Treatment of Hemoglobin SC Disease With Hydroxyurea
Status:
Terminated
Trial end date:
2017-03-31
Target enrollment:
Participant gender:
Summary
Sickle cell disease (SCD), specifically hemoglobin SC disease (HbSC), is a subtype of sickle
cell disease with typically higher hemoglobin and milder or later disease complications.
Sickle cell disease is a disorder in which red blood cells (RBCs) are abnormally shaped. This
can result in painful episodes, serious infections, and damage to body organs. One medication
used to treat sickle cell disease is hydroxyurea.
Hydroxyurea therapy offers significant benefits for infants, children, and adolescents with
sickle cell anemia. These include a reduction in the frequency of pain crises and acute chest
syndrome (inflammation of the lungs). Hydroxyurea has been given to many HbSC patients but
HbSC patients were not included in the large clinical trials used to test hydroxyurea in SCD,
so less is known about how HbSC patients respond to hydroxyurea.
The purpose of this research study is to see if hydroxyurea, a medication given to many
children with the most common type of sickle cell, those who are homozygous for the sickle
mutation (HbSS), helps children who have HbSC. The investigators will see if it helps by
giving a questionaire when the medication is started, and then every two months at a clinic
visit. The questionaire, called the Pediatric Quality of Life Inventory (PedsQL™) Sickle Cell
Disease Module version 3.0, measures quality of life. The investigators will also see how
hydroxyurea changes laboratory test numbers, and blood thickness.