Sickle Cell Disease: Stem Cell Transplantation

Patient Guideline Summary

Publication Date: September 27, 2021

Last Updated: March 3, 2023

Objective

This patient summary means to discuss key recommendations from the American Society of Hematology (ASH) for stem cell transplantation for sickle cell disease.

Overview

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.

Treatment

Standard treatment is a drug called hydroxyurea combined with repeated red blood cell transfusions.

This does not cure the disease; it only reduces symptoms.

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.

Donors must be matched to prevent immune reactions, as in other blood transfusions.
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.

ACS is sudden chest pain, cough, fever, low oxygen levels, and lung damage.

Stem cells from blood relatives, preferably siblings, produce the best results, as does performing HSCT before the age of 16.

If a matched sibling donor is not available, using an alternative donor should take place in a clinical trial.

HSCT Procedure

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.

Abbreviations

ACS: Acute Chest Syndrome
ASH: American Society Of Hematology
HSCT: Hematopoietic Stem Cell Transplantation
SCD: Sickle Cell Disease
UBC: Umbilical Cord Blood

Source Citation

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.

Disclaimer

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.