scholarly journals Trajectories of Adherence to Biologic Disease-Modifying Anti-Rheumatic Drugs in Tuscan Administrative Databases: The Pathfinder Study

2021 ◽  
Vol 10 (24) ◽  
pp. 5743
Author(s):  
Irma Convertino ◽  
Sabrina Giometto ◽  
Rosa Gini ◽  
Massimiliano Cazzato ◽  
Marco Fornili ◽  
...  
Keyword(s):  
Medication Possession Ratio ◽  
Administrative Databases ◽  
Adherence Behavior ◽  
Real World Data ◽  
Implementation Phase ◽  
Biologic Dmard ◽  
Baseline Characteristics ◽  
Disease Modifying ◽  
Scanty Information

Scanty information on clustering longitudinal real-world data is available in the medical literature about the adherence implementation phase in rheumatoid arthritis (RA). To identify and characterize trajectories by analyzing the implementation phase of adherence to biologic Disease-Modifying Anti-Rheumatic Drugs (DMARDs), we conducted a retrospective cohort drug-utilization study using Tuscan administrative databases. RA patients were identified by a validated algorithm, including the first biologic DMARD supply from 2010 to 2015, RA specialist visit in the year before or after the first supply date and RA diagnosis in the five years before or in the year after the first supply date. We observed users for three years or until death, neoplasia, or pregnancy. We evaluated adherence quarterly through the Medication Possession Ratio. Firstly, we identified adherence trajectories and described the baseline characteristics; then, we focused on the trajectory most populated to distinguish the related sub-trajectories. We identified 952 first ever-biologic DMARD users in RA (712 females, mean age 52.7 years old, standard deviation 18.8). The biologic DMARD mostly supplied was etanercept (387 users) followed by adalimumab (233). Among 935 users with at least 3 adherence values, we identified 49 fully-adherent users, 829 continuous users, and 57 early-discontinuing users. Significant differences were observed among the index drugs. After focusing on the continuous users, three sub-trajectories were identified: continuous-steady users (556), continuous-alternate users (207), and continuous-declining users (66). No relevant differences emerged at the baseline. The majority of first ever-biologic DMARD users showed a continuous adherence behavior in RA. The role of adherence potential predictors and the association with effectiveness and safety outcomes should be explored by further studies.

Adherence to Disease-Modifying Therapies at a Multiple Sclerosis Clinic: The Role of the Specialty Pharmacist

2019 ◽  
Vol 33 (5) ◽  
pp. 605-611 ◽  
Author(s):  
Aimee M. Banks ◽  
Megan E. Peter ◽  
Genna M. Holder ◽  
Jacob A. Jolly ◽  
Brandon M. Markley ◽  
...  
Keyword(s):  
Multiple Sclerosis ◽  
Medication Management ◽  
Medication Possession Ratio ◽  
Financial Assistance ◽  
Disease Modifying Therapy ◽  
Pharmacy Claims ◽  
Specialty Pharmacy ◽  
Disease Modifying

Background: Disease-modifying therapy (DMT) delays disease progression and improves quality of life for patients with multiple sclerosis (MS), but adherence to DMT is often suboptimal. Vanderbilt Specialty Pharmacy (VSP) embeds pharmacists within an outpatient MS clinic to provide medication management and address barriers to adherence. Objective: We evaluated rates and predictors of adherence to DMT among patients with MS at an integrated specialty pharmacy. Methods: We included patients with MS who filled ≥3 DMT prescriptions from VSP during the study period. Adherence was defined as medication possession ratio (MPR) or proportion of days covered (PDC) ≥0.8. Reasons for nonadherence were collected from pharmacy claims and electronic medical records. Results: The study included 653 patients. Average MPR and PDC were 0.93 and 0.94, respectively. Eighty-eight percent of patients achieved MPR ≥0.8; 89% achieved PDC ≥0.8. Using financial assistance and having $0 out-of-pocket cost were associated with higher odds of achieving MPR and PDC ≥0.8 ( P < .05). Of the 12% of patients who were nonadherent, most were unreachable for refills. Conclusions: Ensuring financial assistance and low out-of-pocket costs are associated with high adherence to DMT within an integrated specialty clinic, but more work is needed to address adherence in unreachable patients.

scholarly journals ECMO in COVID-19—prolonged therapy needed? A retrospective analysis of outcome and prognostic factors

