Symptom assessment in hypereosinophilic syndrome: Toward development of a patient-reported outcomes tool.
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SUBMITTER: Kovacs N
PROVIDER: S-EPMC7554136 | biostudies-literature |
REPOSITORIES: biostudies-literature
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Project description:IntroductionPsoriasis Area Severity Index (PASI) assessment is complex and time-consuming. A simpler assessment measure more sensitive to changes in symptom severity and predictive of patients' quality of life (Dermatology Life Quality Index, DLQI) is needed. This study aims to evaluate the Optimal Psoriasis Assessment Tool (OPAT) as an alternative to PASI.MethodsThis integrated analysis of three UNCOVER trials (NCT01474512, NCT01597245, and NCT01646177) randomized patients (N = 3866) with moderate-to-severe psoriasis to subcutaneously administered ixekizumab 80 mg Q2W or Q4W, or placebo or etanercept 50 mg Q2W. Pearson correlations were computed for clinical and patient-reported measures with PASI and DLQI.ResultsAs the correlations with PASI and BSA were high and not much higher when adding severity, body surface area (BSA) was used for the clinical measure. BSA was the main measure influencing OPAT. Week 12 regression analyses results showed that PASI had a higher correlation with BSA combined with patient assessments than with BSA alone. Sensitivity analyses were also completed for PASI 75 and 90. For DLQI, correlations with the combined measures were even stronger than with BSA alone. A comprehensive model selection procedure was conducted, which illustrated that the two-term models are preferred.ConclusionThe OPAT is a simple and time-saving alternative to PASI. It can be derived using BSA and patient-reported assessments having strong correlation with PASI and moderate correlation with DLQI.
| S-EPMC8322262 | biostudies-literature
Project description:OBJECTIVES: Despite a documented clinical need, no patient reported outcome (PRO) symptom measure meeting current regulatory requirements for clinically relevant end points is available for the evaluation of treatment benefit in diarrhea-predominant IBS (IBS-D). METHODS: Patients (N=113) with IBS-D participated in five study phases: (1) eight concept elicitation focus groups (N=34), from which a 17-item IBS-D Daily Symptom Diary and four-item IBS-D Symptom Event Log (Diary and Event Log) were developed; (2) one-on-one cognitive interviews (N=11) to assess the instrument's comprehensiveness, understandability, appropriateness, and readability; (3) four data triangulation focus groups (N=32) to confirm the concepts elicited; (4) two hybrid (concept elicitation and cognitive interview) focus groups (N=16); and (5) two iterative sets of one-on-one cognitive interviews (N=20) to further clarify the symptoms of IBS-D and debrief a revised seven-item Diary and four-item Event Log. RESULTS: Of thirty-six concepts initially identified, 22 were excluded because they were not saturated, not clinically relevant, not critical symptoms of IBS-D, considered upper GI symptoms, or too broad or vaguely defined. The remaining concepts were diarrhea, immediate need (urgency), bloating/pressure, frequency of bowel movements, cramps, abdominal/stomach pain, gas, completely emptied bowels/incomplete evacuation, accidents, bubbling in intestines (bowel sounds), rectal burning, stool consistency, rectal spasm, and pain while wiping. The final instrument included a daily diary with separate items for abdominal and stomach pain and an event log with four items completed after each bowel movement as follows: (1) a record of the bowel movement/event and an assessment of (2) severity of immediacy of need/bowel urgency, (3) incomplete evacuation, and (4) stool consistency (evaluated using the newly developed Astellas Stool Form Scale). Based on rounds of interviews and clinical input, items considered secondary or nonspecific to IBS-D (rectal burning, bubbling in intestines, spasms, and pain while wiping) were excluded. CONCLUSIONS: The IBS-D Symptom Diary and Event Log represent a rigorously developed PRO instrument for the measurement of the IBS-D symptom experience from the perspective of the patient. Its content validity has been supported, and future work should evaluate the instrument's psychometric properties.
