scholarly journals Development and validation of the Scleroderma Clinical Trials Consortium Damage Index (SCTC-DI): a novel instrument to quantify organ damage in systemic sclerosis

2019 ◽  
Vol 78 (6) ◽  
pp. 807-816 ◽  
Author(s):  
Nava Ferdowsi ◽  
Molla Huq ◽  
Wendy Stevens ◽  
Marie Hudson ◽  
Mianbo Wang ◽  
...  
Keyword(s):  
Clinical Trials ◽  
Systemic Sclerosis ◽  
Short Form ◽  
Damage Index ◽  
Clinical Importance ◽  
Organ Damage ◽  
Physical Component Summary Score ◽  
Summary Score ◽  
Data Set ◽  
Mortality And Morbidity

ObjectiveWe sought to develop the first Damage Index (DI) in systemic sclerosis (SSc).MethodsThe conceptual definition of ‘damage’ in SSc was determined through consensus by a working group of the Scleroderma Clinical Trials Consortium (SCTC). Systematic literature review and consultation with patient partners and non-rheumatologist experts produced a list of potential items for inclusion in the DI. These steps were used to reduce the items: (1) Expert members of the SCTC (n=331) were invited to rate the appropriateness of each item for inclusion, using a web-based survey. Items with >60% consensus were retained; (2) Using a prospectively acquired Australian cohort data set of 1568 patients, the univariable relationships between the remaining items and the endpoints of mortality and morbidity (Physical Component Summary score of the Short Form 36) were analysed, and items with p<0.10 were retained; (3) using multivariable regression analysis, coefficients were used to determine a weighted score for each item. The DI was externally validated in a Canadian cohort.ResultsNinety-three (28.1%) complete survey responses were analysed; 58 of 83 items were retained. The univariable relationships with death and/or morbidity endpoints were statistically significant for 22 items, with one additional item forced into the multivariable model by experts due to clinical importance, to create a 23-item weighted SCTC DI (SCTC-DI). The SCTC-DI was predictive of morbidity and mortality in the external cohort.ConclusionsThrough the combined use of consensus and data-driven methods, a 23-item SCTC-DI was developed and retrospectively validated.

scholarly journals Outcomes following operatively managed acetabular fractures in patients aged 60 years and older

2020 ◽  
Vol 102-B (12) ◽  
pp. 1735-1742
Author(s):  
Pierre Navarre ◽  
Belinda J. Gabbe ◽  
Xavier L. Griffin ◽  
Matthias K. Russ ◽  
Andrew T. Bucknill ◽  
...  
Keyword(s):  
Short Form ◽  
Physical Component Summary Score ◽  
Summary Score ◽  
Acetabular Fractures ◽  
Royal Melbourne Hospital ◽  
Mortality And Morbidity ◽  
Trauma Outcomes ◽  
Patient Interviews ◽  
Risk Of Mortality ◽  
High Conversion Rate

Aims Acetabular fractures in older adults lead to a high risk of mortality and morbidity. However, only limited data have been published documenting functional outcomes in such patients. The aims of this study were to describe outcomes in patients aged 60 years and older with operatively managed acetabular fractures, and to establish predictors of conversion to total hip arthroplasty (THA). Methods We conducted a retrospective, registry-based study of 80 patients aged 60 years and older with acetabular fractures treated surgically at The Alfred and Royal Melbourne Hospital. We reviewed charts and radiological investigations and performed patient interviews/examinations and functional outcome scoring. Data were provided by the Victorian Orthopaedic Trauma Outcomes Registry (VOTOR). Survival analysis was used to describe conversion to THA in the group of patients who initially underwent open reduction and internal fixation (ORIF). Multivariate regression analyses were performed to identify factors associated with conversion to THA. Results Seven patients (8.8%) had died at a median follow-up of 18 months (interquartile range (IQR) 12 to 25), of whom four were in the acute THA group. Eight patients (10%) underwent acute THA. Of the patients who underwent ORIF, 17/72 (23.6%) required conversion to THA at a median of 10.5 months (IQR 4.0 to 32.0) . After controlling for other factors, transport-related cases had an 88% lower rate of conversion to THA (hazard ratio (HR) 0.12, 95% confidence interval (CI) 0.02 to 0.91). Mean standardized Physical Component Summary Score (PCS-12) of the 12-Item Short Form Health Survey (SF-12) was comparable with the general population (age-/sex-matched) by 12 to 24 months. Over half of patients working prior to injury (14/26) returned to work by six months and two-thirds of patients (19/27) by 12 months. Conclusion Patients over 60 years of age managed operatively for displaced acetabular fractures had a relatively high mortality rate and a high conversion rate to THA in the ORIF group but, overall, patients who survived had mean PCS-12 scores that improved over two years and were comparable with controls. Cite this article: Bone Joint J 2020;102-B(12):1735–1742.

