An ad hoc committee of the National Academies of Sciences, Engineering, and Medicine will conduct a study to review the latest published research and science and produce a report addressing best practices and community experiences in the management and treatment of sickle cell disease (SCD). The committee’s report will:

1. Describe the current healthcare landscape for the management and treatment of SCD and its symptoms for adults and children, including traditional and alternative therapies and home remedies, their prevalence, efficacy, and side-effects, and recent changes or challenges in the provision of such care;
2. Describe the typical course of an acute sickle cell crisis for adults and children, including phases of the crisis and the typical symptoms, signs, complications, functional impact, and treatment throughout the duration of the crisis, and the expected range of time to return to baseline; and
3. Answer the following questions based on published evidence (to the extent possible) and professional judgment (where published evidence is lacking):

a. When and how often is hospitalization used in the treatment of SCD, what factors impact the decision for a patient to present to a hospital for treatment, what factors lead to hospital staff’s decision on whether to admit a patient, what factors determine the length of hospitalization, what factors contribute to hospital readmissions within 30 days of discharge, are any of the discussed factors unique to SCD or used in a different way when making decisions related to hospitalization for individuals with SCD compared to individuals with other impairments, and how have these considerations changed over the past 10 years?

b. When and how often are parenteral narcotic pain medications used to manage SCD pain crises, what are indicators or contraindicators for their use, how have best practices regarding narcotic pain medication changed over the past 10 years, and what circumstances or factors might lead providers not to prescribe narcotic pain medication even when indicated by current best practices? What other pain medications might be administered in the context of a severe pain crisis and what, if anything, can the choice of alternative medications tell us about a patient’s symptom severity?

c. What treatments or interventions used for acute sickle cell crises are equivalent to parenteral narcotic medication in terms of medical severity of the underlying acute crisis (e.g., acute transfusion)?

d. How and why are regular red blood cell (RBC) transfusions used differently in the treatment of SCD and beta thalassemia? What indications or thresholds, if any, would show that SCD requiring chronic RBC transfusions had an equivalent level of medical severity to beta thalassemia requiring chronic RBC transfusions?

e. To what extent does end-organ damage in an individual with SCD contribute to the disease burden and overall severity of SCD, and how, if at all, does this vary by type of organ or organ-specific severity or diagnostic threshold? Might similar objective measurements of end-organ damage denote higher levels of severity or functional impact in an individual with SCD than in an individual without SCD?

The committee will prepare an interim report and a final report, both containing findings and conclusions but not recommendations. The interim report will present the committee’s findings and conclusions pertaining to objectives 3.a (hospitalizations) and 3.b (use of narcotic pain medications in SCD). The final report will present the committee’s findings and conclusions addressing all of the objectives in the statement of task.