Chapter 102: Sickle Cell Disease

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.

Patient Care Process for the Management of Sickle Cell Disease

Collect

• Patient characteristic including age, race, sickle cell disease type (see Table 102-1)

• Patient medical history including frequency of pain episodes and hospitalizations due to acute or chronic complication of sickle cell disease (Table 102-2)

• Clinical signs and symptoms (see Table 102-1)

• Current medications

• Immunization history (see Table 102-4)

• Objective data (see Table 102-2)

  • BP, heart rate (HR), height, weight, and BMI

  • Labs (e.g., serum electrolytes, Scr, BUN, CBC, reticulocyte count, urinalysis)

  • Other diagnostic tests when indicated (e.g., cultures, parvovirus titers, chest x-ray)

Assess

• Risk of infection or sepsis in febrile patients

• Presence of acute complications such as vasoocclusive pain episodes, aplastic crisis, splenic sequestration, acute chest syndrome (see Table 102-2)

• Presence of chronic complications associated with sickle cell disease

• Health maintenance and preventive care (see Table 102-4)

• Pain control for both acute episodes and chronic pain management

Plan^*

• Goals of treatment

• Hydroxyurea therapy regimen including dose, route, frequency and monitoring (see Tables 102-5, Figure 102-6)

• Preventive care such as immunizations, penicillin prophylaxis, and stroke prevention

• Pain management for acute and chronic pain

• Patient education (e.g. purpose of treatment, side effects)

• Referrals to other providers when appropriate (e.g., physician, dietician, psychological support programs)

Implement^*

• Provide patient education regarding all elements of treatment plan

• Use motivational interviewing and coaching strategies to maximize adherence

• Schedule follow-up

Follow-up: Monitor and Evaluate

• Presence of adverse effects

• Occurrence of acute and chronic complications associated with sickle cell disease

• Evaluate growth and development as well quality of life

• Patient adherence to treatment plan using multiple sources of information

KEY CONCEPTS

• Sickle cell disease is an inherited disorder caused by a defect in the gene for β-globin, a component of hemoglobin, and ...