Addressing Sickle Cell Disease

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 Contents Front Matter Summary 1 Introduction 2 Societal and Structural Contributors to Disease Impact 3 Screening, Registries, and Surveillance 4 Complications of Sickle Cell Disease and Current Management Approaches 5 Health Care Organization and Use 6 Delivering High-Quality Sickle Cell Disease Care with a Prepared Workforce 7 Developing and Delivering the Next Generation of Therapies 8 Community Engagement and Patient Advocacy 9 Strategic Plan and Blueprint for Sickle Cell Disease Action Appendix A: Public Meeting Agendas and Submissions to the Committee Appendix B: Literature Search Terms and Strategy Appendix C: Committee and Staff Biographies Appendix D: Newborn Screening Results Reporting Protocols for Sickle Cell Disease and Sickle Cell Trait Appendix E: Sickle Cell Data Collection Program Appendix F: Georgia Comprehensive Sickle Cell Center: A Case Study Appendix G: Emory Adult Cystic Fibrosis Program Appendix H: Health Resources and Services Administration Sickle Cell Disease Programs Appendix I: Select Treatments Currently Under Development for Sickle Cell Disease Appendix J: Other Training Models for Hematologists Appendix K: Sickle Cell Community-Based Organizations and Patient Groups in the United States Appendix L: Summary Table of Strategic Plan and Blueprint for Sickle Cell Disease Action Appendix M: Summary Table of Sickle Cell Trait Discussion in Report Appendix N: Glossary