Most patients had no sickle cell complications post-HCT; complications included osteonecrosis, vaso-occlusive crisis, nephropathy, stroke<\/p>\n<\/h3>\n
By Elana Gotkine HealthDay Reporter<\/b>
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WEDNESDAY, Dec. 10, 2025 (HealthDay News) — Late effects are rare for patients with sickle cell disease (SCD) undergoing allogeneic hematopoietic cell transplantation (HCT), according to a study presented at the annual meeting of the American Society of Hematology, held from Dec. 6 to 9 in Orlando, Florida.<\/p>\n
Elizabeth Stenger, M.D., from the Emory University School of Medicine <\/em>in Atlanta, and colleagues examined the cumulative incidence of late effects estimated seven years post-HCT among patients who underwent curative HCT for SCD.<\/p>\n A total of 1,013 patients with SCD underwent first HCT at 112 centers at a median age of 12 years. The researchers found that at a median follow-up of 60 months, 86 percent of patients remained cured and most remained free of acute or chronic graft-versus-host disease (70 and 74 percent, respectively). Most patients had no SCD complications post-HCT (74 percent); osteonecrosis, vaso-occlusive crisis, nephropathy, stroke, acute coronary syndrome, and priapism were reported as complications (4, 1, 1, 1, 1, and <1 percent, respectively). The most common late effects at seven years post-HCT were liver toxicity, pulmonary toxicity, diabetes, gonadal dysfunction, avascular necrosis, and renal failure requiring dialysis (10, 8, 6, 6, 4, and 2 percent, respectively). Two percent of patients developed malignancy post-HCT. Specific late effects were significantly associated with factors such as older age at HCT, hydroxyurea pre-HCT, recipient female sex, recipient cytomegalovirus-positive status, year of HCT, and alternative donor.<\/p>\n “Right now, allogeneic hematopoietic cell transplantation is the only known and available option for this population capable of eliminating the full spectrum of disease symptoms,” Stenger said in a statement. “Especially if we do it while patients are young or before the onset of organ damage, these patients can go on to live much more normal lives.”<\/p>\n Several authors disclosed ties to the biopharmaceutical industry.<\/p>\n