scholarly journals Third party laboratory data management: Perspective with respect to clinical data management

2014 ◽  
Vol 5 (1) ◽  
pp. 41 ◽  
Author(s):  
Jasmin Johnson ◽  
Vishwanath Kanagali ◽  
D Prabu
Blood ◽  
2020 ◽  
Vol 136 (Supplement 1) ◽  
pp. 38-39
Author(s):  
Katharina Versmold ◽  
Carina Raiser ◽  
Imi Faghmous ◽  
Pablo Katz ◽  
Aijing Shang ◽  
...  

Background Paroxysmal nocturnal hemoglobinuria (PNH) is a life-threatening disease of dysregulated complement activation. It is a rare disease with an estimated incidence of 1 to 1.5 cases per million people globally. Eculizumab is a humanized monoclonal anti-complement component 5 antibody that was approved for the treatment of patients with PNH in the United States and European Union in 2007, yet unmet medical needs remain. Up to half of patients continue to require blood transfusions despite treatment with eculizumab, and hemolytic activity remains detectable in many patients (Brodsky et al. Blood. 2008). Eculizumab is not available in many countries. In places where treatment is approved, there are further impediments to access, such as cost of treatment, reimbursement issues, infrastructure limitations, and patient restrictions (Risitano et al. Am J Hematol. 2018; Risitano et al. Front Immunol. 2019). Data published on real-world outcomes of eculizumab are limited. Here we describe a study that will retroactively analyze data from patients with PNH treated with eculizumab at the Essen University Hospital in Germany. Study Design and Methods This retrospective, secondary data use, cohort study will include all patients at the Essen University Hospital who were diagnosed with PNH and treated with eculizumab prior to April 2018. Clinical data from medical records were entered into an electronic case report form (eCRF). Source data verification has been performed for all clinical data. Laboratory data were extracted directly from the hospital computer system. The Essen University hospital also checked and verified missing laboratory data. Patient-level data in the eCRF and laboratory data were fully anonymized. The primary objective of the study is to understand the remaining unmet medical need by describing the eculizumab dose and frequency of dose adjustment and describing the proportions of patients who experience intravascular and extravascular hemolysis while on treatment. The secondary objectives include explorations of the association between lactate dehydrogenase (LDH) and hemoglobin stabilization with clinical outcomes (eg, breakthrough hemolysis and the need for red blood cell transfusion), the association between PNH clone size and clinical outcomes and the risk of thrombosis, the changes in LDH and hemoglobin levels over time, the need for red blood cell transfusion during eculizumab treatment, and the proportion of eculizumab-treated patients with positive monospecific Coombs test results. In addition, opportunities to apply machine-learning methodologies to predict patients who may not respond to eculizumab will be explored. Many of the analyses will be descriptive. The associations between LDH and hemoglobin with clinical outcomes will be evaluated using rank correlation coefficients or logistic regression. Multivariable regression will be used to explore the prognostic value of clone size on clinical outcomes and thrombosis events. This retrospective study includes 85 patients with PNH with complete clinical and laboratory data (Table). The median age of the cohort was 38 years old, and the cohort was split evenly between men and women. Many patients received a diagnosis of PNH prior to the availability of eculizumab, as the year of diagnosis was 2010 or earlier for 53 patients (62%). The median years of follow-up from initiation of eculizumab was 4.7 years. Overall, 34% had aplastic anemia at diagnosis, and symptoms of fatigue, abdominal pain, and kidney failure were reported in 60%, 34%, and 15%, respectively. At data cutoff, 92% of patients were still alive. Summary The patient demographics in this study are comparable to other studies in PNH, suggesting a representative population. With median follow-up time of nearly 5 years, this study will allow for a long-term assessment of the patient experience with eculizumab. High-quality study data is ensured via full source data verification of clinical data and verification of missing laboratory data. These study results will help to address many research questions in PNH, identify the remaining unmet medical need, and also inform new drug development. Disclosures Versmold: F. Hoffmann-La Roche Ltd: Other: All authors received support for third party writing assistance, furnished by Scott Battle, PhD, provided by F. Hoffmann-La Roche, Basel, Switzerland.. Raiser:F. Hoffmann-La Roche Ltd: Other: All authors received support for third party writing assistance, furnished by Scott Battle, PhD, provided by F. Hoffmann-La Roche, Basel, Switzerland.. Faghmous:F. Hoffmann-La Roche Ltd: Ended employment in the past 24 months, Other: All authors received medical writing support for this abstract, furnished by Scott Battle, funded by F. Hoffmann-La Roche Ltd, Basel, Switzerland. ; Kite Pharma: Current Employment. Katz:F. Hoffmann-La Roche Ltd: Current Employment, Other: All authors received support for third party writing assistance, furnished by Scott Battle, PhD, provided by F. Hoffmann-La Roche, Basel, Switzerland.. Shang:F. Hoffmann-La Roche Ltd: Current Employment, Current equity holder in publicly-traded company, Other: All authors received support for third party writing assistance, furnished by Scott Battle, PhD, provided by F. Hoffmann-La Roche, Basel, Switzerland.. Xu:F. Hoffmann-La Roche Ltd: Current Employment, Other: All authors received support for third party writing assistance, furnished by Scott Battle, PhD, provided by F. Hoffmann-La Roche, Basel, Switzerland.. Röth:Novartis: Consultancy, Honoraria; Roche: Consultancy, Honoraria, Research Funding; Sanofi: Consultancy, Honoraria; Biocryst: Consultancy, Honoraria; Apellis: Consultancy, Honoraria; Alexion Pharmaceuticals Inc.: Consultancy, Honoraria, Research Funding.


