Novel Biomarkers and Surrogate Endpoints Clarify Sickle Cell Disease Status and the Effects of Disease Modifying Therapies

Novel Biomarkers and Surrogate Endpoints Clarify Sickle Cell Disease Status and the Effects of Disease Modifying Therapies

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We will have an exhibit in the Caribbean Ballroom please stop by to see us!

We will be leading an educational seminar Saturday 3:30-5:30: Novel Biomarkers and Surrogate Endpoints Clarify Sickle Cell Disease Status and the Effects of Disease Modifying Therapies

https://sched.co/NRdQ

The clinical pain and fatigue characteristic of sickle cell disease is difficult to objectively quantify.  Clinicians cannot depend on their physical exam, imaging studies, or laboratory data to validate claims of pain by their patients.  This challenge often creates physician and patient dissatisfaction, and makes it difficult to assess individual patient response to SCD modifying therapy in clinical trials. Biomarkers associated with cellular adhesion, inflammation, coagulation, anemia/hemolysis, cellular aggregates, microparticles and nitric oxide metabolism may help to objectively assess patient-reported outcomes (PROs) and response to disease modifying therapy.  There is an urgent need for validated biomarkers in sickle cell disease that facilitate open communication between the healthcare team, industry, and individuals with SCD.  We will highlight the current “state of the art” in the application of biomarkers and surrogate endpoints as applied to SCD in this educational session.

Presenters:


Patrick Hines, MD/PhD
Associate Professor of Pediatric Critical Care Medicine
Children’s Hospital of Michigan
Research Associate, Department of Physiology, WSU School of Medicine
Functional Fluidics, Founder and CEO
Industry Perspective on Biomarkers and Surrogate Endpoints in SCD Clinical Trials

Kenneth R. Bridges, MD
Vice President of Medical Affairs
Global Blood Therapeutics
 Industry Perspective on Biomarkers and Surrogate Endpoints in Clinical Practice

Charles Stark, PharmD
Senior Vice President of Research and Development
Emmaus Life Sciences, Inc

We will be presenting an Abstract on Sunday from 3:30-5:00

3. JSCDH-D-19-00036 "Development and Evaluation of a Sickle Cell Disease Severity Index to Validate Clinical Adhesion Biomarkers"
Corresponding author:  Jennell White, PhD

https://sched.co/LVKm

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