Mitchell Cairo, MD, Chief of Pediatric Hematology, Oncology and Stem Cell Transplantation; Director of the Children and Adolescent Cancer and Blood Diseases Center; Associate Chairman of the Department of Pediatrics; and Professor of Pediatrics, Medicine, Pathology, Microbiology and Immunology at New York Medical College discusses his study at the 2018 American Society of Hematology (ASH) Annual Meeting in San Diego.
In this study, the investigators performed transplants in 19 patients, age 3 to 20 years, with frequent or severe SCD symptoms, including 15 patients who received a transplant from their mothers and four who received a transplant from their fathers. Two years after the transplant, the researchers found that the patients experienced improved cardiovascular and neurological function as well as demonstrated improvements in health-related quality of life and were considered stable. The findings suggest that 90% of patients will go on to lead normal lives. This approach has the potentially to change the treatment of sickle cell disease and provide patients with the disease a viable and highly effective stem cell transplantation treatment option.
At this year’s ASH conference, Dr. Cairo presented 3-year follow-up of this study, which showed there are no patients who have any signs of sickle cell disease and sickle symptoms. His study showed that myeloablative and haploidentical hematopoietic stem cell transplantation from parental donors that utilized CD34 enrichment and mononuclear cell addback significantly improved health-related quality of life and neurocognition of high-risk patients with sickle cell disease.
The study concluded that rapid hematological reconstitution, long-term stable white blood cell and red blood cell donor chimerism, stable to improved cardiac and pulmonary function, and low cumulative incidence of acute and chronic graft-versus-host disease.