From the Guidelines
Sickle cell disease in pediatric patients requires comprehensive management focusing on prevention of complications and prompt treatment of acute events, with hydroxyurea therapy being a crucial component, as evidenced by the 2021 guideline from the American Heart Association/American Stroke Association 1.
Overview of Sickle Cell Disease Management
Children with sickle cell disease should receive prophylactic penicillin (penicillin V 125mg twice daily for children under 3 years, 250mg twice daily for older children) starting at diagnosis to prevent pneumococcal infections. Vaccination schedules should be strictly followed, including additional vaccines like pneumococcal conjugate and polysaccharide vaccines. Daily folic acid supplementation (1mg) is recommended to support red blood cell production.
Pain Management and Hydroxyurea Therapy
For pain management during vaso-occlusive crises, a stepwise approach using acetaminophen, NSAIDs, and opioids as needed is appropriate, with adequate hydration (1.5 times maintenance fluids). Hydroxyurea therapy should be considered for children with frequent pain crises or history of acute chest syndrome, typically starting at 15-20 mg/kg/day and titrating based on response and monitoring blood counts, as supported by the National Heart, Lung, and Blood Institute-funded Pediatric Hydroxyurea Phase III Clinical Trial (BABY HUG) 1.
Stroke Risk Assessment and Management
Regular transcranial Doppler ultrasound screening should begin at age 2 to assess stroke risk, with chronic transfusion therapy initiated if velocities are elevated, as recommended by the 2008 scientific statement from the American Heart Association Stroke Council and the Council on Cardiovascular Disease in the Young 1. Parents should be educated about fever management (immediate medical attention for temperatures ≥101.5°F), signs of splenic sequestration (abdominal pain, pallor), and triggers to avoid (dehydration, extreme temperatures).
Key Considerations
- Sickle cell disease causes abnormal hemoglobin that distorts red blood cells into a sickle shape under stress, leading to vascular occlusion, hemolysis, and progressive organ damage.
- Early intervention and consistent preventive care are essential for improving outcomes in affected children.
- The 2021 guideline from the American Heart Association/American Stroke Association 1 provides recommendations for the prevention of stroke in patients with sickle cell disease, including the use of hydroxyurea therapy and chronic transfusion therapy.
From the Research
Sickle Cell Disease in Pediatrics
- Sickle cell disease (SCD) is a heterogeneous inherited disorder of hemoglobin that causes chronic hemolytic anemia, vaso-occlusion, and endothelial dysfunction, often leading to multiorgan damage in infancy and throughout childhood 2.
- The most common types of SCD are homozygous hemoglobin S (HbSS disease), hemoglobin SC disease, and sickle β thalassemia, with HbSS disease and sickle β(0) thalassemia often referred to as sickle cell anemia due to similar severity 2.
Management and Treatment
- Screening and preventive measures, including infection prophylaxis and vaccination, have significantly improved outcomes for children with SCD 2.
- Evidence-based therapies, such as hydroxyurea and transfusion, play an important role in preventing progression of select complications 2.
- Hydroxyurea has been shown to decrease the frequency of pain episodes and other acute complications in adults and children with sickle cell anemia of HbSS or HbSβºthal genotypes, and in preventing life-threatening neurological events in those with sickle cell anemia at risk of primary stroke 3, 4.
Stroke Prevention
- Transcranial Doppler (TCD) screening and preventive chronic transfusion have been shown to lower the occurrence of stroke in pediatric patients with SCD 5.
- Hydroxyurea prescription and specific antibiotic and vaccination regimes have also been shown to reduce the occurrence of stroke in pediatric patients with SCD 5.
- However, there is still a need for an epidemiological survey to define ideal screening and preventative care for adults with SCD, as well as to identify silent cerebral stroke and prevent its complications 5.
Current Era of Sickle Cell Disease
- Hydroxyurea remains the gold standard for treatment of SCD, despite the development of new therapeutics, due to its long-standing record of efficacy and safety 6.
- Hydroxyurea is not only a disease modifier but also has additional clinical benefits, is affordable, and has prompted expanded research in areas such as underutilization and pharmacogenomics 6.