Hematopoietic Cell Transplantation Offers Long-Term relief for Sickle Cell Disease Patients

Patients who underwent hematopoietic cell transplantation for sickle cell disease saw high rates of survival without disease symptoms and low rates of severe side effects or complications years after their procedure, according to a new study. the study included over 1,000 patients, representing the largest and most complete analysis of long-term transplant outcomes to date in people living with sickle cell disease.

A majority of patients in this cohort are alive; the transplant worked so they no longer show symptoms of their sickle cell disease, and most have had no late effects post-transplant. There are families that realy want to know the data, and this will be among the largest and most statistically well-powered studies that can provide this details to patients we are counseling about transplant.

Elizabeth Stenger, MD, lead study author, associate professor in the department of pediatrics at Emory University School of Medicine and pediatric hematologist/oncologist at the Aflac Cancer and Blood Disorders Center of Children’s Healthcare of Atlanta, Georgia

hematopoietic cell transplantation can eliminate symptoms of sickle cell disease by giving patients the ability to make healthy blood cells instead of ones prone to sickling. For the procedure,patients first undergo a chemotherapy-based conditioning regimen to clear the bone marrow of their own stem cells. Stem cells from a healthy donor are than infused and migrate to the bone marrow, where they begin producing healthy blood cells.

This procedure has been in use for several decades, but strategies for donor matching and conditioning have evolved over time. Long-term outcomes from hematopoietic cell transplantation,as it is currently practiced,have not been well studied in people living with sickle cell disease.