Although hydroxyurea is an evidence-based therapy in sickle cell disease, there are barriers to its use in children and adolescents.
Benefits of hydroxyurea in patients with homozygous sickle cell anemia:
(1) reduction in severe pain episodes
(2) reduction of the acute chest syndrome
(3) reduction of other complications
(4) increased survival
Barriers to hydroxyurea therapy:
(1) patient/parent or provider uncertainty about the effectiveness of treatment
(2) patient/parent or provider fear of adverse effects (including perceived carcinogenic potential)
(3) complexity of the treatment regimen
(4) provider discomfort with managing the therapy
(5) real or perceived risk of noncompliance
(6) lack of reimbursement and/or high cost
