scholarly journals Chances and challenges of a long-term data repository in multiple sclerosis: 20th birthday of the German MS registry

2021 ◽  
Vol 11 (1) ◽  
Author(s):  
Lisa-Marie Ohle ◽  
David Ellenberger ◽  
Peter Flachenecker ◽  
Tim Friede ◽  
Judith Haas ◽  
...  
Keyword(s):  
Multiple Sclerosis ◽  
Sociodemographic Factors ◽  
Healthcare Research ◽  
Structure Design ◽  
Data Repository ◽  
Research Projects ◽  
Real World Data ◽  
Data Repositories ◽  
Term Data

AbstractIn 2001, the German Multiple Sclerosis Society, facing lack of data, founded the German MS Registry (GMSR) as a long-term data repository for MS healthcare research. By the establishment of a network of participating neurological centres of different healthcare sectors across Germany, GMSR provides observational real-world data on long-term disease progression, sociodemographic factors, treatment and the healthcare status of people with MS. This paper aims to illustrate the framework of the GMSR. Structure, design and data quality processes as well as collaborations of the GMSR are presented. The registry’s dataset, status and results are discussed. As of 08 January 2021, 187 centres from different healthcare sectors participate in the GMSR. Following its infrastructure and dataset specification upgrades in 2014, more than 196,000 visits have been recorded relating to more than 33,000 persons with MS (PwMS). The GMSR enables monitoring of PwMS in Germany, supports scientific research projects, and collaborates with national and international MS data repositories and initiatives. With its recent pharmacovigilance extension, it aligns with EMA recommendations and helps to ensure early detection of therapy-related safety signals.

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 GAMS – An infrastructure for the long-term preservation and publication of research data from the Humanities

2018 ◽  
Vol 71 (1) ◽  
pp. 207-216 ◽  
Author(s):  
Johannes Hubert Stigler ◽  
Elisabeth Steiner
Keyword(s):  
System Architecture ◽  
Academic Research ◽  
Research Data ◽  
Data Repository ◽  
Data Repositories ◽  
Domain Specific ◽  
Preservation Policy ◽  
Long Term Preservation ◽  
Data Centres

Research data repositories and data centres are becoming more and more important as infrastructures in academic research. The article introduces the Humanities’ research data repository GAMS, starting with the system architecture to preservation policy and content policy. Challenges of data centres and repositories and the general and domain-specific approaches and solutions are outlined. Special emphasis lies on the sustainability and long-term perspective of such infrastructures, not only on the technical but above all on the organisational and financial level.

scholarly journals Introduction to the Special JeSLIB Issue on Data Curation in Practice

2021 ◽  
Vol 10 (3) ◽  
Author(s):  
Cynthia Hudson Vitale ◽  
Jake R. Carlson ◽  
Hannah Hadley ◽  
Lisa Johnston
Keyword(s):  
Research Integrity ◽  
Scientific Communication ◽  
Research Data ◽  
Data Curation ◽  
Research Projects ◽  
Academic Institutions ◽  
Data Repositories ◽  
Communication Processes ◽  
Potential Impact

Research data curation is a set of scientific communication processes and activities that support the ethical reuse of research data and uphold research integrity. Data curators act as key collaborators with researchers to enrich the scholarly value and potential impact of their data through preparing it to be shared with others and preserved for the long term. This special issues focuses on practical data curation workflows and tools that have been developed and implemented within data repositories, scholarly societies, research projects, and academic institutions.

scholarly journals Sphingosine-1-phosphate Receptor Modulators in Multiple Sclerosis

2018 ◽  
Vol 13 (1) ◽  
pp. 25 ◽  
Author(s):  
Patrick Vermersch
Keyword(s):  
Multiple Sclerosis ◽  
Oral Disease ◽  
Receptor Subtypes ◽  
Sphingosine 1 Phosphate ◽  
The Central Nervous System ◽  
S1p Receptor ◽  
Phase Ii And Iii ◽  
Receptor Modulators ◽  
Term Data

The introduction of oral disease modifying therapies has transformed the treatment landscape for patients with multiple sclerosis (MS). Fingolimod (Gilenya®, Novartis, Basel, Switzerland), the first oral therapy to be approved, has demonstrated clinical efficacy as a result of modulation of subtype 1 sphingosine-1-phosphate (S1P1) receptors. This leads to retention of lymphocytes in the lymph nodes, preventing their entry into the central nervous system. However, fingolimod can cause adverse effects as a result of its interaction with other S1P receptor subtypes, which are expressed in numerous tissues, including cardiac myocytes. More selective S1P receptor agents are currently in phase II and III clinical development. Siponimod, ozanimod, ponesimod and amiselimod have demonstrated efficacy with improved safety profiles compared with fingolimod. While more long-term data are needed, these selective S1P receptor modulators appear to be promising options for the treatment of MS and other disorders associated with autoimmunity and inflammation.