| S-EPMC4077041 | biostudies-other
Project description: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.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.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.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.
| S-EPMC4285435 | biostudies-literature
Project description:β-Thalassemia, a hereditary blood disorder caused by reduced or absent synthesis of the β-globin chain of hemoglobin, is characterized by ineffective erythropoiesis, and can manifest as nontransfusion-dependent thalassemia (NTDT) or transfusion-dependent thalassemia (TDT). Many patients with NTDT develop a wide range of serious complications that affect survival and quality of life (QoL). Patient-reported outcomes (PRO), including health-related QoL (HRQoL), are important tools for determining patient health impairment and selecting appropriate treatment. However, there are currently no disease-specific PRO tools available to assess symptoms related to chronic anemia experienced by patients with NTDT. This study aimed to develop a new, US Food and Drug Administration (FDA)-compliant PRO of chronic anemia symptoms, the NTDT-PRO© tool, for use in patients with NTDT. Participants had a median age of 36 years (range, 18-47) and 60% were female. The initial development of the NTDT-PRO tool involved concept-elicitation interviews with 25 patients from 3 centers (in Lebanon, Greece, and Canada); subsequent interview discussions and clinical input resulted in the generation of 9 items for inclusion in the draft NTDT-PRO. Following a round of cognitive interviews involving 21 patients from 2 centers (in Lebanon and Greece), 4 items (Pain, Headaches, Ability to Concentrate, and Paleness) were removed from the draft NTDT-PRO. The final NTDT-PRO comprises 6 items that measure Tiredness, Weakness, and Shortness of Breath, with or without Physical Activity. The NTDT-PRO is a new disease-specific HRQoL tool for patients with NTDT, developed using a thorough methodology based on FDA 2009 PRO development guidelines.
| S-EPMC6587527 | biostudies-other
Project description:Computer-based, patient-reported symptom survey tools have been described for patients undergoing chemotherapy. We hypothesized that patients undergoing radiotherapy might also benefit, so we developed a computer application to acquire symptom ratings from patients and generate summaries for use at point of care office visits and conducted a randomized, controlled pilot trial to test its feasibility.Subjects were randomized prior to beginning radiotherapy. Both control and intervention group subjects completed the computerized symptom assessment, but only for the intervention group were printed symptom summaries made available before each weekly office visit. Metrics compared included the Global Distress Index (GDI), concordance of patient-reported symptoms and symptoms discussed by the physician and numbers of new and/or adjusted symptom management medications prescribed.One hundred twelve patients completed the study: 54 in the control and 58 in the intervention arms. There were no differences in GDI over time between the control and intervention groups. In the intervention group, more patient-reported symptoms were actually discussed in radiotherapy office visits: 46/202 vs. 19/230. A sensitivity analysis to account for within-subjects correlation yielded 23.2 vs. 10.3 % (p = 0.03). Medications were started or adjusted at 15.4 % (43/280) of control visits compared to 20.4 % (65/319) of intervention visits (p = 0.07).This computer application is easy to use and makes extensive patient-reported outcome data available at the point of care. Although no differences were seen in symptom trajectory, patients who had printed symptom summaries had improved communication during office visits and a trend towards a more active symptom management during radiotherapy.
