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Hydroxyurea use in young infants with sickle cell disease
Sarah B Schuchard1,2, Jennifer R Lissick2, Amanda Nickel2
1SSM Health Cardinal Glennon Children's Hospital, St. Louis, Missouri.
Insights
Hydroxyurea (HU) is safe and effective for infants with sickle cell disease, showing a stronger response when started between 5-12 months. Early HU treatment in sickle cell disease patients leads to fewer hospitalizations and transfusions.
Area of Science:
- Hematology
- Pediatric Medicine
- Pharmacology
Background:
- Hydroxyurea (HU) is a proven treatment for sickle cell disease (SCD), reducing complications and improving patient survival.
- Existing evidence suggests initiating HU therapy after nine months of age.
Purpose of the Study:
- To evaluate the safety and efficacy of initiating hydroxyurea (HU) therapy in pediatric patients with sickle cell disease (SCD) at different early age groups.
- To compare clinical outcomes, laboratory data, and toxicity profiles among infants and young children starting HU treatment before five years of age.
Main Methods:
- A retrospective study analyzed 65 pediatric patients with SCD who began HU treatment between 2008 and 2016.
- Patients were divided into three cohorts based on age at HU initiation: 0-1 year (cohort 1), 1-2 years (cohort 2), and 2-5 years (cohort 3).
- Clinical events, laboratory values, and treatment toxicity were assessed and compared across the three age cohorts.
Main Results:
- Cohort 1 (0-1 year) exhibited higher hemoglobin and MCV, and lower absolute reticulocyte count and absolute neutrophil count (ANC) at 24 months compared to cohort 3 (2-5 years).
- Baseline and sustained Hemoglobin F (HbF) levels were significantly higher in cohort 1 compared to older cohorts.
- Cohort 1 experienced fewer hospitalizations and transfusions, with no significant difference in toxicity observed across groups.
Conclusions:
- Hydroxyurea (HU) is safe and effective when initiated in infants aged 5 to 12 months with sickle cell disease (SCD).
- Starting HU therapy earlier, specifically in the 5-12 month age range, generates a more robust therapeutic response compared to initiation in older children.
- Early intervention with HU in SCD patients demonstrates improved clinical outcomes and sustained efficacy.
Background:
Hydroxyurea (HU) reduces complications and improves quality and duration of life in sickle cell disease. Evidence supports the use of HU starting after nine months of age.
Procedures:
We performed a retrospective study of patients starting HU at less than five years of age between January 1, 2008, and December 31, 2016. We evaluated clinical events, laboratory data, and toxicity between three different age groups: cohort 1 (0-1 year), cohort 2 (1-2 years), and cohort 3 (2-5 years).
Results:
Sixty-five patients were included in the analysis. The mean age was 7.2 months (n = 35), 19.5 months (n = 13), and 35.5 months (n = 17) for cohorts 1, 2, and 3, respectively. Cohort 1 had higher hemoglobin (P = 0.0003) and MCV (P = 0.0199) and lower absolute reticulocyte count (P = 0.0304) at 24 months of age compared with cohort 3. The absolute neutrophil count (ANC) was lower compared with both older cohorts (P = 0.0364, 0.0025). The mean baseline hemoglobin F in cohort 1 was 31.5% compared with 19.7% and 16.5% in cohorts 2 and 3, respectively (P = 0.002, P < 0.0001). The mean duration of therapy was 31.3 months, 57.6 months (P = 0.018), and 29.1 months (P = 0.401), respectively. Mean Hb F levels remained higher in cohort 1 (29.9%) compared with cohorts 2 and 3 (20.4%, P = 0.007; 20.6%, P = 0.003). Cohort 1 experienced fewer hospitalizations (P = 0.0025), pain crises (P = 0.0618), and transfusions (P = 0.0426). There was no difference in toxicity between groups.
Conclusion:
HU is safe and effective in patients 5 to 12 months of age and generated a more robust response compared with initiation in older patients.
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