Low-Intensity Regimens for Sickle Cell Disease Transplantation Associated With Risk of Secondary Neoplasms

Low-intensity regimens for sickle cell disease (SCD) transplantations were associated with a higher risk of secondary neoplasms, including leukemia and myelodysplastic syndromes (MDS), according to findings of an observational cohort study recently published in the Journal of Clinical Oncology. 

Dr Mary Eapen, MS, MBBS, Froedtert Hospital, Milwaukee, Wisconsin , and coauthors aimed to analyze the incidence and risk factors associated with secondary neoplasms after transplantation for SCD by observing a cohort of patients who had received transplantation as well as low-intensity treatment regimens for safety hazards. 

In this study, 1,096 SCD transplants occurring between 1991 and 2016, with 22 secondary neoplasms reported, were examined for risk factors by Fine-Gray regression models. Present secondary neoplasms included leukemia/MDS (n=15) and solid tumors (n=7). 

Results found a 10-year incidence rate of 1.7% for leukemia/MDS (95% confidence interval [CI]) and a 10-year incidence of 2.4% for any secondary neoplasm (95% CI).

Low-intensity regimens were associated with a higher risk of leukemia/MDS (hazard ratio [HR], 22.69; 95% CI) or any other secondary neoplasm (HR, 7.78; 95% CI) than other intense regimens. 

As low-intensity regimens for SCD rely on tolerance induction and establishment of mixed-donor chimerism, these regimens “may in part mitigate the higher risk for leukemia/MDS,” Dr Eapen et al concluded. 

Dr Suzanne Lentzsch, associate editor of the Journal of Clinical Oncology added, “the data will provide the foundation to discuss the risk of secondary malignancies after hematopoietic cell transplant for SCD,” adding, “it will improve the determination of the risk-benefit ratio, especially in the light of the emerging indication of gene therapy for SCD.”

Source: 

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