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Deferiprone, efficacy and safety.

V P Choudhry1, H P Pati, Anita Saxena

  • 1Department of Hematology, All India Institute of Medical Sciences, New Delhi, India.

Indian Journal of Pediatrics
|April 15, 2004
PubMed
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Deferiprone effectively reduces iron levels in thalassemia patients, but joint pain (arthropathy) and low white blood cell counts (neutropenia) are common side effects requiring monitoring. Most cases of neutropenia and arthropathy are manageable without drug discontinuation.

Area of Science:

  • Hematology
  • Pharmacology
  • Pediatrics

Background:

  • Deferiprone (L1) is an oral iron chelator used for thalassemia.
  • Conflicting observations exist regarding its efficacy and toxicity, particularly joint toxicity in Indian patients.
  • A study was designed to assess Deferiprone's safety and efficacy in a larger cohort of Indian thalassemic children.

Purpose of the Study:

  • To evaluate the safety and efficacy of Deferiprone in Indian children with thalassemia.
  • To investigate the incidence of adverse effects, specifically arthropathy and neutropenia, at different dosages.
  • To establish monitoring guidelines for Deferiprone therapy.

Main Methods:

  • A one-year prospective study involving 75 thalassemic children aged 4-14 years.

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  • Patients were divided into three groups: 50 mg/kg Deferiprone, 75 mg/kg Deferiprone, and a control group without chelation.
  • Periodic investigations were conducted to monitor efficacy (serum ferritin) and safety (joint pain, blood counts).

    • Main Results:

    • Significant reduction in serum ferritin levels observed in both Deferiprone groups (P < 0.01), with greater reduction in the 75 mg/kg group.
    • Arthropathy occurred in 50% of the 50 mg/kg group and 28.6% of the 75 mg/kg group; only one patient required drug withdrawal.
    • Leukopenia and neutropenia developed in 12 patients, generally not severe and often manageable upon re-challenge with Deferiprone.

    Conclusions:

    • Deferiprone is an effective iron chelator for pediatric thalassemia.
    • Arthropathy and neutropenia are frequent but often manageable side effects requiring vigilant monitoring.
    • Most cases of neutropenia and arthropathy do not necessitate drug cessation and can be managed conservatively.