Addressing Sickle Cell Disease : A Strategic Plan and Blueprint for Action

Sickle cell disease (SCD) is a genetic condition that affects approximately 100,000 people in the United States and millions more globally.

Individuals with SCD endure the psychological and physiological toll of repetitive pain as well as side effects from the pain treatments they undergo.

Some adults with SCD report reluctance to use health care services, unless as a last resort, due to the racism and discrimination they face in the health care system.

Additionally, many aspects of SCD are inadequately studied, understood, and addressed. Addressing Sickle Cell Disease examines the epidemiology, health outcomes, genetic implications, and societal factors associated with SCD and sickle cell trait (SCT).

This report explores the current guidelines and best practices for the care of patients with SCD and recommends priorities for programs, policies, and research.

It also discusses limitations and opportunities for developing national SCD patient registries and surveillance systems, barriers in the healthcare sector associated with SCD and SCT, and the role of patient advocacy and community engagement groups. Table of ContentsFront MatterSummary1 Introduction2 Societal and Structural Contributors to Disease Impact3 Screening, Registries, and Surveillance4 Complications of Sickle Cell Disease and Current ManagementApproaches5 Health Care Organization and Use6 Delivering High-Quality Sickle Cell Disease Care with a PreparedWorkforce7 Developing and Delivering the Next Generation of Therapies8 Community Engagement and Patient Advocacy9 Strategic Plan and Blueprint for Sickle Cell Disease ActionAppendix A: Public Meeting Agendas and Submissions to the CommitteeAppendix B: Literature Search Terms and StrategyAppendix C: Committee and Staff BiographiesAppendix D: Newborn Screening Results Reporting Protocols forSickle Cell Disease and Sickle Cell TraitAppendix E: Sickle Cell Data Collection ProgramAppendix F: Georgia Comprehensive Sickle Cell Center: A Case StudyAppendix G: Emory Adult Cystic Fibrosis ProgramAppendix H: Health Resources and Services Administration SickleCell Disease ProgramsAppendix I: Select Treatments Currently Under Development forSickle Cell DiseaseAppendix J: Other Training Models for HematologistsAppendix K: Sickle Cell Community-Based Organizations and PatientGroups in the United StatesAppendix L: Summary Table of Strategic Plan and Blueprint forSickle Cell Disease ActionAppendix M: Summary Table of Sickle Cell Trait Discussion in ReportAppendix N: Glossary