Perfusion ◽  
2021 ◽  
pp. 026765912199599
Author(s):  
Esther Dreier ◽  
Maximilian Valentin Malfertheiner ◽  
Thomas Dienemann ◽  
Christoph Fisser ◽  
Maik Foltan ◽  
...  
Keyword(s):  
Lung Compliance ◽  
Ecmo Support ◽  
Short Term ◽  
Single Center Study ◽  
Venovenous Extracorporeal Membrane Oxygenation ◽  
Baseline Characteristics ◽  
Ecmo Therapy ◽  
Vv Ecmo

Background: The role of venovenous extracorporeal membrane oxygenation (VV ECMO) in patients with COVID-19-induced acute respiratory distress syndrome (ARDS) still remains unclear. Our aim was to investigate the clinical course and outcome of those patients and to identify factors associated with the need for prolonged ECMO therapy. Methods: A retrospective single-center study on patients with VV ECMO for COVID-19-associated ARDS was performed. Baseline characteristics, ventilatory and ECMO parameters, and laboratory and virological results were evaluated over time. Six months follow-up was assessed. Results: Eleven of 16 patients (68.8%) survived to 6 months follow-up with four patients requiring short-term (<28 days) and seven requiring prolonged (⩾28 days) ECMO support. Lung compliance before ECMO was higher in the prolonged than in the short-term group (28.1 (28.8–32.1) ml/cmH2O vs 18.7 (17.7–25.0) ml/cmH2O, p = 0.030). Mechanical ventilation before ECMO was longer (19 (16–23) days vs 5 (5–9) days, p = 0.002) and SOFA score was higher (12.0 (10.5–17.0) vs 10.0 (9.0–10.0), p = 0.002) in non-survivors compared to survivors. Low viral load during the first days on ECMO tended to indicate worse outcomes. Seroconversion against SARS-CoV-2 occurred in all patients, but did not affect outcome. Conclusions: VV ECMO support for COVID-19-induced ARDS is justified if initiated early and at an experienced ECMO center. Prolonged ECMO therapy might be required in those patients. Although no relevant predictive factors for the duration of ECMO support were found, the decision to stop therapy should not be made dependent of the length of ECMO treatment.

scholarly journals Long-term effectiveness in patients previously treated with cladribine tablets: a real-world analysis of the Italian multiple sclerosis registry (CLARINET-MS)

2020 ◽  
Vol 13 ◽  
pp. 175628642092268 ◽  
Author(s):  
Francesco Patti ◽  
Andrea Visconti ◽  
Antonio Capacchione ◽  
Sanjeev Roy ◽  
Maria Trojano ◽  
...  
Keyword(s):  
Multiple Sclerosis ◽  
Real World ◽  
Event Analysis ◽  
Treatment Change ◽  
Real World Data ◽  
Indirect Measure ◽  
Disease Modifying Drugs ◽  
Disease Modifying ◽  
Using Data

Background: The CLARINET-MS study assessed the long-term effectiveness of cladribine tablets by following patients with multiple sclerosis (MS) in Italy, using data from the Italian MS Registry. Methods: Real-world data (RWD) from Italian MS patients who participated in cladribine tablets randomised clinical trials (RCTs; CLARITY, CLARITY Extension, ONWARD or ORACLE-MS) across 17 MS centres were obtained from the Italian MS Registry. RWD were collected during a set observation period, spanning from the last dose of cladribine tablets during the RCT (defined as baseline) to the last visit date in the registry, treatment switch to other disease-modifying drugs, date of last Expanded Disability Status Scale recording or date of the last relapse (whichever occurred last). Time-to-event analysis was completed using the Kaplan–Meier (KM) method. Median duration and associated 95% confidence intervals (CI) were estimated from the model. Results: Time span under observation in the Italian MS Registry was 1–137 (median 80.3) months. In the total Italian patient population ( n = 80), the KM estimates for the probability of being relapse-free at 12, 36 and 60 months after the last dose of cladribine tablets were 84.8%, 66.2% and 57.2%, respectively. The corresponding probability of being progression-free at 60 months after the last dose was 63.7%. The KM estimate for the probability of not initiating another disease-modifying treatment at 60 months after the last dose of cladribine tablets was 28.1%, and the median time-to-treatment change was 32.1 (95% CI 15.5–39.5) months. Conclusion: CLARINET-MS provides an indirect measure of the long-term effectiveness of cladribine tablets. Over half of MS patients analysed did not relapse or experience disability progression during 60 months of follow-up from the last dose, suggesting that cladribine tablets remain effective in years 3 and 4 after short courses at the beginning of years 1 and 2.

scholarly journals Introducing the integrated, cross-sectional psycho-oncology intervention programme and PIM

