Treatment of Adult Patients With Hemoglobin SC Disease (SCYTHE)
Condition: Hemoglobin SC Disease
MeSH terms: Hemoglobin SC Disease
Intervention: Hydroxyurea
Intervention description: Treat symptomatic HbSC patients to MTD on hydroxyurea, and assess for clinical improvement using the AdultsQLTM 3.0 Sickle Cell Disease Module after 6 months at MTD, compared to entrance scores
Intervention MeSH terms: Hydroxyurea
Phase: Phase 2
Study type: Interventional
Primary purpose: Treatment
Intervention model: Single Group Assignment
Masking: None (Open Label)
Enrollment: 1.0
Sex: All
Minimum age: 18 Years
Maximum age: 69 Years
Healthy volunteers: No

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
      patients with the most common type of sickle cell, those who are homozygous for the sickle
      mutation (HbSS), helps individuals 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 AdultsQLTM 3.0 Sickle Cell Disease Module, measures
      quality of life. The investigators will also see how hydroxyurea changes laboratory test
      numbers, and blood thickness.
    

Eligibility criteria:

        Inclusion Criteria:

          -  Diagnosis of HbSC disease

          -  Score of 80 or lower on the AdultQLTM 3.0 Sickle Cell Disease Module, or any disease
             related complication, including, but not limited to, one or more pain events per year,
             proliferative sickle retinopathy, avascular necrosis, cholelithiasis, or any
             thrombotic event. If the subject has a score >80, they may still enroll on the trial,
             and be analyzed for secondary endpoints. They will be excluded from analysis of the
             primary endpoint.

        Exclusion Criteria:

          -  Failure to meet inclusion criteria

          -  Hydroxyurea usage in the last 3 months.

          -  Chronic RBC transfusion therapy

          -  Packed red blood cell transfusion in the last 3 months (temporary exclusion).

          -  Pregnancy, or refusal to use medically effective birth control if female and sexually
             active.

          -  Current phlebotomy therapy