Most patients had no sickle cell complications post-HCT; complications included osteonecrosis, vaso-occlusive crisis, nephropathy, stroke
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.
Elizabeth Stenger, M.D., from the Emory University School of Medicine in Atlanta, and colleagues examined the cumulative incidence of late effects estimated seven years post-HCT among patients who underwent curative HCT for SCD.
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.
“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.”
Several authors disclosed ties to the biopharmaceutical industry.
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