2020 ◽  
Vol 30 (Supplement_5) ◽  
Author(s):  
T Krieger
Keyword(s):  
Quality Criteria ◽  
Assessment Instruments ◽  
Intervention Programme ◽  
Cross Sectional ◽  
Implementation Phase ◽  
Group Participation ◽  
Early Implementation ◽  
Examination Process

Abstract From the literature and experience, we know that the quality of patient information material (PIM) has a direct impact on its utilization and therefore also on the acceptance and success of an intervention. In this brief introduction session (10 minutes), the innovative “integrated, cross-sectional psycho-oncology” (isPO) programme and the context of its implementation will be sketched. In the programmés development phase, isPO specific-PIM was developed and utilized in its early implementation phase. This will be presented to the audience. Next, an overview regarding the general PIM quality criteria: correctness of content, legibility, comprehensibility and usability in detail will be given. Finally, common guidelines, checklists and quality assessment instruments will be presented, and the role of the target group (participation degree) in the development or examination process will be critically worked out.

scholarly journals Disease-modifying therapy prescription patterns in people with multiple sclerosis by age

2021 ◽  
Vol 14 ◽  
pp. 175628642110064
Author(s):  
Yinan Zhang ◽  
Amber Salter ◽  
Shan Jin ◽  
William J. Culpepper ◽  
Gary R. Cutter ◽  
...  
Keyword(s):  
Multiple Sclerosis ◽  
Disease Activity ◽  
Age Groups ◽  
The Elderly ◽  
Department Of Veterans Affairs ◽  
Real World Data ◽  
Research Committee ◽  
The North ◽  
Disease Modifying ◽  
Magnetic Resonance Imaging Mri

Background: Disease-modifying therapies (DMTs) for multiple sclerosis (MS) are approved for their ability to reduce disease activity, namely clinical relapses and signal changes on magnetic resonance imaging (MRI). Disease activity appears age dependent. Thus, the greatest benefit would be expected in younger people with MS (PwMS) whereas benefits in the elderly are uncertain. Methods: Real-world data were obtained from PwMS from the North American Research Committee on Multiple Sclerosis (NARCOMS) registry and the US Department of Veterans Affairs Multiple Sclerosis Surveillance Registry (MSSR). Results: 6948 PwMS were surveyed from NARCOMS, and the MSSR had 1719 participants. In younger adult PwMS 40-years old or less, 183 (61.4%) in NARCOMS and 179 (70.5%) in the MSSR were prescribed DMTs. Among PwMS over age 60, 1575 (40.1%) in NARCOMS and 239 (36.3%) in the MSSR were prescribed DMTs. More PwMS in the age group of 31–40 ( p = 0.035) and 41–50 ( p = 0.001) in the MSSR were using DMTs compared with PwMS of the same age groups in NARCOMS. Conclusion: These findings suggest that DMTs are under-utilized in the younger population and continue to be commonly prescribed in the elderly. Broader access may explain the higher prescription rate of DMTs in US veterans.

scholarly journals Real-world data of thrombotic microangiopathy management: The key role of ADAMTS13 activity and complement testing

2021 ◽  
Vol 3 ◽  
pp. 100043
Author(s):  
Eleni Gavriilaki ◽  
Eudoxia-Evaggelia Koravou ◽  
Thomas Chatziconstantinou ◽  
Christina Kalpadaki ◽  
Nikoleta Printza ◽  
...  
Keyword(s):  
Real World ◽  
Thrombotic Microangiopathy ◽  
Adamts13 Activity ◽  
Real World Data ◽  
World Data

Augmenting Real-World Data Through Modeling Key Clinical Trial Eligibility Criteria: An Example of Patients With Non–small-Cell Lung Cancer Treated With Pembrolizumab

2021 ◽  
pp. 849-858
Author(s):  
Thomas Jemielita ◽  
Xiaoyun (Nicole) Li ◽  
Thomas Burke ◽  
Kai-Li Liaw ◽  
Wei Zhou ◽  
...  
Keyword(s):  
Lung Cancer ◽  
Cell Death ◽  
Programmed Cell Death ◽  
Small Cell Lung Cancer ◽  
Real World ◽  
Small Cell ◽  
Small Cell Lung ◽  
Real World Data ◽  
Eligibility Criteria ◽  
Baseline Characteristics

