scholarly journals HFR-3 Patient reported outcomes on symptom improvement- a good predictor of histological remission in eosinophilic esophagitis?

2021 ◽  
Author(s):  
Pavit Luthra ◽  
Konstantinos Giaslakiotis ◽  
Sami Hoque
Keyword(s):  
Eosinophilic Esophagitis ◽  
Good Predictor ◽  
Patient Reported Outcomes ◽  
Symptom Improvement ◽  
Patient Reported ◽  
Histological Remission

Qualitative Assessment of Patient-reported Outcomes in Adults With Eosinophilic Esophagitis

2011 ◽  
Vol 45 (9) ◽  
pp. 769-774 ◽  
Author(s):  
Tiffany H. Taft ◽  
Emily Kern ◽  
Laurie Keefer ◽  
David Burstein ◽  
Ikuo Hirano
Keyword(s):  
Eosinophilic Esophagitis ◽  
Patient Reported Outcomes ◽  
Qualitative Assessment ◽  
Patient Reported

scholarly journals OP09 Patient reported outcomes reflect histologic disease activity in patients with Ulcerative Colitis: Interim analysis of the APOLLO study

2021 ◽  
Vol 15 (Supplement_1) ◽  
pp. S008-S009
Author(s):  
B Verstockt ◽  
C Jorissen ◽  
E Hoefkens ◽  
N Lembrechts ◽  
L Pouillon ◽  
...  
Keyword(s):  
Ulcerative Colitis ◽  
Disease Activity ◽  
Patient Reported Outcomes ◽  
Activity Index ◽  
Remission Rate ◽  
Stool Frequency ◽  
Patient Reported ◽  
Endoscopic Remission ◽  
Histological Remission ◽  
General Wellbeing

Abstract Background Treating beyond endoscopic remission, aiming for histological remission, has shown to reduce relapse and hospitalization rates in patients with ulcerative colitis (UC). However, very little is known on how histological remission associates with patient reported outcomes (PROMs). Methods PROMs (Simple clinical colitis activity index [SCCAI], IBD disk and Visual Analogue Scales [VAS]) were prospectively collected through a digital questionnaire in all patients with UC undergoing colonoscopy between July 21st 2020-Jan 21st 2021. Mayo endoscopic sub score and UCEIS were determined, as well as the Nancy histologic index (NHI) of the most affected area. Endoscopic remission was defined as Mayo endoscopic sub score 0 and UCEIS 0; histologic remission as NHI 0, absence of active inflammation as NHI ≤ 1. PRO2 remission was defined as stool frequency ≤ 1 (absolute stool frequency ≤ 3 OR 1–2 stools more than usual) and rectal bleeding score of 0. Results Fifty-six paired assessments were collected in 48 unique patients (Table 1), with a histologic, endoscopic and PRO-2 remission rate of 23.2%, 28.6% and 38.2% respectively. Patients with histologic remission or absence of histologic inflammation had a significantly lower overall IBD disability (p=0.007, p=0.003) and disease activity score (p=0.003, p<0.001), as compared to patients without. In line, NHI correlated with the overall IBD disk (r=0.40, p=0.002) and SCCAI score (r=0.50, p<0.001). Many individual components of both scores (abdominal pain, arthralgia, impact on education and work/interpersonal interactions/sexual function, regulation of defecation, blood loss, general wellbeing, joint pain, numbers of stools during night/day, urgency) differed significantly between patients with and without histologic remission. VAS scores assessing general wellbeing (r=0.33, p=0.01), impact on daily activities (r=0.41, p=0.002), UC-related symptoms (r=0.42, p=0.001) and worries (r=0.40, p=0.002) correlated with histology. Quartile analysis of the overall IBD disk and SCCAI scores confirmed the highest likelihood for histologic remission in patients with the lowest scores (Q1-Q2 vs Q3-Q4 39.3% vs 7.1%, p=0.01; 40.0% vs 9.7%, p=0.01) (Figure 1). Nevertheless, the overall accuracy of the IBD disk (0.75) or SCCAI score (0.76) for histologic remission is lower (p<0.05) than the accuracy of the Mayo endoscopic (0.90) or UCEIS (0.90) score. Table 1: Baseline features Abstract OP09 – Figure 1: Quartile analysis Conclusion In patients with UC, PROMs for disability and clinical disease activity reflect histologic disease activity and should therefore be further explored in (trial) endpoint discussions. However, they cannot fully replace endoscopic and histologic findings, and should be considered complementary.

