ORCID Profile
0000-0002-6655-3797
Current Organisations
University of Lausanne
,
The Bluefield Project to Cure FTD
,
Centre Hospitalier Universitaire Vaudois
,
Istituto Pediatrico della Svizzera Italiana
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Publications
The role of neurofilament light in genetic frontotemporal lobar degeneration
Publisher: Oxford University Press (OUP)
Date: 26-11-2022
DOI: 10.1093/BRAINCOMMS/FCAC310
Abstract: Genetic frontotemporal lobar degeneration caused by autosomal dominant gene mutations provides an opportunity for targeted drug development in a highly complex and clinically heterogeneous dementia. These neurodegenerative disorders can affect adults in their middle years, progress quickly relative to other dementias, are uniformly fatal and have no approved disease-modifying treatments. Frontotemporal dementia, caused by mutations in the GRN gene which encodes the protein progranulin, is an active area of interventional drug trials that are testing multiple strategies to restore progranulin protein deficiency. These and other trials are also examining neurofilament light as a potential biomarker of disease activity and disease progression and as a therapeutic endpoint based on the assumption that cerebrospinal fluid and blood neurofilament light levels are a surrogate for neuroaxonal damage. Reports from genetic frontotemporal dementia longitudinal studies indicate that elevated concentrations of blood neurofilament light reflect disease severity and are associated with faster brain atrophy. To better inform patient stratification and treatment response in current and upcoming clinical trials, a more nuanced interpretation of neurofilament light as a biomarker of neurodegeneration is now required, one that takes into account its relationship to other pathophysiological and topographic biomarkers of disease progression from early presymptomatic to later clinically symptomatic stages.
Results of an international survey on adherence with anticoagulation in children, adolescents, and young adults: Communication from the ISTH SSC Subcommittee on Pediatric and Neonatal Thrombosis and Hemostasis.
Publisher: Elsevier BV
Date: 07-2022
DOI: 10.1111/JTH.15730
Abstract: The ISTH Scientific and Standardization Committee (SSC) Subcommittee on Pediatric/Neonatal Thrombosis and Hemostasis convened a working group on medication adherence to begin to understand the current state of clinical practice to inform priority areas for efforts to improve adherence for children, and adolescents and young adults (AYA) prescribed anticoagulants. We sought to survey an international group of clinicians involved in anticoagulation management in children and/or AYA about perceptions of medication on health outcomes, clinical practice related to medication adherence, and barriers to assessing and improving medication adherence. Clinicians involved in anticoagulation management in children and/or AYA were surveyed via REDCap There were 200 participants, 90% of whom were pediatric hematology/oncology physicians. Based on the results, which demonstrate that clinicians are concerned about impact of poor medication adherence and have limited resources to identify and improve adherence, the working group has identified next steps to further understand impact of medication adherence on anticoagulation-related health outcomes, address the need for validated measures to assess medication adherence for all anticoagulants prescribed to this population, and develop an intervention and implementation research agenda to improve outcomes.
Recommendations for standardized definitions, clinical assessment, and future research in pediatric clinically unsuspected venous thromboembolism: Communication from the ISTH SSC subcommittee on pedia
Publisher: Elsevier BV
Date: 07-2022
DOI: 10.1111/JTH.15731
Related Organisations
Related Funding Activities
No related grants have been discovered for Rodney Pearlman.