scholarly journals Italian multiple sclerosis register as the basis for post-authorization safety studies

2020 ◽  
Vol 30 (Supplement_5) ◽  
Author(s):  
M Ponzio ◽  
M Trojano ◽  
M Capobianco ◽  
M Pugliatti ◽  
M Ulivelli ◽  
...  
Keyword(s):  
Multiple Sclerosis ◽  
Pharmaceutical Industry ◽  
Real World ◽  
Treatment Effectiveness ◽  
Patient Registries ◽  
Real World Data ◽  
World Data ◽  
Disease Modifying Therapy ◽  
Disease Modifying

Abstract While the safety and efficacy of Disease Modifying Therapy (DMT) in multiple sclerosis (MS) are assessed in clinical trials, these are of relatively short duration and always confined to highly selected patient groups. The evaluation of real-world data such as patient registries, is vital as it offers long-term data collection and is patient rather than product-focused during the lifetime of MS, and allows to document a patient's treatment history throughout the disease course. Patient registries can play an important role in monitoring the safety of drus. Regulators and the pharmaceutical industry have shown interest in complementing or even replacing phase 4 clinical studies with data from MS registries. The Italian MS Register (IMSR), in collaboration with the Big MS Data initiative, that also includes the national MS registries of Denmark, France, and Sweden and the international database network MSBase, came together with industry to conduct studies on post-authorization safety (PASS) and treatment effectiveness. The IMSR includes the clinical history of approximately 55,000 patients, or approximatively 45% of the estimated cases of MS in Italy. More than 10,000 patients have a follow-up duration of over 10 years. A Core Protocol outlining principles of PASS projects was created in which aggregated results made available to sponsors and health authorities. The Core Protocol specifies variables, emphasizes improved capture of adverse events, in particular cancer, non-melanoma skin cancers and immunosuppression-related infections, all MedDRA-coded. EUROCAT codes for pregnancy outcomes are also documented. Regulators, the pharmaceutical industry and national-level registries have jointly identified a format of collaboration on PASS for DMT in MS to benefit patients and the larger society. In this way, we hope to contribute to a framework that will include emerging and existing registries with the common goal of contributing to the advancement of knowledge in MS. Key messages The real-world data can contribute to understanding of the impact of disease-modifying therapy on long term. A format of collaboration among clinical research, regulators and pharmaceutical industry could be a winning framework to improve the knowledge on safety and treatment effectiveness in MS.

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 Impact of drug diversity on treatment effectiveness in relapsing-remitting multiple sclerosis (RRMS) in Germany between 2010 and 2018: real-world data from the German NeuroTransData multiple sclerosis registry

BMJ Open ◽  
2021 ◽  
Vol 11 (8) ◽  
pp. e042480
Author(s):  
Stefan Braune ◽  
Fabian Rossnagel ◽  
Heidi Dikow ◽  
Arnfin Bergmann
Keyword(s):  
Multiple Sclerosis ◽  
Real World ◽  
Treatment Effectiveness ◽  
Real World Data ◽  
World Data ◽  
Conversion Rates ◽  
Relapsing Remitting ◽  
Remitting Multiple Sclerosis ◽  
Relapsing Remitting Multiple Sclerosis ◽  
The Impact

ObjectiveTo evaluate the impact of drug diversity on treatment effectiveness in relapsing-remitting multiple sclerosis (RRMS) in Germany.DesignThis study employs real-world data captured in-time during clinical visits in 67 German neurology outpatient offices of the NeuroTransData (NTD) multiple sclerosis (MS) registry between 1 January 2010 and 30 June 2019, including 237 976 visits of 17 553 patients with RRMS. Adherence and clinical effectiveness parameters were analysed by descriptive statistics, time-to-event analysis overall and by disease-modifying therapies (DMTs) stratified by administration modes (injectable, oral and infusion). Three time periods were compared: 2010–2012, 2013–2015 and 2016–2018.ResultsBetween 2010 and 2018, an increasing proportion of patients with RRMS were treated with DMTs and treatment was initiated sooner after diagnosis of MS. Introduction of oral DMT temporarily induced higher readiness to switch. Comparing the three index periods, there was a continuous decrease of annualised relapse rates, less frequent Expanded Disability Status Scale (EDSS) progression and increasing periods without relapse, EDSS worsening and with stability of no-evidence-of-disease-activity 2 and 3 criteria, lower conversion rates to secondary progressive MS on oral and on injectable DMTs.ConclusionSparked by the availability of new mainly oral DMTs, RRMS treatment effectiveness improved clinically meaningful between 2010 and 2018. As similar effects were seen for injectable and oral DMTs more than for infusions, a better personalised treatment allocation in many patients is likely. These results indicate that there is an overall beneficial effect for the whole patient with MS population as a result of the greater selection of available DMTs, a benefit beyond the head-to-head comparative efficacy, resulting from an increased probability and readiness to individualise MS therapy.

