Designed and created by Guideline Central in participation with the American Society of Hematology
Sickle Cell Disease: Stem Cell Transplantation
Patient Guideline Summary
Publication Date: September 28, 2021
Last Updated: March 3, 2023
This patient summary means to discuss key recommendations from the American Society of Hematology (ASH) for stem cell transplantation for sickle cell disease.
- Sickle cell disease is an inherited genetic defect that causes red blood cells to deform under certain conditions. Those deformed cells plug small blood vessels, causing tissue damage.
- We will use the abbreviation SCD throughout this summary to refer to sickle cell disease and ASH for the American Society of Hematology.
- Note: symptoms include anemia, episodic pain, hand and foot swelling, delayed growth, and disturbed vision.
- This patient summary focuses on the management of sickle cell disease.
- Standard treatment is a drug called hydroxyurea combined with repeated red blood cell transfusions.
- Currently, the cure is possible only by transplanting normal stem cells.
- Hematopoietic stem cell transplantation (HSCT) is a major and high-risk process. Therefore, its benefits must exceed its risk to justify its use.
ASH prefers sibling umbilical cord blood (UBC) over bone marrow if it is available. (There are “banks” that store UBC for this use.)
- ASH considers evidence of brain injury, frequent pain, or recurring “acute chest syndrome” (ACS) the sufficient justification for HSCT.
- Stem cells from blood relatives, preferably siblings, produce the best results, as does performing HSCT before the age of 16.
- Reduction of abnormal stem cells in the patient must precede the transplant. This process, called “conditioning,” can be partial or complete.
- Both can be achieved by adjusting the doses of total body irradiation or marrow-toxic drugs (melphalan and fludarabine) or both.
- ASH generally prefers myeloablative (complete elimination) for children and nonmyeloablative (partial elimination) for adults.
- In all cases, the benefits from HSCT must be carefully and thoroughly evaluated to assure that they exceed the risks.
- ACS: Acute Chest Syndrome
- ASH: American Society Of Hematology
- HSCT: Hematopoietic Stem Cell Transplantation
- SCD: Sickle Cell Disease
- UBC: Umbilical Cord Blood
Kanter J, Liem RI, Bernaudin F, Bolaños-Meade J, Fitzhugh CD, Hankins JS, Murad MH, Panepinto JA, Rondelli D, Shenoy S, Wagner J, Walters MC, Woolford T, Meerpohl JJ, Tisdale J. American Society of Hematology 2021 guidelines for sickle cell disease: stem cell transplantation. Blood Adv. 2021 Sep 28;5(18):3668-3689. doi: 10.1182/bloodadvances.2021004394C. PMID: 34581773.
The information in this patient summary should not be used as a substitute for professional medical care or advice. Contact a health care provider if you have questions about your health.