Treatment of childhood sickle cell disease

Abstract

With the advances in our understanding of the complex mechanisms that come into play in sickle cell disease (SCD), medical care improves and patients with SCD live longer. It is, thus, essential to have adequate knowledge of the available and potential treatment modalities for all SCD complications to reduce morbidity and mortality. As in all chronic illnesses, patient education is the most important aspect of treatment. Patients should be enrolled in a routine follow-up program with multidisciplinary care for better outcomes. Penicillin prophylaxis and adequate immunizations must be instated as soon as the diagnosis is made. Regular screening is warranted to predict the risk of central nervous system involvement, pulmonary hypertension, nephropathy and retinopathy. In this chapter, we also discuss the management of acute SCD complications including vaso-occlusive painful crises, fever, cute chest syndrome, acute splenic sequestration, cerebrovascular accidents, priapism, aplastic crisis, hepatobiliary complications and ophthalmologic complications. We also present approaches for chronic complications such as pulmonary hypertension, chronic kidney disease, chronic pain, sickle retinopathy, leg ulcers and avascular necrosis. The indications and risks of blood transfusions are discussed in addition to hematopoietic stem cell transplant, the only curative treatment for SCD.