Validation of phone interview for follow-up in clinical trials on dyspepsia: evaluation of the Glasgow Dyspepsia Severity Score and a Likert-scale symptoms test

2000 ◽  
Vol 12 (8) ◽  
pp. 949-953 ◽  
Author(s):  
Xavier Calvet ◽  
Eva Bustamante ◽  
Antonia Montserrat ◽  
Marta Roqué ◽  
Rafel Campo ◽  
...  
Keyword(s):  
Clinical Trials ◽  
Severity Score ◽  
Likert Scale ◽  
Phone Interview

Skin improvement is a surrogate for favourable changes in other organ systems in early diffuse cutaneous systemic sclerosis

Rheumatology ◽  
2019 ◽  
Vol 59 (7) ◽  
pp. 1715-1724 ◽  
Author(s):  
Tatiana Nevskaya ◽  
Boyang Zheng ◽  
Carl A Baxter ◽  
Dena R Ramey ◽  
Janet E Pope ◽  
...  
Keyword(s):  
Clinical Trials ◽  
Disease Severity ◽  
Research Group ◽  
Severity Score ◽  
Vital Capacity ◽  
Organ Involvement ◽  
Disease Severity Score ◽  
Skin Changes ◽  
Organ Systems

Abstract Objectives Skin improvement in diffuse cutaneous SSc (dcSSc), measured with modified Rodnan skin score (mRSS), is frequently used as a primary outcome in clinical trials, but it is uncertain whether mRSS changes reflect changes in other organ systems. This aim of this study was to explore if skin changes in early dcSSc over 1 and 2 years are associated with changes in severity of other organ involvement. Methods Canadian Scleroderma Research Group database patients with dcSSc, disease duration of ≤5 years, no evidence of initial end-stage organ damage and/or significant comorbidity who had 1 year (n = 154) and 2 years (n = 128) of follow-up data were included. mRSS changes of 25% and/or ≥5 points were considered significant. Organ involvement was assessed by Medsger Disease Severity Score and Canadian Scleroderma Research Group definitions using bivariate, chi-square, ANOVA, adjusted regression and longitudinal mixed effect model analyses. Results Improvement in mRSS was found in 41% of patients at 1 year and in 50% at 2 years. Improved patients showed less forced vital capacity decline (P = 0.012) and less frequent new cardiac involvement (P = 0.02) over 1 year, as well as better lung (by both Disease Severity Score, P = 0.006, and Δforced vital capacity%, P = 0.026), peripheral vascular (P = 0.006) and joint/tendon (P = 0.002) involvement over 2 years. mRSS worsening was consistently linked to less favourable lung outcomes at both 1- and 2-year follow-up visits, and more severe gastrointestinal disease at 2 years. Conclusion Changes in lung function in early dcSSc closely parallel skin changes. mRSS improvement reflects better prognosis for visceral disease and may be a reliable outcome measure in clinical trials.

Independent Association of a Metabolic Syndrome Severity Score with All-Cause Mortality in Type 1 Diabetes—A 10-Year Follow-Up

Diabetes ◽  
2018 ◽  
Vol 67 (Supplement 1) ◽  
pp. 618-P
Author(s):  
GIUSEPPE PENNO ◽  
MONIA GAROFOLO ◽  
ROSA GIANNARELLI ◽  
FABRIZIO CAMPI ◽  
DANIELA LUCCHESI ◽  
...  
Keyword(s):  
Metabolic Syndrome ◽  
Type 1 Diabetes ◽  
Severity Score ◽  
All Cause Mortality ◽  
Independent Association

453-P: Metabolic Syndrome Severity Score, All-Cause Mortality, and Incident Vascular Events in Type 2 Diabetes: A 13-Year, Single-Centre, Follow-Up Study

