HEMATOPOIETIC STEM CELL TRANSPLANTATION IN SICKLE CELL DISEASE

Course overview

SINCE THE SICKLE CELL ANEMIA ACT, STEADY PROGRESS HAS BEEN MADE IN THE SCREENING AND TREATMENT OF SICKLE CELL DISEASE. DESPITE THE DEVELOPMENT OF NEW MEDICAL THERAPIES, SICKLE CELL DISEASE REMAINS AN INCURABLE CONDITION FOR MOST AFFECTED INDIVIDUALS. HEMATOPOIETIC STEM CELL TRANSPLANTATION REPRESENTS THE ONLY CURRENTLY AVAILABLE CURATIVE OPTION FOR INDIVIDUALS LIVING WITH SICKLE CELL DISEASE. HEMATOPOIETIC STEM CELL TRANSPLANTATION MAY BE AUTOLOGOUS, REQUIRING GENETIC MODIFICATION OF THE PATIENT'S STEM CELLS TO CORRECT THE GENETIC MUTATION CHARACTERISTIC OF SICKLE CELL DISEASE, OR ALLOGENEIC, INVOLVING REPLACEMENT OF THE DEFECTIVE STEM CELLS WITH HEALTHY STEM CELLS FROM A SUITABLE DONOR. ALTHOUGH HSCT IS CURATIVE FOR MOST PATIENTS, THE PROCEDURE IS ASSOCIATED WITH SIGNIFICANT TOXICITIES AND OCCASIONALLY FATAL COMPLICATIONS. CARE MUST BE EXERCISED WHEN SELECTING PATIENTS FOR HSCT. THIS ACTIVITY REVIEWS THE THERAPEUTIC ROLE OF HSCT IN SICKLE CELL DISEASE AND HIGHLIGHTS THE ROLE OF THE INTERPROFESSIONAL TEAM IN CARING FOR PATIENTS WHO UNDERGO THE PROCEDURE.