Sickle Cell Disease: Prevention of Complications

Abstract

In spite of more than 100 years of clinical observations and research, many unanswered questions remain in the management of sickle cell disease (SCD). While giant strides in the prevention of pediatric morbidity and mortality have been made in the latter part of the twentieth century, adults with SCD are faced with a chronic, debilitating disease with limited preventive and therapeutic options. Stem cell transplantation, the only cure presently available, is limited by toxicity and restricted eligibility, and no new disease-modifying drug has been introduced since the approval of hydroxyurea in the mid-1990s. The paucity of management options underscores the slow pace of research progress, hindered by factors such as complexity of the phenotype, scarcity of advocacy and funding, and competing research and health-care priorities. The recently published NHLBI guidelines for the management of SCD (Yawn et al. 2014), which we strongly encourage the reader to review, provide a comprehensive compendium of the available evidence and evidence-based recommendations, but necessarily rely heavily on expert panel consensus opinion in many areas. Thus, important preventive and therapeutic interventions remain controversial. By presenting the following cases, we have attempted to review the most important evidence-based guidelines and existing controversies in the prevention of SCD complications. Please note that we will follow the standard nomenclature of sickle cell anemia (SCA) to denote HbSS and HbS/β⁰ thalassemia and SCD to denote any disease subtype (HbSS, HbS/β⁰ or β⁺ thalassemia, HbSC, HbSD, and HbSO). We will use the term vaso-occlusive crisis (VOC) to denote a painful episode/pain crisis.