Diabetes ◽  
2019 ◽  
Vol 68 (Supplement 1) ◽  
pp. 453-P
Author(s):  
MONIA GAROFOLO ◽  
ELISA GUALDANI ◽  
DANIELA LUCCHESI ◽  
LAURA GIUSTI ◽  
VERONICA SANCHO-BORNEZ ◽  
...  
Keyword(s):  
Type 2 Diabetes ◽  
Metabolic Syndrome ◽  
Severity Score ◽  
Single Centre ◽  
Vascular Events ◽  
Follow Up Study ◽  
All Cause Mortality

scholarly journals Real-World Experience with Bezlotoxumab for Prevention of Recurrence of Clostridioides difficile Infection

2020 ◽  
Vol 10 (1) ◽  
pp. 2
Author(s):  
Rosa Escudero-Sánchez ◽  
María Ruíz-Ruizgómez ◽  
Jorge Fernández-Fradejas ◽  
Sergio García Fernández ◽  
María Olmedo Samperio ◽  
...  
Keyword(s):  
Clinical Trials ◽  
Real World ◽  
Recurrence Rates ◽  
Factors Associated ◽  
Clostridioides Difficile ◽  
Multicentre Cohort Study ◽  
Clostridioides Difficile Infection ◽  
Prevention Of Recurrence ◽  
Multicentre Cohort

Bezlotoxumab is marketed for the prevention of recurrent Clostridioides difficile infection (rCDI). Its high cost could be determining its prescription to a different population than that represented in clinical trials. The objective of the study was to verify the effectiveness and safety of bezlotoxumab in preventing rCDI and to investigate factors related to bezlotoxumab failure in the real world. A retrospective, multicentre cohort study of patients treated with bezlotoxumab in Spain was conducted. We compared the characteristics of cohort patients with those of patients treated with bezlotoxumab in the pivotal MODIFY trials. We assessed recurrence rates 12 weeks after completion of treatment against C. difficile, and we analysed the factors associated with bezlotoxumab failure. Ninety-one patients were included in the study. The cohort presented with more risk factors for rCDI than the patients included in the MODIFY trials. Thirteen (14.2%) developed rCDI at 12 weeks of follow-up, and rCDI rates were numerically higher in patients with two or more previous episodes (25%) than in those who had fewer than two previous episodes of C. difficile infection (CDI) (10.4%); p = 0.09. There were no adverse effects attributable to bezlotoxumab. Despite being used in a more compromised population than that represented in clinical trials, we confirm the effectiveness of bezlotoxumab for the prevention of rCDI.

scholarly journals Disability improvement as a clinically relevant outcome in clinical trials of relapsing forms of multiple sclerosis

2021 ◽  
pp. 135245852110002
Author(s):  
Bruce AC Cree ◽  
Jeffrey A Cohen ◽  
Anthony T Reder ◽  
Davorka Tomic ◽  
Diego Silva ◽  
...  
Keyword(s):  
Multiple Sclerosis ◽  
Clinical Trials ◽  
Therapeutic Benefit ◽  
Disease Modifying Therapies ◽  
Long Term Follow Up ◽  
Post Hoc ◽  
Switch Group ◽  
Relevant Outcome

Background: Disease-modifying therapies (DMTs) can reduce the risk of disability worsening in patients with relapsing forms of multiple sclerosis (RMS). High-efficacy DMTs can lead to confirmed or sustained disability improvement (CDI and SDI). Objective and Methods: Post hoc analyses of data from the TRANSFORMS, FREEDOMS, and FREEDOMS II trials and their extensions assessed the effects of fingolimod (0.5–1.25 mg/day) on stabilizing or improving disability over ⩽8 years in participants with RMS. CDI and SDI rates were compared between participants initially randomized to fingolimod, interferon (IFNβ-1a), or placebo. Results: At 8 years’ follow-up in TRANSFORMS, 35.1% (95% confidence interval [CI], 28.2%–43.1%) of assessed participants in the IFNβ-1a–fingolimod switch group and 41.9% (36.6%–47.6%) on continuous fingolimod experienced CDI; disability did not worsen in approximately 70%. Similar results were seen in the combined FREEDOMS population. Proportionally fewer TRANSFORMS participants achieved SDI in the IFNβ-1a–fingolimod switch group than on continuous fingolimod (5.4% [3.0%–9.5%] vs 14.2% [10.8%–18.4%], p = 0.01). Conclusion: CDI and SDI are outcomes of interest for clinical trials and for long-term follow-up of participants with RMS. Monitoring CDI and SDI in addition to disability worsening may facilitate understanding of the therapeutic benefit of RMS treatments.

