Background:
- In sickle cell disease (SCD), the proteins in the red blood cells that carry oxygen do
not behave normally. In parts of the body where there are low levels of oxygen or where
oxygen is used more, the sickle hemoglobin proteins may change shape and stick together.
This causes the red cells to clump, which reduces blood flow. This leads to even lower
oxygen levels and causes damage and/or pain.
- One way to stop the red blood cells from sticking together is to increase the levels of
fetal (baby or good ) hemoglobin. The good hemoglobin then takes the place of the sickle
hemoglobin.
- Hydroxyurea is the only approved drug for SCD. But hydroxyurea works in only about
two-thirds of people with SCD. Even in those cases it sometimes stops working over time.
- Researchers are interested in testing decitabine. The drug may help to increase fetal
hemoglobin levels. But it has not yet been approved to treat SCD.
Objectives:
- To test the safety and effectiveness of decitabine in increasing fetal hemoglobin levels
and improving the symptoms of sickle cell disease.
Eligibility:
- People at least 18 years of age who have sickle cell disease that has not improved after at
least 6 months of hydroxyurea therapy. Those who cannot take hydroxyurea because of side
effects may also participate.
Design:
- Participants will be screened with a physical exam and medical history. They will also
have blood and urine tests, a lung function test, and other tests as required.
- Participants will receive decitabine injections up to twice a week for 1 year. Depending
on the response to treatments, the dose will remain the same or be reduced to once a
week.
- Participants will be monitored with frequent blood tests and other studies as directed
by the study doctors.
- After the study is completed, participants will go back to their usual sickle cell care.
If decitabine has improved a participant's SCD, treatment may be continued under regular
health coverage insurance if this can be arranged.