Most pediatric patients with sickle cell disease (SCD) showed normal or stable to improved organ function after hematopoietic cell transplantation (HCT), according to a new retrospective study published in Transplantation and Cellular Therapy.

In addition, the study identified central nervous system indication, severe clinical phenotype, older age at transplantation, use of myeloablative conditioning, and development of acute graft-versus-host disease (GVHD) as predictors for organ dysfunction.

Overall organ dysfunction was associated with 16 years old or more (odds ratio [OR], 2.26, 95% CI, 1.35-3.78, P =.002) and severe clinical phenotype (OR, 1.64, 95% CI, 1.02-2.63, P =.043). Moreover, cardiac dysfunction was associated with myeloablative conditioning (OR, 2.71, 95% CI, 1.09-6.77, P =.033) and severe acute GVHD (OR, 2.41, 95% CI, 1.04-5.62, P =.041), whereas neurologic events were associated with central nervous system indication (OR, 2.88, 95% CI, 2.00-4.12, P <.001).

“Overall, our results support consideration of decreased conditioning intensity (balanced against risk for graft rejection) and younger age at HCT, and may provide data for updated expert guidelines,” the authors wrote.

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The proportion of patients with low ejection or shortening fractions was higher post-HCT than pre-HCT (6.0% vs 0.6% and 4.6% vs 0%, respectively). On the other hand, no significant difference was observed in the proportion of patients with lung disease.

Posterior reversible encephalopathy syndrome was a common HCT outcome. More than one-half (56%) of patients presenting with this syndrome or seizures had a central nervous system HCT indication. Eight patients had a stroke at a median of 47 days post-HCT. Most (78.6%) patients had normal or stable brain MRI scan; however, a minor proportion of patients developed new overt cerebral infarct, progressive (silent) cerebral infarct, or new or progressive vasculopathy.

Moreover, 13 patients died more than 1 year after HCT, at a median age of 20.1 years. The most common cause of death was GVHD (n=7), followed by infection (n=4). Acute and chronic GVHD post-HCT was observed in 24.0% and 24.8% of patients, respectively.

The retrospective study included 247 patients (median age at HCT, 9.4 years) whose registry was available from the Sickle Cell Transplant Advocacy and Research Alliance. Most patients had HbSS genotype (88.2%) and severe clinical phenotype (65.4%). Furthermore, most (76.9%) patients received cells from a matched related donor.


Stenger E, Xiang Y, Wetzel M, et al. Long-term organ function after HCT for SCD: a report from the Sickle cell Transplant Advocacy and Research alliance. Transplant Cell Ther. Published online October 20, 2022. doi:10.1016/j.jtct.2022.10.012