); (2) behaviors (e.g., restrictions); (3) cognitions and concerns (e.g., fear); and (4) impact and consequences (e.g., absenteeism, anxiety). Based on patient language, we developed the 17-item DV-QOL instrument. In a cross-sectional validation sample of 197 patients, DV-QOL discriminated between patients with recent versus distant diverticulitis events and correlated highly with Short Form 36 and hospital anxiety and depression scores.ConclusionsPatients with SUDD attribute a wide range of negative psychological, social, and physical symptoms to their condition, both during and after acute attacks; DV-QOL captures these symptoms in a valid, reliable manner.

Impact of National Institutes of Health Gastrointestinal PROMIS Measures in Clinical Practice: Results of a Multicenter Controlled Trial

OBJECTIVES:The National Institutes of Health (NIH) created the Patient Reported Outcomes Measurement Information System (PROMIS) to allow efficient, online measurement of patient-reported outcomes (PROs), but it remains untested whether PROMIS improves outcomes. Here, we aimed to compare the impact of gastrointestinal (GI) PROMIS measures vs. usual care on patient outcomes.METHODS:We performed a pragmatic clinical trial with an off-on study design alternating weekly between intervention (GI PROMIS) and control arms at one Veterans Affairs and three university-affiliated specialty clinics. Adults with GI symptoms were eligible. Intervention patients completed GI PROMIS symptom questionnaires on an e-portal 1 week before their visit; PROs were available for review by patients and their providers before and during the clinic visit. Usual care patients were managed according to customary practices. Our primary outcome was patient satisfaction as determined by the Consumer Assessment of Healthcare Providers and Systems questionnaire. Secondary outcomes included provider interpersonal skills (Doctors’ Interpersonal Skills Questionnaire (DISQ)) and shared decision-making (9-item Shared Decision Making Questionnaire (SDM-Q-9)).RESULTS:There were 217 and 154 patients in the GI PROMIS and control arms, respectively. Patient satisfaction was similar between groups (P>0.05). Intervention patients had similar assessments of their providers’ interpersonal skills (DISQ 89.4±11.7 vs. 89.8±16.0, P=0.79) and shared decision-making (SDM-Q-9 79.3±12.4 vs. 79.0±22.0, P=0.85) vs. controls.CONCLUSIONS:This is the first controlled trial examining the impact of NIH PROMIS in clinical practice. One-time use of GI PROMIS did not improve patient satisfaction or assessment of provider interpersonal skills and shared decision-making. Future studies examining how to optimize PROs in clinical practice are encouraged before widespread adoption.

Addressing Low Colorectal Cancer Screening in African Americans: Using Focus Groups to Inform the Development of Effective Interventions

African Americans have the highest burden of colorectal cancer (CRC) in the United States of America (USA) yet lower CRC screening rates than whites. Although poor screening has prompted efforts to increase screening uptake, there is a persistent need to develop public health interventions in partnership with the African American community. The aim of this study was to conduct focus groups with African Americans to determine preferences for the content and mode of dissemination of culturally tailored CRC screening interventions. In June 2013, 45–75-year-old African Americans were recruited through online advertisements and from an urban Veterans Affairs system to create four focus groups. A semi-structured interview script employing open-ended elicitation was used, and transcripts were analyzed using ATLAS.ti software to code and group data into a concept network. A total of 38 participants (mean age = 54) were enrolled, and 59 ATLAS.ti codes were generated. Commonly reported barriers to screening included perceived invasiveness of colonoscopy, fear of pain, and financial concerns. Facilitators included poor diet/health and desire to prevent CRC. Common sources of health information included media and medical providers. CRC screening information was commonly obtained from medical personnel or media. Participants suggested dissemination of CRC screening education through commercials, billboards, influential African American public figures, Internet, and radio. Participants suggested future interventions include culturally specific information, including details about increased risk, accessing care, and dispelling of myths. Public health interventions to improve CRC screening among African Americans should employ media outlets, emphasize increased risk among African Americans, and address race-specific barriers. Specific recommendations are presented for developing future interventions.