PURPOSE To compare and characterize baseline characteristics and overall survival (OS) differences by key oncology eligibility criteria for real-world patients from the Flatiron Health database with advanced non–small-cell lung cancer (NSCLC) who received pembrolizumab monotherapy. METHODS Real world data (RWD) were from the Flatiron Health advanced NSCLC database and include patients who initiated pembrolizumab monotherapy (first, second, or third line of therapy) by November 30, 2019. At the data cutoff (May 31, 2020), the median survival follow-up time was 8.4 months. Eligible patients satisfy the criteria of Eastern Cooperative Oncology Group performance status of 0/1 and laboratory values indicative of adequate organ function. RWD were analyzed for all patients and patients with a programmed cell death ligand-1 tumor proportion score ≥ 1%. Patients were divided into three categories: ineligible, eligible, and unknown (who satisfy all observed criteria, with at least one missing). An augmented population was also formed, which combines the latter two groups through a propensity-based adjustment. RESULTS At the data cutoff, N = 3,877 patients with NSCLC received pembrolizumab monotherapy (1L = 2,682, 2L = 946, and 3L = 249). OS was consistently lower for the ineligible with similar survival for the eligible and augmented. Among all patients, the median OS in months (95% CI) was 8.2 (7.5 to 9.6), 16.3 (14.5 to 18.4), 16.4 (15.1 to 19.3), and 16.8 (15.6 to 18.5) for the ineligible (47%, n = 1,827), unknown (27%, n = 1,045), eligible (26%, n = 1,005), and augmented, respectively. The results were similar for patients with a programmed cell death ligand-1 tumor proportion score ≥ 1%. CONCLUSION Real-world patients who received pembrolizumab monotherapy and meet key clinical eligibility criteria exhibited similar baseline characteristics and OS profiles as the unknown and augmented patient groups. Population augmentation is a feasible approach for improving the power of RWD analysis.

58 Relationship Between Early Laboratory Measures and Neurological Injury in Neonates Undergoing Therapeutic Hypothermia

2021 ◽  
Vol 26 (Supplement_1) ◽  
pp. e39-e41
Author(s):  
Lilian Kebaya ◽  
Mong Tieng Ee ◽  
Michael Miller ◽  
Soume Bhattacharya
Keyword(s):  
Therapeutic Hypothermia ◽  
Roc Curve ◽  
Base Excess ◽  
Curve Analysis ◽  
Neurological Injury ◽  
Acid Base ◽  
Roc Curve Analysis ◽  
Baseline Characteristics ◽  
Laboratory Measures

Abstract Primary Subject area Neonatal-Perinatal Medicine Background Hypoxic-ischemic encephalopathy (HIE) is a major contributor to morbidity and mortality. Therapeutic hypothermia (TH) is the standard of care for neonates with moderate to severe HIE. Brain magnetic resonance imaging (MRI) is the imaging modality of choice for confirmation of HIE, assessment of injury severity, and prognostication. Reliable, inexpensive and widely available laboratory measures for early identification of risk for neurological injury can play a critical role in the optimal management of neonatal HIE, especially in the resource-limited setting. Our study examined whether derangements in early routine laboratory measures (acid-base, haematological, metabolic) were worse in neonates with MRI findings of neurological injury. Objectives Primary objective: To evaluate the role of early laboratory measures in predicting neurological injury as detected by MRI at 72 hours. Secondary objective: To evaluate the role of early laboratory measures in predicting survival to NICU discharge in patients with HIE. Design/Methods This single-centre, retrospective cohort study included neonates ≥ 35 weeks gestation with moderate to severe HIE, who had undergone therapeutic hypothermia. Based on findings of brain MRI completed within 72 hours of life, our cohort was divided into 2 groups: neonates with, and without, evidence of neurological injury consistent with HIE. Baseline characteristics, as well as laboratory measures, were compared between groups, and a receiver operating characteristic (ROC) curve analysis was conducted to determine the cut-off for prediction of neurological injury based on the highest sensitivity and specificity values. Results 104 neonates were analyzed. Baseline characteristics (Table 1) were similar between both groups, except for cord venous pH and base excess (BE), which were significantly lower in the abnormal MRI group (p = 0.02). In bivariate analysis, pH (at 1 h of age, p = 0.027), BE (at 1 h, p = 0.001, and 6 h of age, p = 0.004), ionized calcium (at 6 h of age, p = 0.02), and platelets (at 1 h of age, p = 0.004) were significantly different in neonates with abnormal MRI. In ROC curve analysis, BE at 1 h of life was the best predictor of abnormal MRI (AUC = 0.71, p = 0.002), with a cut-off value of ≤ -14.95, sensitivity of 67% and specificity of 66% (Figure 1). Conclusion Among neonates with HIE undergoing TH, early laboratory measures such as acid-base status, ionized calcium, and platelet count were worse in neonates with abnormal MRI, in comparison to neonates with normal MRI. Base excess at 1 h of life is a good predictor of abnormal MRI. Future prospective studies to validate these findings are needed

scholarly journals Emulated Clinical Trials from Longitudinal Real-World Data Efficiently Identify Candidates for Neurological Disease Modification: Examples from Parkinson’s Disease