scholarly journals Patient-Reported Outcomes in Eosinophilic Esophagitis and Achalasia

2016 ◽  
Vol 14 (1) ◽  
pp. 51-60 ◽  
Author(s):  
Alain Schoepfer ◽  
Alex Straumann ◽  
Ekaterina Safroneeva
Keyword(s):  
Eosinophilic Esophagitis ◽  
Patient Reported Outcomes ◽  
Patient Reported

scholarly journals Systematic Review of Outcome Measures Used in Observational Studies of Adults with Eosinophilic Esophagitis

2021 ◽  
pp. 1-25
Author(s):  
Alain M. Schoepfer ◽  
Camilla Schürmann ◽  
Sven Trelle ◽  
Marcel Zwahlen ◽  
Christopher Ma ◽  
...  
Keyword(s):  
Systematic Review ◽  
Outcome Measures ◽  
Eosinophilic Esophagitis ◽  
Observational Studies ◽  
Patient Reported Outcomes ◽  
Case Series ◽  
Open Label ◽  
Topical Corticosteroids ◽  
Therapeutic Trials ◽  
Patient Reported

<b><i>Background:</i></b> Over the last 20 years, diverse outcome measures have been used to evaluate the effectiveness of therapies for eosinophilic esophagitis (EoE). This systematic review aims to identify the readouts used in observational studies of topical corticosteroids, diet, and dilation in adult EoE patients. <b><i>Methods:</i></b> We searched MEDLINE and Embase for prospective and retrospective studies (cohorts/case series, randomized open-label, and case-control) evaluating the use of diets, dilation, and topical corticosteroids in adults with EoE. Two authors independently assessed the articles and extracted information about histologic, endoscopic, and patient-reported outcomes and tools used to assess treatment effects. <b><i>Results:</i></b> We included 69 studies that met inclusion criteria. EoE-associated endoscopic findings (assessed either as absence/presence or using Endoscopic Reference Score) were evaluated in 24/35, 11/17, and 9/17 studies of topical corticosteroids, diet, and dilation, respectively. Esophageal eosinophil density was recorded in 32/35, 17/17, and 11/17 studies of topical corticosteroids, diet, and dilation, respectively. Patient-reported outcomes were not uniformly used (only in 14, 8, and 3 studies of topical corticosteroids, diet, and dilation, respectively), and most tools were not validated for use in adults with EoE. <b><i>Conclusions:</i></b> Despite the lack of an agreed set of core outcomes that should be recorded and reported in studies in adult EoE patients, endoscopic EoE-associated findings and esophageal eosinophil density are commonly used to assess disease activity in observational studies. Standardization of outcomes and data supporting the use of outcomes are needed to facilitate interpretation of evidence, its synthesis, and comparisons of interventions in meta-analyses of therapeutic trials in adults with EoE.

scholarly journals Aripiprazole lauroxil 2-month formulation with 1-day initiation in patients hospitalized for an acute exacerbation of schizophrenia: exploratory efficacy and patient-reported outcomes in the randomized controlled ALPINE study

2021 ◽  
Vol 21 (1) ◽  
Author(s):  
Henry A. Nasrallah ◽  
Peter J. Weiden ◽  
David P. Walling ◽  
Yangchun Du ◽  
Baiyun Yao ◽  
...  
Keyword(s):  
Quality Of Life ◽  
Acute Exacerbation ◽  
Caregiver Burden ◽  
Patient Reported Outcomes ◽  
Trial Registration ◽  
Symptom Improvement ◽  
Satisfaction Questionnaire ◽  
Randomized Controlled ◽  
Patient Reported

