nmdp logo

Sickle cell disease (SCD)

Transplant advances and outcomes

Sickle cell disease (SCD) affects approximately 100,000 Americans and occurs in about 1 out of every 365 African American births. [1]  

This hemoglobinopathy is primarily found in individuals with African, Mediterranean, South American, Southeast Asian, and Middle Eastern lineages. SCD leads to lifelong morbidity and reduced life expectancy through end-organ damage. 

Although supportive care, drug therapies, and red blood cell transfusions can ease symptoms and extend lifespan, allogeneic hematopoietic cell transplantation (HCT) is the only established potential cure for SCD. [2-7] The 10-year survival for patients who survive at least two years after HCT was 96% in a recent analysis.[2]

Recommended timing for transplant consultation

  • Children (especially under age 13) with available matched sibling donors should be referred at diagnosis
  • All patients with aggressive course (stroke, end-organ complications, frequent pain crises)
  • All patients with an alternative donor option and any of the following:
    • Stroke or silent cerebral infarct or cognitive impairment >24 hours
    • Abnormal transcranial Doppler (TCD) velocity of ≥ 200 cm/sec or > 185 cm/sec with intracranial vasculopathy
    • Frequent episodes of acute chest syndrome or severe vaso-occulusive pain crises or combination of both in the preceding 2–3 years
    • Regular red blood cell transfusions to prevent sickle cell disease complications
    • Tricuspid value regurgitant jet (TRJ) velocity ≥2.7 m/sec (mainly in adults)
    • Chronic pain ≥6 months (leg ulcers, avascular necrosis)

References

  1. Centers for Disease Control and Prevention: Sickle Cell Disease, Data and Statistics; accessed 13 January, 2018. Access 
  2. St. Martin, A. et al. Long-term survival after hematopoietic cell transplant for sickle cell disease compared to the United States population. Transplantation and Cellular Therapy. 2022; 28(6): 325.e1–325.e7. Access 
  3. Walters MC, De Castro LM, Sullivan KM, et al. Indications and results of HLA-identical sibling hematopoietic cell transplantation for sickle cell disease. Biol Blood Marrow Transplant. 2016; 22(2): 207-211. Access
  4. Saraf SL, Oh AL, Patel PR, et al. Nonmyeloablative stem cell transplantation with alemtuzumab/low-dose irradiation to cure and improve the quality of life of adults with sickle cell disease. Biol Blood Marrow Transplant. 2016; 22(3): 441-448. Access
  5. Bhatia M, Jin Z, Baker C, et al. Reduced toxicity, myeloablative conditioning with BU, fludarabine, alemtuzumab and SCT from sibling donors in children with sickle cell disease. Bone Marrow Transplant. 2014; 49(7): 913-920. Access 
  6. King AA, Kamani N, Bunin N, et al. Successful matched sibling donor marrow transplantation following reduced intensity conditioning in children with hemoglobinopathies. Am J Hematol. 2015; 90(12): 1093-1098. Access
  7. Lucarelli G, Isgrò A, Sodani P, et al. Hematopoietic SCT for the Black African and non-Black African variants of sickle cell anemia. Bone Marrow Transplant. 2014; 49(11): 1376-1381. Access
  8. Gluckman E, Cappelli B, Bernaudin F, et al. Sickle cell disease: an international survey of results of HLA-identical sibling hematopoietic stem cell transplantation. Blood. 2017; 129(11): 1548-1556. Access
  9. NMDP and ASTCT Recommended Timing for Transplant Consultation. Download PDF 
  10. National Comprehensive Cancer Network. NCC Guidelines. 2023. Access