A new three-year study shows that “so-called low-intensity blood stem cell transplants, which use milder conditioning agents than standard stem cell transplants, do not appear to damage the lungs and may help improve lung function in some patients with sickle cell disease.” NIH has the release.
One of the leading causes of death in people with sickle cell disease is damage to lung tissue and worsened lung function. Using a low-intensity blood stem cell transplant, which “tend to be better tolerated by adults,” may help more people get treatment.
The only cure for sickle cell disease has historically been bone marrow and blood stem cell transplants, but “relatively few adults have undergone the treatments due to health risks associated with high doses of chemotherapy required to prepare for transplants. In addition, the process requires a genetically well-matched donor, usually a sibling who does not have SCD.”
At least one third of the sickle cell stem cell transplants are low-intensity, which are “slightly less effective than the standard transplants.” However, “adults who often have more pre-existing organ damage than children tend to do better with them and also experience a lower risk for complications such as graft-versus-host disease.”
Researchers conducted “a variety of pulmonary function tests” in 97 patients who underwent a low-intensity blood stem cell transplant. After three years, “overall lung function among the patients remained stable,” and measures like diffusing capacity of the lungs for carbon monoxide and six-minute walk distance “improved significantly following transplant.”