Abstract Background A randomized, controlled, phase 3b study (ALPINE) evaluated efficacy and safety of a 2-month formulation of aripiprazole lauroxil (AL) using a 1-day initiation regimen in patients hospitalized for an acute exacerbation of schizophrenia. Paliperidone palmitate (PP) was used as an active control. Exploratory endpoint assessments included severity of illness, positive and negative symptoms, quality of life, caregiver burden, and satisfaction with medication. Methods Adults were randomly assigned to AL 1064 mg q8wk or PP 156 mg q4wk as inpatients, discharged after 2 weeks, and followed through week 25. Exploratory efficacy measures included the 3 original PANSS subscales, Clinical Global Impression−Severity (CGI-S) subscale, and caregiver Burden Assessment Scale. Exploratory patient-reported outcomes (PROs) included the Quality of Life Enjoyment and Satisfaction Questionnaire Short Form (Q-LES-Q-SF) and the Medication Satisfaction Questionnaire. Within-group changes from baseline through week 25 were analyzed for AL and PP separately. PROs were summarized based on observed data. Results Of 200 patients randomized (AL, n = 99; PP, n = 101), 99 completed the study (AL, n = 56; PP, n = 43). For AL, PANSS subscale and CGI-S scores improved from baseline through week 25 (mean [SE] change from baseline at week 25: Positive, −7.5 [0.70]; Negative, −3.9 [0.46]; General, −11.8 [0.83]; CGI-S, −1.3 [0.12]). Caregiver burden also improved (mean [SD] changes from baseline at week 9: −8.4 [10.15]; week 25: −8.9 [12.36]). Most AL patients were somewhat/very satisfied with treatment at each timepoint (70.8%–74.7%); mean Q-LES-Q-SF total scores were stable in the outpatient period. For PP, results were similar: PANSS Positive, −7.3 (0.67); Negative, −3.6 (0.69); General, −10.9 (1.22); CGI-S, −1.4 (0.16); caregiver burden, week 9: −8.8 (11.89) and week 25: −9.2 (14.55); satisfaction with treatment, 64.7%–69.3%; and stable Q-LES-Q-SF scores. Conclusions ALPINE patients initiating the 2-month AL formulation using the 1-day initiation regimen as inpatients and continuing outpatient care experienced schizophrenia symptom improvement, sustained patient satisfaction with medication, stable quality of life, and reduced caregiver burden. A similar benefit pattern was observed for PP. These results support the feasibility of starting either long-acting injectable in the hospital and transitioning to outpatient treatment. Trial registration ClinicalTrials.gov identifier: NCT03345979 [trial registration date: 15/11/2017].

scholarly journals Discrepancies between patient-reported outcomes, and endoscopic and histological appearance in UC

Gut ◽  
2016 ◽  
Vol 66 (12) ◽  
pp. 2063-2068 ◽  
Author(s):  
Jean-Frédéric Colombel ◽  
Mary E Keir ◽  
Alexis Scherl ◽  
Rui Zhao ◽  
Gert de Hertogh ◽  
...  
Keyword(s):  
Patient Reported Outcomes ◽  
Symptomatic Relief ◽  
Mucosal Healing ◽  
Cytokine Expression ◽  
Inactive Disease ◽  
Faecal Calprotectin ◽  
Inflammatory Biomarker ◽  
Patient Reported ◽  
Histological Remission ◽  
Sensitivity Specificity

ObjectiveBoth endoscopy and histology may be included in the definition of mucosal healing in UC. This study aimed to establish the association between patient-reported outcomes, specifically symptom measures, and the presence of inflammation as measured by endoscopy and histology in UC.DesignUsing patient data from an observational multicentre study of UC (n=103), rectal bleeding (RB) and stool frequency (SF) symptom subscores of the Mayo Clinic Score (MCS) were compared with the endoscopic subscore (MCSe) and histology. Faecal calprotectin and biopsy cytokine expression were also evaluated.ResultsWhen identifying UC patients with inactive disease, RB scores were superior to SF scores and the combination (sensitivity/specificity: MCSe=0/1, RB 77%/81%, SF 62%/95%, RB+SF 54%/95%; MCSe=0, RB 87%/66%, SF 76%/83%, RB+SF 68%/86%). Across different definitions of mucosal healing (MCSe≤1; 0; or 0 plus inactive histology), a larger subset of patients reported increased SF (39%, 25% and 27%, respectively) compared with RB (24%, 13% and 10%). Faecal calprotectin and inflammatory cytokine expression were higher in patients with active disease compared with patients with mucosal healing, but there were no differences between patients using increasingly stringent definitions of mucosal healing.ConclusionsEndoscopically inactive disease is associated with absence of RB but not with complete normalisation of SF. Achieving histological remission did not improve symptomatic relief. In addition, in these patients, higher inflammatory biomarker levels were not observed. These data suggest that non-inflammatory changes, such as bowel damage, may contribute to SF in UC.

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