Unmet Need

Despite an increase in treatments for sickle cell disease (SCD), a large gap in life expectancy persists between those with SCD and the general population1

Average life expectancy for people with SCD vs general population2-8

Line graph showing a persistent life-expectancy gap between people with sickle cell disease and the general populationLine graph showing a persistent life-expectancy gap between people with sickle cell disease and the general population

HSCT=hematopoietic stem cell transplantation.

Current disease-modifying treatments can help manage SCD, but significant need remains to specifically address hemolysis and low hemoglobin9,10

Most common disease-modifying treatments focus on preventing VOCs. They include9:

Hydroxyurea10,11

  • Modifies disease by increasing the level of fetal hemoglobin and reducing HbS polymerization and RBC sickling
  • Indicated to reduce the frequency of painful crises and reduce the need for blood transfusions in sickle cell anemia

Crizanlizumab10,12

  • Modifies disease by decreasing blood cell adhesion
  • Indicated to reduce the frequency of VOCs

L-glutamine10,13

  • Modifies disease by improving redox ratio to reduce oxidative stress and RBC sickling
  • Indicated to reduce the acute complications of sickle cell disease

Blood transfusion14

  • Used to increase Hb levels or to address certain SCD complications
  • RBC transfusions can help complications like acute chest syndrome, acute splenic sequestration, and stroke; however, patients require monitoring for alloimmunization, iron overload, and transfusion reactions

Disease-modifying treatments specifically designed to address hemolysis and low hemoglobin are critically needed and are
in development.9,15

Hb=hemoglobin; HbS=sickle hemoglobin; RBC=red blood cell; VOC=vaso-occlusive crisis.

References:

1. Jiao B, Johnson KM, Ramsey SD, Bender MA, Devine B, Basu A. Long-term survival with sickle cell disease: a nationwide cohort study of Medicare and Medicaid beneficiaries. Blood Adv. 2023;7(13):3276-3283. doi:10.1182/bloodadvances.2022009202 2. Karkoska KA, McGann PT. Trends in sickle cell disease mortality: 1979-2020. Pediatrics. 2024;154(6):e2024067341. doi:10.1542/peds.2024-067341 3. McGough M. How does U.S. life expectancy compare to other countries? Peterson-KFF Health System Tracker. Accessed July 21, 2026. www.healthsystemtracker.org/chart-collection/u-s-life-expectancy-compare-countries/ 4. Mortality in the United States, 2020. Centers for Disease Control and Prevention. National Center for Health Statistics. Accessed July 21, 2026. https://www.cdc.gov/nchs/products/databriefs/db427.htm 5. Wailoo K. Sickle cell disease—a history of progress and peril. N Engl J Med. 2017;376(9):805-807. doi:10.1056/NEJMp1700101 6. Mortality trends in the United States, 1900-2018. Centers for Disease Control and Prevention. Accessed July 7, 2026. www.cdc.gov/nchs/data-visualization/mortality-trends/index.htm 7. Chakravarthy R, Friedman DL. Long-term health outcomes following curative therapies for sickle cell disease. Hematology Am Soc Hematol Educ Program. 2022;2022(1):272-276. doi:10.1182/hematology.2022000373 8. History of sickle cell disease. American Red Cross. Accessed July 7, 2026. www.redcrossblood.org/donate-blood/blood-types/diversity/african-american-blood-donors/history-of-sickle-cell-disease.html 9. Xu JZ, Thein SL. Revisiting anemia in sickle cell disease and finding the balance with therapeutic approaches. Blood. 2022;139(20):3030-3039. doi:10.1182/blood.2021013873 10. Tanhehco YC, Nathu G, Vasovic LV. Development of curative therapies for sickle cell disease. Front Med. 2022;9:1055540. doi:10.3389/fmed.2022.1055540 11. DROXIA. Prescribing Information. Bristol-Myers Squibb Company; 2021. 12. ADAKVEO. Prescribing Information. Novartis Pharmaceuticals Corporation; 2024. 13. ENDARI. Prescribing Information. Emmaus Medical, Inc.; 2025. 14. Evidence-based management of sickle cell disease: expert panel report, 2014. National Institutes of Health. National Heart, Lung, and Blood Institute. Accessed July 7, 2026. http://www.nhlbi.nih.gov/health-pro/guidelines/sickle-cell-disease-guidelines 15. Kavanagh PL, Fasipe TA, Wun T. Sickle cell disease: a review. JAMA. 2022;328(1):57-68. doi:10.1001/jama.2022.10233