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Efficacy of fixed low dose hydroxyurea in Indian children with sickle cell anemia: a single centre experience

Dipti L Jain1, Mohini Apte, Roshan Colah

  • 1Department of Pediatrics, Government Medical College, Nagpur; and * National Institute of Immunohaematology, Mumbai, India. Correspondence to: Dr Dipti Jain,Professor, Department of Pediatrics, Governement Medical College, Nagpur, Maharashtra 440 003, India. drvijayasarathi@gmail.com.

Indian Pediatrics
|June 27, 2013
PubMed

Insights

Fixed low-dose hydroxyurea (HU) effectively improved fetal hemoglobin and reduced clinical events in Indian children with sickle cell anaemia (SCA). The treatment was found to be safe over a two-year follow-up period.

Area of Science:

  • Pediatric Hematology
  • Sickle Cell Disease Research
  • Pharmacological Interventions

Background:

  • Limited data exists on hydroxyurea (HU) efficacy in Indian children with sickle cell anaemia (SCA).
  • Evaluation of fixed low-dose HU is crucial for this specific pediatric population.

Purpose of the Study:

  • To assess the efficacy and safety of a fixed low dose of hydroxyurea (HU).
  • To evaluate hematological and clinical outcomes in Indian children with severe sickle cell anaemia (SCA).

Main Methods:

  • A cohort of 144 children (<18 years) with severe SCA received fixed low-dose HU (10 mg/kg/day).
  • Participants were monitored for two years for hematological and clinical efficacy and safety parameters.
  • Key metrics included fetal hemoglobin (HbF%), total hemoglobin, MCV, vasoocclusive crises, blood transfusions, and hospitalizations.

Main Results:

  • Significant increases in HbF%, total hemoglobin, and mean corpuscular volume were observed.
  • Marked reductions in vasoocclusive crises, blood transfusions, acute chest syndrome, sequestration crises, and hospitalizations occurred.
  • No major adverse events were reported, indicating a favorable safety profile.

Conclusions:

  • Fixed low-dose hydroxyurea (HU) demonstrates significant efficacy in improving hematological markers.
  • HU treatment led to a substantial decrease in clinical complications and hospitalizations in Indian children with SCA.
  • The study confirms that fixed low-dose HU is a safe and effective therapeutic option for pediatric SCA in India.
Abstract

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