A 5-Year-Old With a Headache, Limp, and Projectile Vomiting

Afif Harb, MD; Antoine Saliba, MD

Disclosures

June 10, 2020

All children with homozygous sickle cell anemia or sickle beta zero thalassemia who are younger than 16 years should undergo annual screening with TCD ultrasonography. Values greater than 170 cm/sec are worrisome, and patients with a value higher than 200 cm/sec should be started on indefinite transfusion therapy.

Iron overload can be measured using several techniques. Biopsy is the criterion standard for measurement of liver iron concentration, but this technique is invasive. Serial measurement of serum ferritin levels is practical but has questionable accuracy. MRI is a noninvasive and reliable technique for measurement of liver iron concentration. On the basis of the degree of iron overload, these patients should be treated with iron chelation therapy.

Three chelators are available: deferoxamine (parenteral), deferiprone (oral), and deferasirox (oral).[9] Most knowledge on the efficacy and safety of these chelators in reducing iron overload comes from studies in patients with thalassemia who had iron overload and in patients with sickle cell disease. Currently, most evidence supports the use of deferasirox in treating iron overload because of its safe profile, easy means of administration, good rate of patient adherence, and efficacy in decreasing excess iron in the body.[10]

After diagnosis, the patient in this case underwent acute exchange transfusion and was later moved to lifelong transfusion therapy to prevent stroke recurrence. She was also given deferasirox (30 mg/kg/d) orally once daily to prevent transfusion iron overload and its complications.

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