| S-EPMC4769076 | biostudies-other
Project description:Accurate documentation of patient symptoms in the electronic medical record (EMR) is important for high-quality patient care.To explore inconsistencies between patient self-report on an Eye Symptom Questionnaire (ESQ) and documentation in the EMR.This investigation was an observational study in comprehensive ophthalmology and cornea clinics at an academic institution among a convenience sample of 192 consecutive eligible patients, of whom 30 declined participation. Patients were recruited at the Kellogg Eye Center from October 1, 2015, to January 31, 2016. Patients were eligible to be included in the study if they were 18 years or older.Concordance of symptoms reported on an ESQ with data recorded in the EMR. Agreement of symptom report was analyzed using ? statistics and McNemar tests. Disagreement was defined as a negative symptom report or no mention of a symptom in the EMR for patients who reported moderate to severe symptoms on the ESQ. Logistic regression was used to investigate if patient factors, physician characteristics, or diagnoses were associated with the probability of disagreement for symptoms of blurry vision, pain or discomfort, and redness.A total of 162 patients (324 eyes) were included. The mean (SD) age of participants was 56.6 (19.4) years, 62.3% (101 of 162) were female, and 84.9% (135 of 159) were white. At the participant level, 33.8% (54 of 160) had discordant reporting of blurry vision between the ESQ and EMR. Likewise, documentation was discordant for reporting glare (48.1% [78 of 162]), pain or discomfort (26.5% [43 of 162]), and redness (24.7% [40 of 162]), with poor to fair agreement (? range, -0.02 to 0.42). Discordance of symptom reporting was more frequently characterized by positive reporting on the ESQ and lack of documentation in the EMR (Holm-adjusted McNemar P?<?.03 for 7 of 8 symptoms except for blurry vision [P?=?.59]). Return visits at which the patient reported blurry vision on the ESQ had increased odds of not reporting the symptom in the EMR compared with new visits (odds ratio, 5.25; 95% CI, 1.69-16.30; Holm-adjusted P?=?.045).Symptom reporting was inconsistent between patient self-report on an ESQ and documentation in the EMR, with symptoms more frequently recorded on a questionnaire. These results suggest that documentation of symptoms based on EMR data may not provide a comprehensive resource for clinical practice or "big data" research.
| S-EPMC5404734 | biostudies-other
Project description:ObjectivesMeasuring patient-reported outcomes (PROs) has become increasingly important for assessing quality of care and guiding patient management. However, PROs have yet to be integrated with traditional clinical outcomes (such as length of hospital stay), to evaluate perioperative care. This study aimed to use longitudinal PRO assessments to define the postoperative symptom recovery trajectory in patients undergoing thoracic surgery for lung cancer.MethodsNewly diagnosed patients (N = 60) with stage I or II non-small cell lung cancer who underwent either standard open thoracotomy or video-assisted thoracoscopic surgery lobectomy reported multiple symptoms from before surgery to 3 months after surgery, using the MD Anderson Symptom Inventory. We conducted Kaplan-Meier analyses to determine when symptoms returned to presurgical levels and to mild-severity levels during recovery.ResultsThe most-severe postoperative symptoms were fatigue, pain, shortness of breath, disturbed sleep, and drowsiness. The median time to return to mild symptom severity for these 5 symptoms was shorter than the time to return to baseline severity, with fatigue taking longer. Recovery from pain occurred more quickly for patients who underwent lobectomy versus thoracotomy (8 vs 18 days, respectively; P = .022). Patients who had poor preoperative performance status or comorbidities reported higher postoperative pain (all P < .05).ConclusionsAssessing symptoms from the patient's perspective throughout the postoperative recovery period is an effective strategy for evaluating perioperative care. This study demonstrates that the MD Anderson Symptom Inventory is a sensitive tool for detecting symptomatic recovery, with an expected relationship among surgery type, preoperative performance status, and comorbid conditions.
