Updates on Sickle Cell Disease

This program will include a discussion of data that were presented in abstract form. These data should be considered preliminary until published in a peer-reviewed journal.

Outline

US Sickle Cell Disease Population

SCD and Mortality in the United States

Distribution of SCD Population in the United States

Pathophysiology Overview

Pathogenesis of SCD

Old View of SCD

Adhesion in the SCD Microvasculature

Molecular Pathophysiology

Pain in Sickle Cell Disease

Pain in Sickle Cell Disease (cont)

Hospital Visits of Variable Frequency

The Iceberg Proportion of Days in Pain, Crisis, Utilization

SCD Is a Multi-System Disease

Clinical Manifestations of SCD

Barriers to Care

Negative Attitude and Stigma

Barriers to Care in Sickle Cell Disease

Hydroxyurea Mainstay of SCD Therapy

Blood Transfusion Management in SCD

L-glutamine Recently FDA-Approved for SCD

Targets for Improvement

Targets for Improvement

Targets for Improvement

Targets for Improvement

Targets for Improvement

Selectins Mediate WBC Adhesion, Rolling

Crizanlizumab Background

SUSTAIN Trial

SUSTAIN Trial Time to First SCPC Significantly Increased

Rivipansel

Nitric Oxide in SCD

Nitric Oxide in SCD (cont)

Voxelotor

Voxelotor HOPE Study Part A

Stem Cell Transplant

Sickle Cell Disease Is Potentially Amenable to Gene Therapy

Overview of Gene Therapy for SCD

Lentiglobin Gene Therapy Overview in Patients With SCD

What About Gene Editing

Abbreviations