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Sickle cell disease (SCD)

Transplant advances and outcomes

Sickle cell disease (SCD) affects approximately 100,000 people in the United States. It is found primarily in people whose ancestors originated in Sub-Saharan Africa, South and Central America, the Caribbean, Saudi Arabi, India and Mediterranean countries. More than 90% of those affected in the U.S. are non-Hispanic Black. [1]

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, there are only three potentially curative options: allogeneic hematopoietic cell transplantation (HCT) or two gene therapies recently approved by the FDA. [2-8]

In a 2022 analysis, the 10-year survival for SCD patients who survived at least two years after HCT was 96%. [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)

Support for patients considering HCT for sickle cell disease

NMDP works as an extension of hematology/oncology practices to address patient needs beyond clinical care. The NMDP Patient Support Center team provides tailored information and assistance to patients considering HCT as a treatment option, preparing for HCT or who had HCT. There is no cost to the patient or the hematology/oncology practice.

Patients and their loved ones can receive:

  • Step-by-step guidance from an NMDP patient navigator who specializes in HCT
  • Emotional and practical support
  • Navigation in their language from bilingual NMDP navigators and social workers (English and Spanish) and interpreter services
Explore the patient programs
  • Financial grants, including a Sickle Cell Disease Fertility Preservation Grant
  • Patient-friendly education in multiple languages and formats, including videos in English and Spanish
  • Peer support from patients and caregivers who have been through transplant

References

  1. Centers for Disease Control and Prevention: Data and statistics on sickle cell disease. Accessed July 9, 2025. Access
  2. St. Martin, A. Hebert KM, Serret-Larmande 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. US Food & Drug Administration: FDA approves first gene therapies to treat patients with sickle cell disease. Accessed July 9, 2025. Access
  9. 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
  10. NMDP and ASTCT Recommended Timing for Transplant Consultation. Download PDF