| S-EPMC4554973 | biostudies-literature
Project description:A questionnaire-based study was conducted to assess long-term patient reported outcomes (PROs) following definitive IMRT-based treatment for early stage carcinomas of the tonsillar fossa.Participants had received IMRT with or without systemic therapy for squamous carcinoma of the tonsillar fossa (T1-2 and N0-2b) with a minimum follow-up of 2years. Patients completed a validated head and neck cancer-specific PRO instrument, the MD Anderson Symptom Inventory-Head and Neck module (MDASI-HN). Symptoms were compared between treatment groups of interest and overall symptom burden was evaluated.Of 139 participants analyzed, 51% had received ipsilateral neck IMRT, and 62% single modality IMRT alone (no systemic therapy). There were no differences in mean severity ratings for the top-ranked individual symptoms or symptom interference for those treated with bilateral versus ipsilateral neck IMRT alone. However, 40% of those treated with bilateral versus 25% of those treated with ipsilateral neck RT alone reported moderate-to-severe levels of dry mouth (p=0.03). Fatigue, numbness/tingling, and constipation were rated more severe for those who had received systemic therapy (p<0.05 for each), but absolute differences were small. Overall, 51% had no more than mild symptom ratings across all 22 symptoms assessed.The long-term patient reported symptom profile in this cohort of tonsil cancer survivors treated with definitive IMRT-based treatment showed a majority of patients with no more than mild symptoms, low symptom interference, and provides an opportunity for future comparison studies with other treatment approaches.
| S-EPMC4762263 | biostudies-literature
Project description:To examine the relationship between objective treadmill test outcomes and subjective symptom outcomes among patients with claudication treated with stent revascularization (ST) compared with supervised exercise (SE).Five scales of the Peripheral Artery Questionnaire and Walking Impairment Questionnaire were correlated with peak walking time and treadmill claudication onset time.The correlation between change in disease-specific quality of life (QOL) and change in peak walking time differed according to treatment group, with statistically significant correlations for all five scales for the ST group and weaker trends for the SE group, only one of which was statistically significant. In contrast, improvements in disease-specific QOL correlated well with increases in claudication onset time, with no significant interaction with treatment group for any of the five scales.Disease-specific QOL results at 6 months in the Claudication: Exercise Vs. Endoluminal Revascularization (CLEVER) study show that improved maximal treadmill walking in patients with claudication treated with SE correlated poorly with self-reported symptom relief. Conversely, patients treated with ST showed good correlation between improved maximal treadmill walking and self-reported symptom improvement. The correlation between claudication onset time and self-reported symptom relief was good across treatment groups. This finding indicates that traditional objective treadmill test outcomes may not correlate well with symptom relief in patients with claudication. Future studies should investigate these data and improve understanding of patient relevance of traditional objective treadmill-based treatment outcomes.
| S-EPMC4724411 | biostudies-literature
Project description:Background Chronic kidney disease (CKD) and kidney failure in childhood are associated with significant and life-altering morbidities and lower quality of life. Emerging evidence suggests that management should be guided in part by symptom burden; however, there is currently no standardized assessment tool for quantifying symptom burden in this pediatric population. This study aimed to develop and refine a patient-reported symptom assessment tool for children with CKD/kidney failure (PRO-Kid), to evaluate the frequency and impact of symptoms. Methods This was a prospective observational study of children and caregivers of children with CKD/kidney failure at two Canadian pediatric care centers. Building on previously published patient-reported outcome measures (PROs) for the assessment of symptom burden in other populations, we drafted a 13-item questionnaire. Cognitive interviews were performed with children and caregivers of children with CKD/kidney failure to iteratively refine the questionnaire. Results Twenty-four participants completed cognitive interviewing (11 children, 13 caregivers). The most common symptoms endorsed were feeling left out, feeling sad/depressed, inability to focus, tiredness, nausea, vomiting, not wanting to eat, and changes in the taste of food. Feeling left out was added to the questionnaire as almost all participants voiced this as a frequent and impactful symptom, resulting in a 14-item questionnaire. Conclusions PRO-Kid is the first pediatric CKD/kidney failure-specific PRO tool to assess symptom burden. Future work should validate this tool in a larger cohort so that it may be used to improve the care of children living with CKD/kidney failure. Graphical abstract A higher resolution version of the Graphical abstract is available as Supplementary information Supplementary Information The online version contains supplementary material available at 10.1007/s00467-021-05269-4.
| S-EPMC8579900 | biostudies-literature