Related Experiment Video
Updated: Aug 7, 2026
Comprehensive Evaluation of the Effectiveness and Safety of Placenta-Targeted Drug Delivery Using Three Complementary Methods
Published on: September 10, 2018
Deferiprone, efficacy and safety
V P Choudhry1, H P Pati, Anita Saxena
1Department of Hematology, All India Institute of Medical Sciences, New Delhi, India.
Insights
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.
- 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.
Objective:
Deferiprone (L1), the new oral iron chelator has been studied in several countries for its efficacy and toxicity with some conflicting observations. Toxicity involving joints has been reported more frequently in Indian patients. The authors planned to include larger number of Indian thalassemics in studying safety and efficacy of Deferiprone.
Methods:
Seventy five thalassemic children (4-14 yr) were studied for one year with various investigations done periodically. Thirty patients (group A) received 50 mg/kg dose and 21 others (group B) received 75 mg/kg dose of Deferiprone. Rest of the patients were followed up without any chelator.
Results:
The serum ferritin levels reduced significantly in both groups (P < 0.01 each); more in 75 mg/kg than the 50 mg/kg group. Arthropathy appeared in 15 (50%) patients in Group A and 6 (28.6%) of Group B after 1-12 (mean 6) months of L1 treatment; however, only one patient needed withdrawal of L1. Eleven patients needed indomethacin for pain relief. Seropositivity for antinuclear factor and rheumatoid factor had no relation to dose or duration of L1 therapy, arthropathy or the serum ferritin level. Twelve patients developed leucopenia (< 3.0 x 10(9)/L) and neutropenia (0-1.8 x 10(9)/L) after 2-11 months of L1 therapy and was not related to the dose or duration of therapy. The drug was restarted in 10 patients and only one of them developed a second episode of neutropenia.
Conclusion:
Deferiprone is an effective iron chelator, but arthropathy and neutropenia are very frequent side effects and need strict monitoring during therapy. Most of the neutropenia are neither very severe nor recur with re-challenge with the drug. Similarly, arthropathy does not need withdrawal of drug in majority of patients.
More Related Videos
Related Concept Videos
01:21Factors Affecting Drug Response: Overview
01:26Drug Administration and Therapy Phases: Overview
The pharmaceutical phase focuses on leveraging the physicochemical properties of the drug to design and manufacture an effective product. Variants include orally administered tablets or capsules, topical creams or ointments, and parenteral-delivery solutions or emulsions.
The pharmacokinetic phase...
01:29Therapeutic Drug Monitoring: Affecting Factors
01:14Effect of Hepatic Disease on Pharmacokinetics: Active Drug, Metabolite and Fraction of Metabolized Drug
01:22Effect of Hepatic Disease on Pharmacokinetics: Pathophysiologic Assessment and Liver Function Test
01:08Effect of Hepatic Disease on Pharmacokinetics: Dose Adjustments Due to Hepatic Impairment