Hydroxyurea and Transfusion: Pilot Study of Combination Therapy for Patients With Sickle Cell Anemia

Locations

1 United States site

Age

2 to 24 Years

Genotypes

SS

Phase

Phase 2

Study type

Interventional

Gender

All

Interventions

Drug

Compensation

Unknown

About the study

This study will prospectively investigate the feasibility, safety, and transfusion
requirements of adding hydroxyurea to simple chronic transfusions for patients with sickle
cell anemia already on chronic transfusions.

participation requirements

1. Diagnosis of SCA (Hb SS or Sβ0 thalassemia).

2. On simple chronic transfusion therapy for stroke prevention (primary or secondary
prevention) for ≥1 year with no plans to stop simple chronic transfusion in the next
year.

participation restrictions

1. Poor adherence to simple transfusion regimen as defined by having an HbS >45% at any
time in the last year AND a transfusion interval >5 weeks.

2. Treatment with hydroxyurea in the 12 months prior to study enrollment.

3. Abnormal initial laboratory values (temporary exclusions):

1. Absolute neutrophil count <1.5 x 10^9/L 2. Platelet count <100 x 10^9/L 3. Serum creatinine more than twice upper limit for age 4. Pregnancy or unwillingness to use a medically acceptable form of contraception if sexually active.

Locations

  • Washington, District of Columbia, United States, Children's National Health System, 20010