Researchers reported excellent long-term survival results after hematopoietic cell transplantation in patients with sickle cell disease (SCD) who survive the first 2 years post-transplant and published their results in Transplantation and Cellular Therapy.

The study included records of 950 patients who all had survived 2 years after the transplant procedure, thus excluding those who presumably as a direct result of the transplantation.

“Hematopoietic cell transplant for [SCD] is curative but is associated with life-threatening complications, most of which occur within the first 2 years after transplantation,” the authors said. “We felt it timely to report on SCD transplant recipients who were alive for at least 2-year after transplantation, not previously reported.”

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Patients were followed up for a median of 5 years and a total of 300 were followed for more than 7 years. Among those patients surviving ≥2 years post-transplant, the probability of 10-year survival was 96%.

Age at transplantation and donor type (human leukocyte antigen-matched siblings vs other donors) were determined as risk factors for late death. Graft failure occurred more often in those donated by nonhuman leukocyte antigen-matched siblings, and no advantage was observed among the alternative donors in terms of survival. Ultimately, the risk of death declined over time, but it remained higher than that of the general US population.

The authors expect the durable survival of transplanted patients with SCD to inform emerging curative therapies including gene editing and gene therapy. However, they caution that deaths were observed several years post-transplant, highlighting the need for long-term and close monitoring of these patients.

Reference

StMartin A, Hebert KM, Serret-Larmande A, et al. Long-term survival after hematopoietic cell transplant for sickle cell disease compared to the United States population. Transplant Cell Ther. Published online March 14, 2022. doi:10.1016/j.jtct.2022.03.014