scholarly journals OP0273 THE SCLERODERMA CLINICAL TRIALS CONSORTIUM DAMAGE INDEX (SCTC-DI) IN A SYSTEMIC SCLEROSIS COHORT WITH 10-YEARS FOLLOW-UP

2021 ◽  
Vol 80 (Suppl 1) ◽  
pp. 166.2-166
Author(s):  
M. G. Lazzaroni ◽  
M. Breda ◽  
F. Franceschini ◽  
P. Airò
Keyword(s):  
Clinical Trials ◽  
Systemic Sclerosis ◽  
Multivariable Analysis ◽  
Damage Index ◽  
Organ Damage ◽  
Severe Damage ◽  
Time Points ◽  
Cutaneous Involvement ◽  
Male Sex

Background:Systemic Sclerosis (SSc) is characterized by increased mortality and organ damage accrual. A composite SSc Damage Index was recently developed by the Scleroderma Clinical Trials Consortium (SCTC-DI) and was demonstrated as a predictor of mortality both in the Australian derivation cohort and in the Canadian validation cohort.Objectives:To evaluate in a single centre cohort of SSc patients with 10-years follow-up: (1) the evolution of organ damage over time; (2) factors associated with the development and accrual of organ damage.Methods:A retrospective analysis was performed on patients prospectively followed in our centre from 1989 to 2019. Organ damage was evaluated with SCTC-DI (0-55 scale; moderate damage >5, severe damage>12) and comorbidities with Charlson Comorbidity Index (CCI, which includes the age of the patient). Patients were included when a follow-up of at least 10 years was available together with SCTC-DI at the diagnosis (baseline, T0), 1 year (T1), 5 years (T5) and 10 years (T10) after the diagnosis. Univariable and multivariable analysis (logistic regression) were performed when appropriated.Results:253 SSc patients were included (female 93%; Caucasians: 99%; median age at diagnosis: 52 years (IQR: 43-60); diffuse cutaneous subset: 15%; anti-centromere (ACA)+ 55%; anti-Topoisomerase 1 + 20%; anti-RNA polymerase III+: 4%; ever smokers: 28%). Median interval between the first SSc symptom other than Raynaud’s phenomenon and the diagnosis was 1 year. SCTC-DI progressively increased from diagnosis to T10 (p<0.0001; Kruskal-Wallis test).Moderate damage (score:6-12) was observed in 22 patients at T0 (8.7%), in 30 at T1 (11.9%), in 45 at T5 (17.7%) and in 73 at T10 (28.9%). None of the patients had severe damage (score:13-55) at T0 and T1, while it was present in 6 at T5 (2.4%) and in 13 at T10 (5.1%).At T0 no difference in SCTC-DI scores was observed when comparing different subgroups according to gender (female vs. male), disease subsets (diffuse vs. limited) and autoantibodies (ACA- vs. ACA+). At T1, SCTC-DI score was higher in patients with diffuse vs. limited cutaneous subset, and ACA- vs ACA+ (p<0.0001 for both).Multivariable analysis demonstrated that a moderate or severe organ damage (SCTC-DI score >5) at 5 years was positively associated with diffuse cutaneous involvement (p:0.009, OR 4.55, 1.46-14.1), SCTC-DI at T0 (p:0.015, OR 1.34, 1.06-1.70) and at T1 (p:<0.0001, OR 1.65, 1.30-2.07), and negatively associated with ACA+ (p:0.024, OR 0.32, 0.12-0.86), while CCI and male sex showed no association. At 10 years SCTC-DI>5 was associated with diffuse cutaneous involvement (p:0.013, OR 4.30, 1.36-13.7), SCTC-DI at T5 (p:<0.0001, OR 1.67, 1.38-2.01), while SCTC-DI at T0, CCI, male sex and ACA+ had no association.Among 253 patients, 90 (36%) died after >10 years of follow-up. In non-survivors, as compared to survivors, SCTC-DI score was significantly higher at the baseline (T0) and during the entire follow-up (p<0.0001 for every timepoint).Conclusion:At the end of 10-years follow-up (T10), 35% of patients in our cohort had moderate or severe organ damage (SCTC-DI score>5). Diffuse cutaneous involvement was associated with higher SCTC-DI scores at different time points (T5 and T10). Organ damage, quantified by SCTC-DI at different time points, was confirmed as a factor associated with mortality in patients who reached more than 10 years of follow-up.References:[1]Ferdowsi N, et al. Ann Rheum Dis. 2019;78:807-16.Disclosure of Interests:None declared

Compliance of subjects using electronic patient-reported outcomes (ePRO) in multinational prostate trials.

