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Long-term hydroxyurea treatment in children with sickle cell disease: tolerance and clinical outcomes
Mariane de Montalembert1, Valentine Brousse, Caroline Elie
1Service de Pédiatrie Générale, Hôpital Necker, Paris, France. mariane.demontal@nck.ap-hop-paris.fr
Insights
Hydroxyurea treatment is generally well-tolerated in children with sickle cell disease (SCD). Few severe side effects were observed, with most discontinuations due to treatment failure or non-compliance.
Area of Science:
- Pediatrics
- Hematology
- Pharmacology
Background:
- Sickle cell disease (SCD) is a genetic blood disorder requiring long-term management.
- Hydroxyurea is a medication used to reduce complications in SCD patients.
- Assessing the long-term tolerability and safety of hydroxyurea in pediatric SCD populations is crucial.
Purpose of the Study:
- To evaluate the tolerability and safety of hydroxyurea treatment in a cohort of children with sickle cell disease.
- To identify reasons for hydroxyurea discontinuation in this pediatric population.
Main Methods:
- A prospective study enrolled 225 children with SCD.
- Patients received hydroxyurea treatment with a median duration of 3.8 years.
- Data on treatment duration, side effects, and reasons for discontinuation were collected.
Main Results:
- No severe side effects were attributed to hydroxyurea treatment.
- Hydroxyurea was discontinued in 81 children.
- Primary reasons for discontinuation included treatment failure (30 cases) and non-compliance (17 cases).
- Other reasons for withdrawal included hypersplenism, abnormal transcranial Doppler findings, and stroke.
Conclusions:
- Hydroxyurea demonstrates a favorable safety profile in children with sickle cell disease.
- Treatment discontinuation is primarily linked to efficacy and adherence issues rather than severe adverse events.
- Long-term hydroxyurea therapy is a viable option for managing pediatric SCD, with careful monitoring for specific complications.
Abstract:
Two hundred twenty-five SCD children have been enrolled in a study assessing the tolerability of hydroxyurea treatment. Mean age at inclusion was 9.2+/-4.4 years, median duration of treatment was 3.8 years. Ten and 75 patients have been treated respectively for more than 10 and 5 years. No severe side effect was related to hydroxyurea treatment, which was discontinued in 81 children mainly for treatment failure (30 cases) or non-compliance (17 cases). Treatment was also withdrawn in 5 of 6 children who had developed hypersplenism, in 3 because of a pathological transcranial Doppler, and in 2 after a stroke.
