Risk-based Therapy for Sickle Cell Anemia: A Feasibility Study

Study Overview
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Brief Summary
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Sickle cell anemia (SCA) patients experience organ damage that begins at an early age and results in significant morbidity and early mortality. Although all SCA patients share the same genetic mutation, the clinical complications are highly variable with some patients experiencing frequent and severe complications, while others have few serious complications. If SCA severity could be predicted early in life, those patients at greatest risk for complications could receive treatment prior to the onset of organ damage. No general SCA severity predictor or one that can be informative early in life exists. The investigators preliminary research has identified the absolute reticulocyte count (ARC) as a potential early predictive risk marker for SCA complications in pediatric patients. A higher ARC between ages 2 and 6 months of age is associated with an increased risk of hospitalization in the first 3 years of life; the mean ARC for the 36 patients who were hospitalized for SCA complications was significantly higher than that of the remaining 23 in those who were not hospitalized. Moreover, total hospitalizations were nearly three times higher by age 2 years in those infants who had an ARC of > 200 than for those infants whose ARC was <200. The proposed study will determine if ARC can be used as a risk-stratifier in asymptomatic infants with SCA and ascertain its value in targeting hydroxyurea therapy to those infants at highest risk of SCA sequelae.

Official Title
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Reticulocyte as Risk Marker: Targeted Therapy for Infants With Sickle Cell Anemia

Conditions
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Sickle Cell Anemia

Intervention / Treatment
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* Drug: Hydroxyurea
* Other: Placebo


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: ages 6-12 months Sickle cell anemia (HbSS) steady state absolute reticulocyte count between 2-6 months is available in the medical record Exclusion Criteria: receiving hydroxyurea or chronic monthly blood transfusions patient enrolled in preliminary study

Ages Eligible for Study
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Minimum Age: 6 Months
Maximum Age: 12 Months

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Prevention
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Placebo Comparator: Sugar water<br>Placebo arm | Other: Placebo<br> <br> * Other names: Sugar water;|
| Active Comparator: Hydroxyurea<br>Treatment Arm | Drug: Hydroxyurea<br> <br> * Other names: Hydrea;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Sickle Cell Clinical Change from Baseline to Study Completion ( 18 months) | Participants will be have a study visit at baseline and then every month for the 18 month duration of the study. Study visits will include a physical exam, laboratory monitoring and interval history to assess the change (if any) every 4 weeks of sickle cell complications. | Every 4 weeks for the 18 months study duration |