Low-intensity, nonmyeloablative conditioning regimens were associated with a greater risk for secondary neoplasms, including leukemia or myelodysplastic syndrome (MDS), among patients with sickle cell disease (SCD) who underwent hematopoietic stem cell transplant (HSCT), according to the results of a study published in the Journal of Clinical Oncology.

Although HSCT can be curative for children with SCD, there is risk for substantial potential morbidities, including secondary neoplasms, and death. The aim of this study was to determine the incidence and risk factors for secondary neoplasms after HSCT for SCD.

The study evaluated data from 1096 transplants for SCD from a publicly-available data source that occurred between 1991 and 2016. The cohort included 22 patients who developed a secondary neoplasm and 1074 patients who did not.

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The median age of the cohort was 15 and 51% of patients were male. There were 45% of patients who developed a secondary neoplasm and 11% who did not whose donor was a haploidentical relative. Peripheral blood was the stem cell source in 64% of cases and 16% of patients without secondary neoplasm, whereas bone marrow was used for 27% and 71%, respectively.

The 10-year incidence for secondary neoplasms was 2.4%, including 1.7% for leukemia or MDS and 0.7% for solid tumors. The median time to diagnosis of a secondary neoplasm was 40 months.

The use of a low-intensity, nonmyeloablative conditioning regimen was significantly associated with a higher risk for secondary neoplasms (hazard ratio [HR], 7.78; 95% CI, 2.20-27.53; P =.0015) and leukemia or MDS (HR, 22.69; 95% CI, 4.34-118.66; P =.0002) compared with a myeloablative regimen.

The authors concluded that “choosing conditioning regimens likely to result in full-donor chimerism may in part mitigate the higher risk for leukemia or MDS.”

Disclosures: Some of the study authors declared affiliations with biotech, pharmaceutical, or device companies. Please see the original reference for a full list of disclosures.


Eapen M, Brazasukas R, Williams DA, et al. Secondary neoplasms after hematopoietic cell transplant for sickle cell disease. J Clin Oncol. Published online January 9, 2023. doi: 10.1200/JCO.22.01203