878-P: Real-World Data from U.S. Patients Using a Long-Term Implantable Continuous Glucose Monitoring (CGM) System: Age Effect on Glycemic Control

Diabetes ◽  
2020 ◽  
Vol 69 (Supplement 1) ◽  
pp. 878-P
Author(s):  
KATHERINE TWEDEN ◽  
SAMANWOY GHOSH-DASTIDAR ◽  
ANDREW D. DEHENNIS ◽  
FRANCINE KAUFMAN
Keyword(s):  
Glycemic Control ◽  
Continuous Glucose Monitoring ◽  
Real World ◽  
Glucose Monitoring ◽  
Age Effect ◽  
Real World Data ◽  
World Data

Long-term outcome of minimally invasive mitral valve annuloplasty in disproportionate mitral regurgitation

2020 ◽  
Vol 41 (Supplement_2) ◽  
Author(s):  
M Albano ◽  
S Nagumo ◽  
M Vanderheyden ◽  
J Bartunek ◽  
C Collet ◽  
...  
Keyword(s):  
Mitral Valve ◽  
Mitral Regurgitation ◽  
Minimally Invasive ◽  
Real World ◽  
Independent Predictor ◽  
Real World Data ◽  
Long Term Outcome ◽  
World Data ◽  
Mitral Valve Annuloplasty

Abstract Background Hypothetical concept of disproportionate secondary mitral regurgitation (SMR) has been recently introduced to facilitate patient's selection for mitral valve intervention. However, real world data validating this concept are unavailable. Purpose To investigate long-term effects of minimally invasive mitral valve annuloplasty (MVA) in patients with disproportionate (dSMR) versus proportionate SMR. Methods The study population consisted of 44 consecutive patients (age 67±9,5 years; 64% males) on guidelines-directed therapy with advanced heart failure (HF), reduced LV ejection fraction (EF) (32±9,7%) and SMR undergoing isolated mini-invasive MVA. Patients with organic mitral regurgitation or concomitant myocardial revascularization were excluded. To assess SMR disproportionality, the PISA-derived effective regurgitant orifice area (EROA) and regurgitant volume (RV) were compared to the estimated EROA and RV by using Gorlin formula and pooled real world data. Results According to EROA, a total of 20 (46%) and 24 (54%) patients, respectively, had dSMR and proportionate SMR (pSMR). According to RV, a total of 17 (39%) had dSMR and 27 (61%) had pSMR. Patients with dSMR showed significantly lower prevalence of male gender and higher prevalence of diabetes mellitus than patients with pSMR (p<0,001). Moreover, we observed smaller LV end-diastolic volume, larger EROA and RV (both p<0,01) and higher LV EF (p=0,02) in the dSMR versus the pSMR group. Other baseline characteristics were similar. During median follow up of 4.39 y (IQR 2,2–9,96y), a total of 25 (56%) patients died from any cause while 21 (47%) individuals were readmitted for worsening HF. Patients with dSMR versus pSMR according to both EROA and RV showed significantly lower rate of HF readmissions (both p<0.05) (Figure 1, 2). In Cox regression analysis combining clinical and imaging parameters, dSMR was the only independent predictor of HF readmissions (HR 0.20, 95% CI 0.07–0.60, p=0.004). In contrast, mortality was similar between dSMR and pSMR (NS) with age as the only independent predictor (HR 1,10; 95% CI 1,03–1,18, p=0,003). Conclusions Minimally invasive MVA is associated with significant reduction of HF readmissions in patients with dSMR versus pSMR while the mortality is similar. This suggests the importance of other parameters, i.e. age and degree of LV remodeling, to guide clinical management in SMR. Funding Acknowledgement Type of funding source: None

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