Safety of Low-Intensity Blood Stem Cell Transplants for Lung Health in Sickle Cell Disease: Study

Safety of Low-Intensity Stem Cell Transplants for Sickle Cell

A study published in the Annals of the American Thoracic Society indicates that low-intensity blood stem cell transplants, which involve milder conditioning agents than those used in standard transplants, do not appear to harm the lungs and may potentially improve lung function in some patients with sickle cell disease (SCD).

Damage to lung tissue and impaired lung function are significant complications and major causes of death in individuals with sickle cell disease, a severe blood disorder. The new study examines whether less intensive types of transplants, which are often better tolerated by many adults, contribute to or exacerbate lung damage on their own.

Until recently, bone marrow and blood stem cell transplants were the only available cure for sickle cell disease. However, relatively few adults have received these treatments due to the health risks associated with the high doses of chemotherapy needed to prepare for the transplants.

Additionally, the process requires a genetically compatible donor, typically a sibling without sickle cell disease. The procedure involves transplanting blood stem cells from the donor to produce healthy red blood cells that replace the defective, "sickled" cells. These sickled cells obstruct blood flow throughout the body, leading to numerous issues such as severe pain episodes, infections, stroke, and acute chest syndrome, which deprives the lungs of oxygen.

For the study, scientists examined 97 patients with sickle cell disease who received a low-intensity, or non-myeloablative, blood stem cell transplant between 2004 and 2019. The researchers performed various pulmonary function tests. The results indicate that low-intensity blood stem cell transplants, which use less harsh conditioning agents compared to standard transplants, do not seem to damage the lungs and may even improve lung function in some patients with sickle cell disease (SCD).

Reference: Ruhl, A. P., Shalhoub, R., Jeffries, N., Limerick, E. M., Leonard, A., Barochia, A. V., Tisdale, J. F., Fitzhugh, C. D., & Hsieh, M. M. (2024). Pulmonary function after non-myeloablative hematopoietic cell transplant for sickle cell disease. Annals of the American Thoracic Society. https://doi.org/10.1513/AnnalsATS.202309-771OC