scholarly journals Automated Production of Research Data Marts from a Canonical Fast Healthcare Interoperability Resource (FHIR) Data Repository: Applications to COVID-19 Research

2021 ◽  
Author(s):  
Leslie A Lenert ◽  
Andrey V. Ilatovskiy ◽  
James Agnew ◽  
Patricia Rudsill ◽  
Jeff Jacobs ◽  
...  
Keyword(s):  
Clinical Data ◽  
Data Model ◽  
Assessment Tools ◽  
Research Network ◽  
Data Repository ◽  
Common Data Model ◽  
Real World Data ◽  
Data Repositories ◽  
Automated Production ◽  
Healthcare Data

AbstractObjectiveObjective: The COVID-19 pandemic has enhanced the need for timely real-world data (RWD) for research. To meet this need, several large clinical consortia have developed networks for access to RWD from electronic health records (EHR), each with its own common data model (CDM) and custom pipeline for extraction, transformation, and load operations for production and incremental updating. However, the demands of COVID-19 research for timely RWD (e.g., 2-week delay) make this less feasible.Methods and MaterialsWe describe the use of the Fast Healthcare Interoperability Resource (FHIR) data model as a canonical model for representation of clinical data for automated transformation to the Patient-Centered Outcomes Research Network (PCORnet) and Observational Medical Outcomes Partnership (OMOP) CDMs and the near automated production of linked clinical data repositories (CDRs) for COVID-19 research using the FHIR subscription standard. The approach was applied to healthcare data from a large academic institution and was evaluated using published quality assessment tools.ResultsSix years of data (1.07M patients, 10.1M encounters, 137M laboratory results), were loaded into the FHIR CDR producing 3 linked real-time linked repositories: FHIR, PCORnet, and OMOP. PCORnet and OMOP databases were refined in subsequent post processing steps into production releases and met published quality standards. The approach greatly reduced CDM production efforts.ConclusionsFHIR and FHIR CDRs can play an important role in enhancing the availability of RWD from EHR systems. The above approach leverages 21st Century Cures Act mandated standards and could greatly enhance the availability of datasets for research.

Real-World Data Regarding Long-Term Administration of Natalizumab from a Neurology Department along Literature Review

2021 ◽  
Vol 20 ◽  
Author(s):  
Eugenia Irene Davidescu ◽  
Irina Odajiu ◽  
Constantin-Dragoș Sandu ◽  
Amalia Ghergu ◽  
Dimela Luca ◽  
...  
Keyword(s):  
Multiple Sclerosis ◽  
Adverse Events ◽  
Safety Profile ◽  
Extended Period ◽  
Real World Data ◽  
Good Tolerability ◽  
Number Of Patients ◽  
Mild Reduction ◽  
Term Administration

Background: Natalizumab is a humanized monoclonal antibody with high efficacy and an acceptable safety profile used in the treatment of patients with multiple sclerosis (MS). Objectives: Our aim was to report data regarding long-term administration of Natalizumab in patients with relapsing-remitting multiple sclerosis (RRMS) from our clinic. Methods: A retrospective observational study was performed including RRMS patients who underwent treatment with ≥ 24 Natalizumab infusions. We analyzed the EDSS values, the relapse rate and the rate and type of adverse events related to Natalizumab administration. Results: 51 subjects were included with a predominance of women (62.74%), an average age of 40.43±1.49 years, a mean disease duration of 9.86±0.7 years and mean number of Natalizumab infusions of 45.58±2.74. An increased number of patients (80.39%) were relapse-free and there was observed a mild reduction of the mean EDSS value following Natalizumab initiation in patients who had not been treated with other disease modifying therapies anteriorly. Among the encountered adverse events we registered: increased liver transaminases (13.72%), local infections (7.84%) and dysmenorrhea in one patient. The rate of severe adverse events was 3.92 and there were registered no cases of Progressive Multifocal Leukoencephalopathy (PML). Conclusions: Natalizumab proves to be effective, has an adequate safety profile and can be administered with good tolerability for a rather extended period of time, provided that the patients are closely monitored.

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.

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