BONE MARROW TRANSPLANTATION IN SICKLE CELL DISEASE

Course overview

SINCE THE SICKLE CELL ANEMIA ACT, THERE HAS BEEN PROGRESS IN THE SCREENING AND TREATMENT OF SICKLE CELL DISEASE. FOR INSTANCE, IN MOST PARTS OF THE UNITED STATES, SICKLE CELL SCREENING IS DONE BEFORE BABIES ARE DISCHARGED FROM THE HOSPITAL. AS A RESULT, PARENTS KNOW THE STATUS OF THEIR BABY, LEADING TO EARLY MEDICAL INTERVENTION AT A YOUNG AGE, AND REDUCING MORBIDITY AND MORTALITY. TREATMENT OF SICKLE CELL DISEASE HAS ALSO IMPROVED, INCLUDING THE USE OF PENICILLIN PROPHYLAXIS FOR PATIENTS LESS THAN 5 YEARS OLD, THE USE OF HYDROXYUREA FOR INCREASING THE NUMBER OF FETAL HEMOGLOBIN, BLOOD TRANSFUSIONS, AND PAIN MEDICATIONS INCLUDING OPIOIDS. MOST OF THESE TREATMENTS ARE PALLIATIVE AND UNFORTUNATELY, PATIENTS STILL HAVE A POOR QUALITY OF LIFE BECAUSE OF EXTREME PAIN EPISODES, END-ORGAN DAMAGE, AND ALSO A REDUCED LIFE EXPECTANCY. IN SEPTEMBER 2018, THE NATIONAL HEART, LUNG AND BLOOD INSTITUTE (NHLBI) LAUNCHED THE CURE SICKLE CELL INITIATIVE. ADVANCEMENT IN NEW GENE THERAPY HAS SHOWN A PROMISING RESULT IN BOTH PRECLINICAL AND CLINICAL TRIALS. THIS NEW TECHNOLOGY WILL ENTAIL REMOVING THE PATIENT’S STEM CELLS FROM THE BONE MARROW AND THEN ADDING A THERAPEUTIC GENE TO THOSE CELLS WHICH WILL THEN LEAD TO THE PRODUCTION OF ANTI-SICKLING CELLS. THIS ACTIVITY REVIEWS THE ROLE OF BMT IN SICKLE CELL DISEASE AND HIGHLIGHTS THE ROLE OF THE INTERPROFESSIONAL TEAM IN THE MANAGEMENT OF THESE PATIENTS.