2020 ◽  
Author(s):  
Daphna Laifenfeld ◽  
Chen Yanover ◽  
Michal Ozery-Flato ◽  
Oded Shaham ◽  
Michal Rozen-Zvi ◽  
...  
Keyword(s):  
Parkinson’S Disease ◽  
Parkinson's Disease ◽  
Clinical Trials ◽  
Real World ◽  
Late Onset ◽  
Real World Data ◽  
World Data ◽  
Healthcare Data ◽  
Beneficial Effects ◽  
Disease Modifying

AbstractReal-world healthcare data hold the potential to identify therapeutic solutions for progressive diseases by efficiently pinpointing safe and efficacious repurposing drug candidates. This approach circumvents key early clinical development challenges, particularly relevant for neurological diseases, concordant with the vision of the 21stCentury Cures Act. However, to-date, these data have been utilized mainly for confirmatory purposes rather than as drug discovery engines. Here, we demonstrate the usefulness of real-world data in identifying drug repurposing candidates for disease-modifying effects, specifically candidate marketed drugs that exhibit beneficial effects on Parkinson’s disease (PD) progression. We performed an observational study in cohorts of ascertained PD patients extracted from two large medical databases, Explorys SuperMart (N=88,867) and IBM MarketScan Research Databases (N=106,395); and applied two conceptually different, well-established causal inference methods to estimate the effect of hundreds of drugs on delaying dementia onset as a proxy for slowing PD progression. Using this approach, we identified two drugs that manifested significant beneficial effects on PD progression in both datasets: rasagiline, narrowly indicated for PD motor symptoms; and zolpidem, a psycholeptic. Each confers its effects through distinct mechanisms, which we explored via a comparison of estimated effects within the drug classification ontology. We conclude that analysis of observational healthcare data, emulating otherwise costly, large, and lengthy clinical trials, can highlight promising repurposing candidates, to be validated in prospective registration trials, for common, late-onset progressive diseases for which disease-modifying therapeutic solutions are scarce.

scholarly journals Predictors of adherence and persistence to disease-modifying therapies in Multiple Sclerosis

2021 ◽  
Vol 14 ◽  
pp. 175628642110310
Author(s):  
Gisela Zanga ◽  
Estefania Drzewiscki ◽  
Paula Tagliani ◽  
Maximiliano Smietniansky ◽  
Maria M. Esnaola y Rojas ◽  
...  
Keyword(s):  
Multiple Sclerosis ◽  
Medication Possession Ratio ◽  
Care Program ◽  
Cross Sectional Study ◽  
Response To Treatment ◽  
Cross Sectional ◽  
National Medical ◽  
Disease Modifying Therapies ◽  
Disease Modifying ◽  
Predictors Of Adherence

Background and Aims: In multiple sclerosis (MS), non-adherence/non-persistence is related to suboptimal response to treatment, including disease relapses and the need for more expensive healthcare. The aim of this study was to identify predictors related to adherence to disease modifying therapies (DMTs) in a cohort of Argentinian MS patients. Methods: We conducted a cross-sectional study at the National Medical Care Program from Argentina. MS patients with at least one claim for a DMT from 1 January 2017 to 1 October 2017 were identified. A telephone survey was performed to assess clinical and demographic factors. The medication possession ratio (MPR) was used to estimate adherence; MPR <80% defined non-adherence. Associations were studied using a logistic regression model. Results: Our database included 648 MS patients. A total of 360 patients (60% females, mean age 55.3 years) accepted to participate. Of these, 308 (85.5%) patients were receiving DMT at the time of the survey. Some 198 (63.7%) were receiving injectable therapies. Optimal adherence was 47.7%. Adherence was associated with oral medication [odds ratio (OR) 1.83 95% confidence interval (CI) 1.13–3.00, p = 0.014]. A factor related to oral drugs was higher educational level (OR 2.86 95%CI 1.41–5.81, p = 0.004). Conclusion: This real-world study showed better adherence and persistence on treatment with oral therapies in MS patients in Argentina.

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