Disease-Specific Indications and Outcomes

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Sickle Cell Disease

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 potential cure for SCD. [2-7]

Two ongoing prospective clinical trials on HCT to treat SCD

The STRIDE2 trial (BMT CTN 1503) is a phase II, multi-center clinical trial of HCT versus standard of care in adolescents and young adults with SCD comparing 2-year overall survival of the two treatment arms. This trial is currently enrolling patients and has been approved by Medicaid for transplant insurance coverage of Medicaid-eligible beneficiaries who participate in this Coverage with Evidence Development clinical trial.

BMT CTN 1507 is a Phase II, single arm, multi-center trial, designed to estimate the efficacy and toxicity of haploidentical HCT in two groups of patients with SCD: 1) children with SCD with strokes; and 2) adults with severe SCD.

For more information, visit bmtctn.net.

Advances

 

Recent research provides new data for clinical decision-making in patients with SCD:

  • Related donor HCT for SCD can yield overall survival of >90% in both adults and children [2-7]
  • Adults with high-risk SCD can be effectively treated using a non-myeloablative HCT conditioning regimen [3]
  • HCT for SCD leads to lower post-transplant health care utilization and improved quality of life compared to non-HCT controls [8,9]

The Sickle Cell Unrelated Transplant (SCURT) study (BMT CTN 0601) evaluated the role of unrelated donor HCT in treating severe SCD and in children with a history of severe SCD manifesting as strokes, frequent pain crises, or acute chest syndrome. Results of the SCURT study were published in the journal Blood in November 2016. [9]

On-demand CME: Improving SCD Outcomes

Mark C. Walters, M.D., Gregory J. Kato, M.D., Lakshmanan Krishnamurti, M.D., Marsha Treadwell, Ph.D., and Linda J. Burns, M.D explore new models for clinical decision-making in SCD, clinical trials, including those for HCT, and strategies for overcoming barriers to patient care. Transplant recipient, Ines Lukombo, shares how she worked with her care team to choose HCT as a cure for her SCD.

Access CME >

 

Outcomes

 

Review outcomes for allogeneic transplantation in patients with SCD below. View additional slides showing demographic data and hematopoietic cell transplant trends.

Data in this section have been prepared by CIBMTR® (Center for International Blood and Marrow Transplant Research), our research program.

Figure 1: Pediatric SCD Survival, Unrelated HCT

75-pediatric-sickle-cell-disease-survival-unrelated-marrow-hct.jpg

Download slide "Pediatric SCD Survival, Unrelated HCT"

Referral Timing Guidelines

 

These guidelines highlight disease categories that include patients at risk for disease progression and who should be referred for a consultation for hematopoietic cell transplantation. [10]

Transplant Consultation Guidelines: Hemoglobinopathies

  • Sickle Cell Disease
    • With aggressive course (stroke, end-organ complications, frequent pain crises)
     
  • Transfusion-Dependent Thalassemias
    • At diagnosis
     
Download as slides (PPT)
View complete Referral Timing Guidelines


References

  1. Centers for Disease Control and Prevention: Sickle Cell Disease, Data and Statistics; accessed 13 January, 2018. Access
  2. 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
  3. 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
  4. 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
  5. 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
  6. 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
  7. 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
  8. Arnold SD, Jin Z, Sands S, et al. Allogeneic hematopoietic cell transplantation for children with sickle cell disease is beneficial and cost-effective: A single-center analysis. Biol Blood Marrow Transplant. 2015; 21(7): 1258-1265. Access
  9. Shenoy S, Eapen M, Panepinto JA, et al. A BMT CTN phase II trial of unrelated donor marrow transplantation for children with severe sickle cell disease. Blood. 2016; 128(21): 2561-2567. Access
  10. NMDP/Be The Match and ASBMT Recommended Timing for Transplant Consultation, 2018. Download (PDF)