From the Guidelines
The best management for this 9-year-old girl with sickle cell disease presenting with fatigue, lethargy, pallor, hepatosplenomegaly, and severe anemia (Hb 5 g/dL) is option C, Hydroxyurea. This patient is experiencing symptoms that can be managed with hydroxyurea, which increases total and fetal hemoglobin and decreases vaso-occlusive complications, including episodes of painful events and acute chest syndrome, hospitalizations, and need for transfusion 1. The use of hydroxyurea has a positive impact on health-related quality of life in people with HbSS or Sb0-thalassemia, and it is recommended to be offered to every child with HbSS or Sb0-thalassemia at 9 months of age, even without clinical symptoms 1. While splenectomy may be considered in some cases, the provided evidence does not strongly support it as the primary treatment for this patient's current condition. Some key points to consider in the management of this patient include:
- Hydroxyurea is taken once a day orally and is supplied as a capsule, fast-dissolving tablet, or compounded liquid 1.
- Myelosuppression is a potential adverse effect, and a CBC and reticulocyte count should be monitored every 1 to 3 months depending on how long the child has been taking the medication and whether the child has attained a stable dose 1.
- Regular blood transfusions may be used temporarily but do not address the underlying problem, and they carry the risk of iron overload, alloimmunization, and other complications 1.
- Corticosteroids have no role in managing splenic sequestration, and their use is not supported by the provided evidence. It is essential to prioritize the patient's morbidity, mortality, and quality of life when making management decisions, and hydroxyurea is the most appropriate option based on the available evidence.
From the Research
Management of Sickle Cell Disease
The patient's symptoms, including fatigue, lethargy, pallor, hepatomegaly, and splenomegaly, along with a low hemoglobin level and positive sickle cell test, suggest a diagnosis of sickle cell disease. The best management for this patient would be:
- Hydroxyurea therapy, as it has been shown to decrease the frequency of pain episodes and other acute complications in adults and children with sickle cell anemia 2, 3, 4, 5
- The use of hydroxyurea has been associated with improved hematological parameters, including increased fetal hemoglobin levels and decreased neutrophil counts 2, 4
- Hydroxyurea therapy has also been shown to reduce the rate of transfusions, hospitalizations, and acute chest syndrome events 2, 3, 4
Comparison of Treatment Options
The following treatment options were considered:
- Regular blood transfusions: while this can help reduce the incidence of strokes and acute chest syndrome, it is associated with the burden of monthly visits and excessive iron stores 6
- Corticosteroids: there is no evidence to suggest that corticosteroids are effective in managing sickle cell disease
- Splenectomy: this is not a recommended treatment for sickle cell disease, as it can increase the risk of infections and other complications
Recommended Course of Action
Based on the available evidence, hydroxyurea therapy is the recommended course of action for this patient. It is essential to monitor the patient's response to therapy and adjust the dose as needed to minimize adverse effects. Regular follow-up appointments and laboratory tests are necessary to assess the patient's hematological parameters and overall health status 2, 3, 4, 5