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

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


Lead Sponsor: Children's National Research Institute

Source Children's National Research Institute
Brief Summary

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.

Overall Status Withdrawn
Start Date February 2014
Completion Date June 2015
Primary Completion Date June 2015
Phase Phase 2
Study Type Interventional
Primary Outcome
Measure Time Frame
Sickle Cell Clinical Change from Baseline to Study Completion ( 18 months) Every 4 weeks for the 18 months study duration

Intervention Type: Drug

Intervention Name: Hydroxyurea

Arm Group Label: Hydroxyurea

Other Name: Hydrea

Intervention Type: Other

Intervention Name: Placebo

Arm Group Label: Sugar water

Other Name: Sugar water



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

Gender: All

Minimum Age: 6 Months

Maximum Age: 12 Months

Healthy Volunteers: No

Facility: Children's National Medical Center
Location Countries

United States

Verification Date

October 2017

Responsible Party

Type: Principal Investigator

Investigator Affiliation: Children's National Research Institute

Investigator Full Name: Emily Riehm Meier

Investigator Title: Attending Physician

Has Expanded Access No
Condition Browse
Number Of Arms 2
Arm Group

Label: Sugar water

Type: Placebo Comparator

Description: Placebo arm

Label: Hydroxyurea

Type: Active Comparator

Description: Treatment Arm

Study Design Info

Allocation: Randomized

Intervention Model: Parallel Assignment

Primary Purpose: Prevention

Masking: None (Open Label)

Source: ClinicalTrials.gov