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Published on: March 14, 2017
A trial of deferiprone in transfusion-dependent iron overloaded children
G N Lucas1, B J Perera, E A Fonseka
1Lady Ridgeway Hospital for Children, Colombo.
Insights
Deferiprone effectively reduces iron overload in children with transfusion dependence. This oral iron chelator demonstrated significant improvements in serum ferritin levels with manageable side effects like arthropathy and neutropenia.
Area of Science:
- Pediatric Hematology
- Pharmacology
- Clinical Medicine
Background:
- Iron overload is a serious complication in children requiring frequent blood transfusions.
- Effective iron chelation therapy is crucial to prevent organ damage and improve patient outcomes.
Purpose of the Study:
- To evaluate the efficacy and safety of deferiprone in treating transfusion-dependent iron overloaded children.
- To assess the impact of deferiprone on serum ferritin levels and identify adverse events.
Main Methods:
- A prospective study was conducted in 5 pediatric medical units.
- 54 children (2-15 years) with transfusion-dependent iron overload received oral deferiprone (75 mg/kg/day).
- Efficacy was measured by serum ferritin assays; safety was monitored via blood counts and liver function tests.
Main Results:
- Deferiprone therapy significantly reduced mean serum ferritin levels from 5743 ng/ml to 3558 ng/ml (p < 0.001).
- 47 out of 48 patients showed decreased serum ferritin levels.
- Adverse events included one case of severe neutropenia and arthropathy in 11 children, both manageable.
Conclusions:
- Deferiprone is a safe and effective oral iron chelating agent for children with transfusion-dependent iron overload.
- Strict medical supervision is recommended during deferiprone treatment.
- The benefits of deferiprone in managing iron overload outweigh the risks when properly monitored.
Objective:
To determine the efficacy and safety of deferiprone.
Design:
Prospective study.
Setting:
5 paediatric medical units at the Lady Ridgeway Hospital for Children (LRHC), Colombo.
Patients:
Transfusion-dependent iron overloaded children in the age group 2 to 15 years.
Intervention:
Patients were given a total daily dose of 75 mg/kg of deferiprone orally in divided doses.
Measurements:
Efficacy of deferiprone therapy was assessed by 4-monthly serum ferritin assays using the ELISA technique. Safety of deferiprone therapy was assessed by 4-weekly white cell counts, platelet counts and serum transaminase levels. The Z-test was used to assess the significance of the difference between the mean initial serum ferritin level and the mean subsequent serum ferritin level.
Results:
54 patients received deferiprone therapy for a mean duration of 9 +/- 3 months. Initial serum ferritin levels ranged from 1500 to 10,700 ng/ml with a mean of 5743. Subsequent serum ferritin levels, obtained in 48 patients ranged from 740 to 7300 ng/ml with a mean of 3558 (p < 0.001). In 47 of the 48 patients subsequent serum ferritin levels were lower than initial levels. One child developed severe neutropaenia, which reverted to normal on discontinuation of treatment. 11 children developed arthropathy, which responded to ibuprofen therapy combined in some cases with a reduction of the dose of deferiprone to 50 mg/kg/day. Serum transaminase levels were raised in 5 patients but reverted to pretreatment values or lower despite continuation of deferiprone therapy.
Conclusions:
Deferiprone is a safe and effective oral iron-chelating agent which can be used, under strict supervision, in transfusion-dependent iron overloaded children.