2014 ◽  
Vol 32 (4_suppl) ◽  
pp. 232-232
Author(s):  
Susan M. Dallabrida
Keyword(s):  
Quality Of Life ◽  
Prostate Cancer ◽  
Clinical Trials ◽  
Patient Reported Outcomes ◽  
Short Form ◽  
Quality Data ◽  
Data Set ◽  
Patient Reported ◽  
Clinic Visits

232 Background: Patient Reported Outcomes (PRO) and electronic PRO (ePRO) are increasingly becoming an important aspect of cancer clinical trials and patient care, especially with regard to measuring drug efficacy, patient quality of life and drug safety. Subject compliance with completion of PRO/ePRO assessments is an important component for obtaining accurate and high-quality data when conducting clinical trials. It has been hypothesized that patient health status, length of time in a trial and country of origin, may affect compliance. Methods: To address this hypothesis, an operational analysis was conducted to assess oncology subject completion compliance of PRO reports using an electronic tablet to determine its suitability and performance in use. Toward this objective, the compliance of prostate cancer patients in completing three electronic questionnaires that were administered at clinic visits was evaluated. Subjects were asked to complete the Brief Pain Inventory – Short Form (BPI-SF) at every clinic visit. At some clinic visits, subjects were asked to additionally complete the Functional Assessment of Cancer Therapy – Prostate (FACT-P) and the Euro Quality of Life 5 Dimensions (EQ-5D). Questionnaires were completed electronically on the tablet. Percent completion was calculated as the number of questionnaires completed divided by the number of questionnaires expected, based on attended clinic visits compiled for this review and the administration schedule for the questionnaires. Results: This review draws on the experience of over 1,000 subjects from 21 countries, and describes the individual and overall compliance with the expected questionnaire completion, the variance between subsequent visits, and compliance by country. Conclusions: The collection of ePRO using a clinic-based tablet yielded a highly complete data set in prostate cancer subjects demonstrating that this is an effective and feasible approach for recording symptoms and quality of life assessments.

scholarly journals Systemic Sclerosis Pathogenesis and Emerging Therapies, beyond the Fibroblast

2019 ◽  
Vol 2019 ◽  
pp. 1-15 ◽  
Author(s):  
Andrea Sierra-Sepúlveda ◽  
Alexia Esquinca-González ◽  
Sergio A. Benavides-Suárez ◽  
Diego E. Sordo-Lima ◽  
Adrián E. Caballero-Islas ◽  
...  
Keyword(s):  
Clinical Trials ◽  
Immune System ◽  
Systemic Sclerosis ◽  
Autoimmune Disease ◽  
State Of The Art ◽  
Therapeutic Targets ◽  
Organ Damage ◽  
The State ◽  
Pathogenic Mechanism ◽  
Emerging Therapies

Systemic sclerosis (SSc) is a complex rheumatologic autoimmune disease in which inflammation, fibrosis, and vasculopathy share several pathogenic pathways that lead to skin and internal organ damage. Recent findings regarding the participation and interaction of the innate and acquired immune system have led to a better understanding of the pathogenesis of the disease and to the identification of new therapeutic targets, many of which have been tested in preclinical and clinical trials with varying results. In this manuscript, we review the state of the art of the pathogenesis of this disease and discuss the main therapeutic targets related to each pathogenic mechanism that have been discovered so far.

scholarly journals Predictors of disease worsening defined by progression of organ damage in diffuse systemic sclerosis: a European Scleroderma Trials and Research (EUSTAR) analysis