2019 ◽  
Vol 14 (3) ◽  
pp. 160-172 ◽  
Author(s):  
Aynaz Nourani ◽  
Haleh Ayatollahi ◽  
Masoud Solaymani Dodaran

Background:Data management is an important, complex and multidimensional process in clinical trials. The execution of this process is very difficult and expensive without the use of information technology. A clinical data management system is software that is vastly used for managing the data generated in clinical trials. The objective of this study was to review the technical features of clinical trial data management systems.Methods:Related articles were identified by searching databases, such as Web of Science, Scopus, Science Direct, ProQuest, Ovid and PubMed. All of the research papers related to clinical data management systems which were published between 2007 and 2017 (n=19) were included in the study.Results:Most of the clinical data management systems were web-based systems developed based on the needs of a specific clinical trial in the shortest possible time. The SQL Server and MySQL databases were used in the development of the systems. These systems did not fully support the process of clinical data management. In addition, most of the systems lacked flexibility and extensibility for system development.Conclusion:It seems that most of the systems used in the research centers were weak in terms of supporting the process of data management and managing clinical trial's workflow. Therefore, more attention should be paid to design a more complete, usable, and high quality data management system for clinical trials. More studies are suggested to identify the features of the successful systems used in clinical trials.


2019 ◽  
Vol 14 (1) ◽  
pp. 10-23 ◽  
Author(s):  
Aynaz Nourani ◽  
Haleh Ayatollahi ◽  
Masoud Solaymani Dodaran

Background:A clinical data management system is a software supporting the data management process in clinical trials. In this system, the effective support of clinical data management dimensions leads to the increased accuracy of results and prevention of diversion in clinical trials. The aim of this review article was to investigate the dimensions of data management in clinical data management systems.Methods:This study was conducted in 2017. The used databases included Web of Science, Scopus, Science Direct, ProQuest, Ovid Medline and PubMed. The search was conducted over a period of 10 years from 2007 to 2017. The initial number of studies was 101 reaching 19 in the final stage. The final studies were described and compared in terms of the year, country and dimensions of the clinical data management process in clinical trials.Results:The research findings indicated that none of the systems completely supported the data management dimensions in clinical trials. Although these systems were developed for supporting the clinical data management process, they were similar to electronic data capture systems in many cases. The most significant dimensions of data management in such systems were data collection or entry, report, validation, and security maintenance.Conclusion:Seemingly, not sufficient attention has been paid to automate all dimensions of the clinical data management process in clinical trials. However, these systems could take positive steps towards changing the manual processes of clinical data management to electronic processes.


Author(s):  
R. A. Greenes ◽  
A. N. Pappalardo ◽  
C. W. Marble ◽  
G. O. Barnett

Author(s):  
Beth Harper ◽  
Zachary Smith ◽  
Jane Snowdon ◽  
Robert DiCicco ◽  
Rezzan Hekmat ◽  
...  

1985 ◽  
Vol 69 (5) ◽  
pp. 607-611 ◽  
Author(s):  
Pietro Delva ◽  
Mario De Gasperi ◽  
Maurizio Degan ◽  
Grazia Covi ◽  
Alessandro Lechi

1. Outward bumetanide-sensitive Na+-K+ co-transport was studied in the erythrocytes of 51 subjects, 24 normotensive subjects and 27 hypertensive patients, matched for sex and age. 2. Three kinetic parameters of this cation transport system were considered: velocity of efflux at saturating internal sodium (Nai) concentrations (Vmax.), apparent affinity for sodium (K50%) and index of co-operativity among Nai sites (Hill's n). 3. We correlated these values with clinical and laboratory data determined routinely in hypertension. 4. There were no significant differences between normotensive and hypertensive subjects for the values considered and we did not find any significant correlations between co-transport and clinical data.


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