Patients with shoulder pain referred to specialist care; treatment, predictors of pain and disability, emotional distress, main symptoms and sick-leave: a cohort study with a six-months follow-up

2020 ◽  
Vol 20 (4) ◽  
pp. 775-783
Author(s):  
Kaia B. Engebretsen ◽  
Jens Ivar Brox ◽  
Niels Gunnar Juel
Keyword(s):  
Clinical Trials ◽  
Cohort Study ◽  
Sick Leave ◽  
Shoulder Pain ◽  
Emotional Distress ◽  
Outpatient Clinic ◽  
Treatment Duration ◽  
Specialist Care ◽  
Yellow Flags

AbstractObjectivesRecommendations for referral of patients with shoulder pain from primary to specialist care are mainly clinical. Several patients are referred without meeting these criteria for referral, whereas some are referred for a second opinion although surgery is not recommended. The aims of this study were to describe a shoulder pain cohort in specialist healthcare according to demographic data, clinical, and psychological factors; evaluate changes in pain and disability, distress and main symptoms from baseline to six-month follow-up; and to assess predictors of pain and disability, changes in the main symptoms and sick-leave at six-months. Results were compared to previous randomised trials conducted at the same clinic in patients with subacromial shoulder pain.MethodsThis prospective study included 167 patients from an outpatient clinic in specialist healthcare with shoulder pain for more than 6 weeks. Clinical (pain duration, intensity, pain sites), sociodemographic (age, gender, educational level, work status) and psychological variables (emotional distress (HSCL-10), fear of pain, screening of “yellow flags”, health-related quality of life) were collected. Shoulder pain and disability (SPADI-score) were assessed and the patients were asked about their outcome expectation and to predict their status of their shoulder problem the next month. They underwent a clinical interview, a clinical assessment of shoulder function and orthopaedic tests for diagnostic purposes. After six months they received a questionnaire with main variables.ResultsOf the 167 patients (55% women), 50% had symptoms for more than 12 months and 37 (22%) were on sick-leave. Characteristics were in general comparable to patients previously included in clinical trials at the same department. The SPADI-score was 46 (23) points. Mean emotional distress was within the normal range (1.7 (SD 0.6)). More than 80% had received treatment before, mainly physiotherapy in addition to the GPs treatment. One hundred and thirty-seven patients (82%) were re-referred to physiotherapy, 74 (44%) in the outpatient clinic specialist healthcare, and 63 (38%) in primary care. One hundred and eighteen (71%) answered the follow-up questionnaire. Mean change in SPADI-score was 10.5 points (95% CI (6.5–14.5)), and 29% of the patients improved more than the smallest detectable difference (SDD). The percentage sick-listed was 19.5%, and mean change in main symptoms (−9 to +9) was 3.4 (SD 3.9). The subgroup of patients receiving physiotherapy in outpatient specialist care did not show any significant change in the main variables. The prediction models suggested that a lower level of education, more fear of pain and a high baseline SPADI-score, predicted a higher SPADI-score at follow-up. A high baseline HSCL-10 score was the only significant predictor for a high HSCL-10 score. At follow-up, less pain at rest predicted more change in main symptoms and more yellow flags (a higher score on the Örebro screening test) predicted sick-leave.ConclusionsWithin the limitations of a cohort study, patients with persistent shoulder pain referred to an outpatient specialist clinic had similar baseline characteristics but shorter treatment duration, inferior clinical results and predictors somewhat different compared with previous clinical trials conducted at the same clinic. The study raises some questions about the effectiveness of the routines in daily clinical practice, the selection of patients, the treatment duration and content.

