The use of HSCT for sickle cell disease is evolving, and no randomized controlled trials were identified in the evidence review for this guideline
In a 2021 clinical guideline issued by the American Society of Hematology and published online Sept. 28 in Blood Advances, eight conditional recommendations are presented for the use of hematopoietic stem cell transplantation (HSCT) in sickle cell disease (SCD).
Julie Kanter, M.D., from the University of Alabama Birmingham, and colleagues developed evidence-based guidelines for the use of HSCT for SCD.
The researchers developed guidelines to address questions about the timing and type of HSCT for SCD that included the following recommendations. For patients with SCD who have experienced an overt stroke or have an abnormal transcranial Doppler ultrasound, human leukocyte antigen-matched HSCT is suggested rather than standard of care (hydroxyurea/transfusion; conditional recommendation based on very low certainty in the evidence). For patients with frequent pain, related matched allogeneic transplantation is suggested rather than standard of care (conditional recommendation based on very low certainty in the evidence about effects). Matched related allogeneic transplantation is suggested over standard of care for patients with recurrent episodes of acute chest syndrome (conditional recommendation based on very low certainty in the evidence about effects). Total body irradiation ≤400 cGy or chemotherapy-based conditioning regimens are suggested for allogeneic HSCT (conditional recommendation based on very low certainty in the evidence about effects).
“The use of HSCT for SCD is evolving,” the authors write. “The conditional nature of the recommendations for all questions results from the short duration of accumulated data and the reliance on evaluation of noncomparative data.”