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Content Update

November 26, 2019

Voxelotor Approved for Treatment of Sickle Cell Disease: Voxelotor (Oxbryta®) is the first hemoglobin S polymerization inhibitor approved by the FDA and is indicated for adults and children older than 12 for the treatment of sickle cell disease. Sickle cell disease affects approximately 100,000 individuals in the US and is a hereditary condition which results in the production of sickle, or abnormally, shaped hemoglobin and has limited treatment options. In a phase 3 randomized, placebo controlled, double blind trial, two doses of voxelotor were compared to placebo. Those treated with the 1500mg dose had a significantly higher hemoglobin response compared to placebo, 51% (95% CI, 41 - 61) versus 7% (95% CI, 1 - 12). Adverse effects were similar across the study groups and the most common being headache and diarrhea.

LEARNING OBJECTIVES

LEARNING OBJECTIVES

Upon completion of the chapter, the reader will be able to:

  1. Explain the underlying causes of sickle cell disease (SCD) and their relationship to patient signs and symptoms.

  2. Identify the typical characteristics of SCD as well as symptoms that indicate complicated disease.

  3. Identify the desired therapeutic outcomes for patients with SCD.

  4. Recommend appropriate pharmacotherapy and nonpharmacotherapy interventions for patients with SCD.

  5. Recognize when chronic maintenance therapy is indicated for a patient with SCD.

  6. Describe the components of a monitoring plan to assess effectiveness and adverse effects of pharmacotherapy for SCD.

  7. Educate patients about the disease state, appropriate therapy, and drug therapy required for effective treatment and prevention of complications.

INTRODUCTION

image “Sickle cell syndrome” refers to a collection of autosomal recessive genetic disorders that are characterized by the presence of at least one sickle hemoglobin (HbS) gene.1,2

Sickle cell disease (SCD) is a chronic illness that is associated with frequent crisis episodes. Acute complications are unpredictable and potentially fatal. Common symptoms include excruciating musculoskeletal pain, life-threatening pneumonia-like illness, cerebrovascular accidents, and splenic and renal dysfunction.2 As the disease progresses, patients may develop organ damage from the combination of hemolysis and infarction. Because of the complexity and severity of SCD, it is imperative that patients have access to comprehensive care with providers who have a good understanding of the countless clinical presentations and the management options of this disorder.

EPIDEMIOLOGY AND ETIOLOGY

Sickle cell trait (SCT) is the heterozygous form (HbAS) of SCD in which a person inherits one normal adult hemoglobin (HbA) gene and one sickle hemoglobin (HbS) gene. These individuals are carriers of the SCT and are usually asymptomatic.2 About 1 in 13 African American babies is born with SCT.3 Symptomatic disease is seen in homozygous and compound heterozygous genotypes of SCD. Sickle ...

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