Responsiveness to Change and Minimally Important Differences of the Patient-Reported Outcomes Measurement Information System Gastrointestinal Symptoms Scales

BackgroundThe NIH-sponsored Patient-Reported Outcomes Measurement Information System (PROMIS) Gastrointestinal (GI) Symptoms scales were developed to assess patients’ GI symptoms in clinical settings.AimsTo assess responsiveness to change and provide minimally important difference (MID) estimates for the PROMIS GI Symptoms scales.MethodsA sample of 256 GI outpatients self-administered the eight PROMIS GI Symptoms scales (gastroesophageal reflux, disrupted swallowing, diarrhea, bowel incontinence/soilage, nausea and vomiting, constipation, belly pain, and gas/bloating/flatulence) at two visits. Patient self-reported and physician-reported assessments of the subjects’ overall GI condition were employed as change anchors. In addition, we prospectively assessed change at both visits using a GI-symptom anchor, the Gastrointestinal Symptom Rating Scale (GSRS). Responsiveness to change was assessed using F-statistics. The minimally changed group was those somewhat better or somewhat worse on the retrospective anchors and changing by one category on the modified GSRS (e.g., from slight to mild discomfort to moderate to moderately severe discomfort).ResultsResponsiveness to change was statistically significant for 6 of 8 PROMIS scales using the self-report GI anchor, 3 of 8 scales using the physician-reported anchor, and 5 of 5 scales using the corresponding GSRS scales as anchors. The MID estimates for scales for improvement and worsening were about 0.5–0.6 SD using the GSRS anchor and generally larger in magnitude than the change for the “about the same” group.ConclusionsThe responsiveness and MID estimates provided here for the PROMIS GI Symptoms scales can aid in scale score interpretation in clinical trials and observational studies.

Mo1131 Development and Validation of Prism: A Patient Reported Outcome Measure for GERD Patients Who Are Partial or Non-Responders to PPI Therapy

Background: Most patients with gastro-esophageal reflux disease (GERD) experience relief following treatment with proton pump inhibitors (PPI). As many as 17-44% of patients, however, exhibit partial or non-response to PPI therapy. Self-report of symptoms is fundamental to diagnosing and monitoring treatment response in these patients, yet most extant GERD PRO instruments fail to meet development best-practices as described by the FDA. We therefore sought to develop and validate a PRO instrument for clinical trials involving patients with GERD who are PPI partial-responders. Methods: We conducted a systematic literature review, held patient focus groups, convened an expert panel, and conducted cognitive interviews in order to establish content validity. Eligible participants took PPI therapy for at least 8 weeks, had undergone an upper endoscopy revealing no more than L.A. Classification Grade A esophagitis, and scored at least 8 points on the GerdQ. We used ATLAS.TI software to code focus group transcripts, generating frequencies at the domain, subdomain, and primary code levels. Results informed the construction of a conceptual framework, and guided development of 26 draft items. These items and subsequent revisions were reviewed by two expert panels and debriefed with cognitive interview participants. These efforts resulted in PRISM, a 21-item instrument that underwent psychometric evaluation during a Phase IIB drug trial (Protocol SPD557-206). Results: A diverse sample participated in focus groups (N=38; mean age=50.8; 55%M), saturating the codebook and supporting a preliminary conceptual framework. Following drafting of items, a new sample of patients participated in cognitive interviews for purposes of item revision (N=20; Age=52.6; 55%M). During the trial, an exploratory item assessment (n=104) revealed that all but one of the PRISM items met expectations for internal and external indices. A confirmatory factor analysis (n=220) resulted in a 4-factor model displaying the highest goodness-of-fit scores. All domains had a high inter-item correlation (Cronbachs α>0.8). Test-retest reliability was excellent with intraclass correlation also exceeding 0.8. Evidence for convergent validity was strong, with highly significant (p<.01) correlations between average weekly PRISM scores and severity anchors, and significant (p<.05) correlations with RDQ sub-scales. Three of 4 PRISM sub-scale scores were highly responsive to changes in both self-reported severity and symptom relief. Cumulative distribution functions revealed significant differences between responders and non-responders. Conclusion: PRISM demonstrates strong validity and psychometric properties in a clinical trial setting. Developed in line with FDA guidance on PROs, PRISM represents an new outcome measure for GERD patients with partial or nonresponse to PPI therapy.