2019 ◽  
Vol 78 (9) ◽  
pp. 1242-1248 ◽  
Author(s):  
Mike Becker ◽  
Nicole Graf ◽  
Rafael Sauter ◽  
Yannick Allanore ◽  
John Curram ◽  
...  
Keyword(s):  
Clinical Trials ◽  
Systemic Sclerosis ◽  
Disease Progression ◽  
Muscle Weakness ◽  
Lung Fibrosis ◽  
Heart Function ◽  
Strong Association ◽  
Organ Damage ◽  
Inclusion Criteria ◽  
Patients At Risk

ObjectivesMortality and worsening of organ function are desirable endpoints for clinical trials in systemic sclerosis (SSc). The aim of this study was to identify factors that allow enrichment of patients with these endpoints, in a population of patients from the European Scleroderma Trials and Research group database.MethodsInclusion criteria were diagnosis of diffuse SSc and follow-up over 12±3 months. Disease worsening/organ progression was fulfilled if any of the following events occurred: new renal crisis; decrease of lung or heart function; new echocardiography-suspected pulmonary hypertension or death. In total, 42 clinical parameters were chosen as predictors for the analysis by using (1) imputation of missing data on the basis of multivariate imputation and (2) least absolute shrinkage and selection operator regression.ResultsOf 1451 patients meeting the inclusion criteria, 706 had complete data on outcome parameters and were included in the analysis. Of the 42 outcome predictors, eight remained in the final regression model. There was substantial evidence for a strong association between disease progression and age, active digital ulcer (DU), lung fibrosis, muscle weakness and elevated C-reactive protein (CRP) level. Active DU, CRP elevation, lung fibrosis and muscle weakness were also associated with a significantly shorter time to disease progression. A bootstrap validation step with 10 000 repetitions successfully validated the model.ConclusionsThe use of the predictive factors presented here could enable cohort enrichment with patients at risk for overall disease worsening in SSc clinical trials.

Dissociation Between Clinical Benefit and Persistent Urate Lowering in Patients with Chronic Refractory Gout Treated with Pegloticase

2019 ◽  
Vol 47 (4) ◽  
pp. 605-612 ◽  
Author(s):  
Michael H. Pillinger ◽  
Theodore R. Fields ◽  
Anthony E. Yeo ◽  
Peter E. Lipsky
Keyword(s):  
Clinical Benefit ◽  
Short Form ◽  
Serum Urate ◽  
Physical Component Summary Score ◽  
Summary Score ◽  
Medical Outcomes ◽  
Link Type ◽  
Patient Reported ◽  
Clinical Benefits ◽  
Specific Health

Objective.To assess clinical benefit in patients with chronic refractory gout who did not meet the protocol-defined criteria of responders to pegloticase.Methods.This analysis used results from 2 randomized controlled trials (ClinicalTrials.gov: NCT00325195, NCT01356498) to assess the clinical efficacy in responders and nonresponders to treatment (8 mg of pegloticase every 2 weeks). Serum urate was measured before each infusion and the following were recorded: assessment of gout flares, tophus reduction, patient’s global assessment (PtGA), tender and swollen joints (TJC and SJC), pain using a 100-mm visual analog scale, and a variety of patient-reported outcomes [Medical Outcomes Study Short Form-36 questionnaire physical component summary score and arthritis-specific health index (ASHI) score].Results.The analysis included 36 persistent urate responders, 49 nonresponders, and 43 patients who received placebo. Results for both responders and nonresponders indicated significant reduction in tophi and improvements from baseline in PtGA, TJC, SJC, pain, and ASHI. No significant improvements were observed in the patients who received placebo.Conclusion.Chronic refractory gout patients not achieving protocol-defined persistent urate lowering still achieve significant clinical benefits with pegloticase treatment, suggesting that transient reduction in serum urate may result in sustained clinical benefit.

Cotyloid Fossa Coverage Percentages May Be Associated With Alpha Angle, Labral Tear, and Clinical Outcomes in Patients With Femoroacetabular Impingement

2021 ◽  
pp. 036354652110569
Author(s):  
Yoichi Murata ◽  
Lauren Pierpoint ◽  
Madeleine DeClercq ◽  
Carly Lockard ◽  
Maitland Martin ◽  
...  
Keyword(s):  
Femoroacetabular Impingement ◽  
Physical Component Summary ◽  
Short Form ◽  
Alpha Angle ◽  
Patient Characteristics ◽  
Physical Component Summary Score ◽  
Summary Score ◽  
Labral Tear ◽  
Patient Reported ◽  
Tear Size