scholarly journals AB1065 VISIT COMPLIANCE IN PATIENTS WITH FAMILIAL MEDITERRANEAN FEVER: RESULTS FROM A GAZI UNIVERSITY FMF COHORT

2020 ◽  
Vol 79 (Suppl 1) ◽  
pp. 1822.1-1822
Author(s):  
R. Bilici Salman ◽  
A. Avanoğlu Güler ◽  
H. Satiş ◽  
H. Karadeniz ◽  
H. Babaoglu ◽  
...  
Keyword(s):  
Logistic Regression ◽  
Family History ◽  
Familial Mediterranean Fever ◽  
Predictive Factors ◽  
Severity Score ◽  
Comorbid Disease ◽  
Rheumatology Clinic ◽  
Mediterranean Fever ◽  
One Year

Background:Follow-up in all rheumatologic patients is critical, particularly Familial Mediterranean Fever (FMF). Current recommendations for all experts by the EULAR state that patients with FMF should be evaluated 6-monthly intervals to monitore the character and frequency of the attacks and the acute phase response. Disease-related complications such as amyloidosis can beasymptomaticand need only a careful follow-up.Objectives:to quantify this phenomenon and to find predictive factors of visit compliance in patients with FMF.Methods:The study included 474 adult patients with a diagnosis of FMF who followed at the outpatient rheumatology clinic of tertiary university hospital, from January 2018 to December 2018. . Demographic, socioeconomic data, familiy history, comorbid disease, medication history, characteristics, the International Severity Score for FMF (ISSF),autoinflammatory disease damage index (ADDI) were recorded. Visit compliance was defined as the presence of two visits in the outpatient rheumatology clinic for FMF last one year for the purposes set out in EULAR suggestion.Those who had fewer than two visits in the last one year were considered noncompliant.Results:230 (48.5%) were compliant while 244 (51.5 %) patients were noncompliant with their rheumatology visit. Both compliant and noncompliant patients had similar median age and disease duration. Female sex and being married was increased the visit compliance.The results of the logistic regression model exploring factors associated with compliance indicated that presence of family history in parents, absence of family history in sibling, treatment with biologic agents, other drug using,presence of more than 2 attacks except fever and adequate medical care were important predictors of visit compliance.Conclusion:In conclusion, if FMF patients visit compliance increase, their functionality, medication adherence and quality of life will increase and flares and complication of disease can decrease. Thus, we highlight some recommendations for FMF specialist, patients and health care providers to improve outcomes.Table 2.Multivariate logistic regression analysis for predictive factors of visit compliance of the patients with FMF, n=430Adj. OR%95 CI**pFamily history in parents(positive history vs negative)1,81,0-3,10.03Family history in sibling(negative history vs positive)1,91,2-3,10.004Comorbid disease status1,30,7-2,50.32Treatment(anakinra&canakinumab vs colchicine)3,71,7-8,20.001Drug using(other drugs vs FMF drugs)2,21,1-4,40.01More than 2 attacks except fever2,31,2-4,00.004Chronic peripheral arthritis2,30,8-6,60.10Proteinuria2,20,7-6,70.14Adequate medical care1,91,2-3,10.003Number of index flare within last 12-month0,90,9-1,00.38ISSF severity score0,80,7-1,10,30Disclosure of Interests:None declared

scholarly journals DNA Methylation at ATP11A cg11702988 Is a Biomarker of Lung Disease Severity in Cystic Fibrosis: A Longitudinal Study

Genes ◽  
2021 ◽  
Vol 12 (3) ◽  
pp. 441
Author(s):  
Fanny Pineau ◽  
Davide Caimmi ◽  
Sylvie Taviaux ◽  
Maurane Reveil ◽  
Laura Brosseau ◽  
...  
Keyword(s):  
Cystic Fibrosis ◽  
Dna Methylation ◽  
Clinical Trials ◽  
Lung Disease ◽  
Disease Severity ◽  
Genome Wide ◽  
A Genome ◽  
Lung Disease Severity ◽  
Epigenetic Biomarker

