Sickle Cell Disease Stem Cell Transplantation Clinical Practice Guidelines (ASH, 2021)

American Society of Hematology (ASH)

These are some of the highlights of the guidelines without analysis or commentary. For more information, go directly to the guidelines by clicking the link in the reference.

October 29, 2021

Clinical practice guidelines on sickle cell disease stem cell transplantation by the American Society of Hematology (ASH) were published in September 2021 in Blood Advances.[1]

For patients with sickle cell disease who have had an overt stroke or who have abnormal transcranial Doppler ultrasound scan results, human leukocyte antigen (HLA)-matched related hematopoietic stem cell transplantation (HSCT) is suggested as an alternative to the standard of care (hydroxyurea and/or transfusion).

Related matched allogeneic transplantation may be considered for patients with sickle cell disease who have frequent pain that resists standard therapies, such as hydroxyurea.

For patients who have recurrent episodes of acute chest syndrome despite receiving standard treatments (eg, hydroxyurea, L-glutamine, crizanlizumab, and long-term transfusion), matched related allogeneic transplantation is suggested.

If HSCT is indicated, but the patient does not have a matched sibling donor, alternative donor transplantation may be considered.

Either total body irradiation (≤400 cGy) or chemotherapy-based conditioning is suggested for patients who are receiving allogeneic HSCT.

For children with sickle cell disease who have an indication for allogeneic HSCT and a matched sibling donor, myeloablative conditioning is suggested, instead of reduced-intensity conditioning that includes melphalan/fludarabine regimens. For adults, nonmyeloablative conditioning is suggested.

Performing allogeneic transplantation at a younger age is suggested for patients who are eligible for HSCT.

When HLA-identical sibling cord blood is available and is associated with an adequate cord blood cell dose and good viability, its use is suggested instead of bone marrow.

For more information, please go to Sickle Cell Anemia.


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