Background: Within the hip joint, the anatomy of the acetabulum and cotyloid fossa is well established. There is little literature describing the association between the size of the cotyloid fossa relative to the acetabulum and characteristics of patients with femoroacetabular impingement (FAI). Purpose/Hypothesis: The purpose was to calculate the cotyloid fossa coverage percentage in the acetabulum and determine its association with patient characteristics, radiographic parameters, intra-articular findings, and preoperative patient-reported outcomes in patients with FAI. We hypothesized there is an association between the cotyloid fossa coverage percentage of the acetabulum and characteristics of patients with FAI. Study Design: Cross-sectional study; Level of evidence, 3. Methods: Patients were included who underwent standard clinical 3-T magnetic resonance imaging of the hip and primary arthroscopic FAI correction surgery during 2015 and 2016. Exclusion criteria were age <18 or >40 years, osteoarthritis, labral reconstruction, previous ipsilateral hip surgery, and hip dysplasia. Measurements of the cotyloid fossa and surrounding lunate cartilage were performed to calculate cotyloid fossa width (CFW) and cotyloid fossa height (CFH) coverage percentages. The relationships between coverage percentages and patient characteristics and intraoperative findings were assessed using independent t tests or Pearson correlations. Results: An overall 146 patients were included. Alpha angle negatively correlated with CFH coverage percentage ( r = −0.19; P = .03) and positively correlated with labral tear size ( r = 0.28; P < .01). CFH coverage percentage was negatively correlated with labral tear size ( r = −0.24; P < .01). Among patients with degenerative tears, CFH was negatively correlated with labral tear size ( r = −0.31; P < .01). However, this association was no longer significant after adjusting for sex (partial r = −0.10; P = .39). Cotyloid fossa coverage was not associated with the condition of the cotyloid fossa synovium (synovitis vs no synovitis). CFW coverage percentage was negatively correlated with the 12-Item Short Form Health Survey (SF-12) physical component summary score ( r = −0.23; P < .01). Conclusion: The CFW and CFH coverage percentages may be associated with alpha angle, labral tear size, and SF-12 physical component summary score in patients with FAI. We may be able to predict the labral condition based on preoperative measurements of CFH and CFW coverage percentages.

Charlson Comorbidity Index Is Related to Organ Damage in Systemic Lupus Erythematosus: Data from KORean lupus Network (KORNET) Registry

2017 ◽  
Vol 44 (4) ◽  
pp. 452-458 ◽  
Author(s):  
Seong-Kyu Kim ◽  
Jung-Yoon Choe ◽  
Shin-Seok Lee
Keyword(s):  
Systemic Lupus Erythematosus ◽  
Disease Activity ◽  
Lupus Erythematosus ◽  
Charlson Comorbidity Index ◽  
Short Form ◽  
Damage Index ◽  
Organ Damage ◽  
Systemic Lupus ◽  
Sf 36 ◽  
Comorbidity Index

Objective.The aim of this study was to identify whether comorbidity status is associated with organ damage in patients with systemic lupus erythematosus (SLE).Methods.A total of 502 patients with SLE enrolled in the KORean lupus Network were consecutively recruited. Data included demographics, age-adjusted Charlson Comorbidity Index (CCIa), disease activity indexes, the Systemic Lupus International Collaborating Clinics/American College of Rheumatology Damage Index (SDI), the Medical Outcomes Study Short Form-36 health survey (SF-36) score, and the Beck Depression Inventory (BDI) score.Results.Of the total patients, 21.1% (n = 106) experienced organ damage (SDI ≥ 1). Univariate correlation analysis revealed that SDI was not statistically correlated with any clinical variables (correlation coefficient r < 0.3 of all). There were significant differences in the BDI, mental component score of the SF-36, Systemic Lupus Erythematosus Disease Activity Index (SLEDAI), CCIa, C-reactive protein, and mean dose of corticosteroid between non-damage (SDI = 0) and damage (SDI ≥ 1) groups. The presence of damage to at least 1 organ in patients with SLE was found to be closely related with higher CCIa, higher SLEDAI, and mean dose of corticosteroid (OR 1.884, 95% CI 1.372–2.586, p < 0.001; OR 1.114, 95% CI 1.041–1.192, p = 0.002; OR 1.036, 95% CI 1.004–1.068, p = 0.026; respectively) in binary logistic regression analysis.Conclusion.This study suggests that organ damage as assessed by the SDI in Korean patients with SLE is related to comorbidities together with disease activity and corticosteroid exposure.

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