Stem Cell Transplantation Guidelines

Hematopoietic stem cell transplant (HSCT), also known as bone marrow transplant, is currently the only well-established treatment that can potentially cure sickle cell disease. In HSCT, unhealthy blood-forming cells are replaced with healthy cells from a donor. 

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Who Should Consider Transplant?

 

ASH guidelines suggest transplant may be considered in several situations, especially when the benefits are likely to outweigh the risks:

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• Stroke or High Stroke Risk:
  HSCT is recommended over usual care (like transfusion therapy) for people who have had a stroke, have a high risk of stroke, or show clear evidence of neurologic damage. This is especially true for children when a matched sibling donor is available. 

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• Frequent Pain Crises:
  For individuals who continue to have frequent pain episodes despite other treatments, transplant from a matched sibling donor may be considered.

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• Recurrent Acute Chest Syndrome:
  Transplant is also suggested for people who have repeated episodes of acute chest syndrome that don’t respond well to other therapies. 

 

What About Donor Options?

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• Matched Sibling Donor (MSD): The guidelines support using a donor who is a close genetic match (usually a brother or sister). Matched sibling transplants generally have better outcomes. 

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• Alternative Donors: If a matched sibling donor isn’t available, using other donors (such as unrelated or partially matched donors) may be considered only within clinical trials so that outcomes and safety can be clearly studied. 

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