Cystic fibrosis (CF) is a chronic genetic disease that mainly affects the respiratory and gastrointestinal systems. No curative treatments are available, but the follow-up in specialized centers has greatly improved the patient life expectancy. Robust biomarkers are required to monitor the disease, guide treatments, stratify patients, and provide outcome measures in clinical trials. In the present study, we outline a strategy to select putative DNA methylation biomarkers of lung disease severity in cystic fibrosis patients. In the discovery step, we selected seven potential biomarkers using a genome-wide DNA methylation dataset that we generated in nasal epithelial samples from the MethylCF cohort. In the replication step, we assessed the same biomarkers using sputum cell samples from the MethylBiomark cohort. Of interest, DNA methylation at the cg11702988 site (ATP11A gene) positively correlated with lung function and BMI, and negatively correlated with lung disease severity, P. aeruginosa chronic infection, and the number of exacerbations. These results were replicated in prospective sputum samples collected at four time points within an 18-month period and longitudinally. To conclude, (i) we identified a DNA methylation biomarker that correlates with CF severity, (ii) we provided a method to easily assess this biomarker, and (iii) we carried out the first longitudinal analysis of DNA methylation in CF patients. This new epigenetic biomarker could be used to stratify CF patients in clinical trials.

scholarly journals SO093LONGITUDINAL DATA ON TREATMENT DURATION AND COMPLIANCE FROM AUTOSOMAL DOMINANT POLYCYSTIC KIDNEY DISEASE CLINICAL TRIALS WITH TOLVAPTAN

2020 ◽  
Vol 35 (Supplement_3) ◽  
Author(s):  
Xiaolei Zhou ◽  
Diana Garbinsky ◽  
John Ouyang ◽  
Eric Davenport ◽  
Indra Agarwal ◽  
...  
Keyword(s):  
Clinical Trial ◽  
Clinical Trials ◽  
Treatment Duration ◽  
Treatment Time ◽  
Treatment Compliance ◽  
Kidney Volume ◽  
Total Kidney Volume ◽  
Treatment Gaps ◽  
Clinical Trial Program

Abstract Background and Aims : Observation of impactful clinical outcomes in a clinical trial setting for ADPKD is challenging due to the life-long progressive nature of ADPKD and longer-term associated outcomes of interest in this population (e.g., renal function decline, cardiovascular events, and mortality). Since 2004, the tolvaptan (TOL) clinical trial program enrolled subjects in multiple clinical studies with the opportunity to enroll in subsequent clinical trials for treatment and outcomes evaluation. Method : Data from 6 ADPKD studies (protocols 156-04-250, 156-04-251, 156-06-260, 156-09-284, 156-09-290, 156-08-271) were pooled and evaluated over time for overall treatment duration, treatment time, and treatment gaps. Treatment duration for the individual clinical trials ranged from 1 week to up to 3 years. Results : Overall, 1,437 subjects received TOL in these ADPKD clinical trials. For these subjects, the mean overall treatment duration was 4.1 years (3.8 years on treatment) with a maximum of 9.7 years (9.0 years on treatment). In this cohort, 513 subjects (35.7%) received TOL treatment for more than 5 years. Mean treatment compliance was 94.1%. Overall, 723 subjects (50.3%) received TOL treatment in ≥2 trials, with a median treatment gap duration between trials of 0.1 years (maximum, 5.6 years). At least 7 years of follow-up data are available for estimated glomerular filtration rate in 241 subjects (mean at baseline, 78.6 mL/min/1.73m2) and for total kidney volume in 130 subjects (mean at baseline, 1,816.9 mL). Conclusion : This analysis provides longitudinal follow-up over an extended timeframe in a large number of subjects treated with TOL, with the greatest number of subjects being enrolled in clinical trials enriched for rapidly progressing ADPKD. Treatment compliance over years was reasonably good despite treatment gaps.

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