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Experience with the oral iron chelator deferiprone in transfusion-dependent children
G N Lucas1, B J C Perera, E A N Fonseka
1Lady Ridgeway Hospital for Children, Colombo 8.
Insights
Deferiprone therapy effectively reduced iron overload in children with transfusion-dependent conditions. However, potential side effects like arthropathy and agranulocytosis require careful monitoring.
Area of Science:
- Pediatric Hematology
- Pharmacology
- Iron Chelation Therapy
Background:
- Iron overload is a serious complication in children requiring frequent blood transfusions.
- Effective iron chelation therapy is crucial for managing long-term health outcomes.
Purpose of the Study:
- To evaluate the efficacy and safety of deferiprone in transfusion-dependent children.
- To assess the impact of deferiprone on serum ferritin levels and identify adverse events.
Main Methods:
- A prospective study was conducted involving 82 transfusion-dependent children aged 1 to 15 years.
- Patients received oral deferiprone at 75 mg/kg/day.
- Efficacy was monitored via serum ferritin assays; safety assessed through white blood cell counts and ALT levels.
Main Results:
- Deferiprone therapy for a mean of 30 months significantly reduced mean serum ferritin levels (5156 to 2809 µg/L, p < 0.001).
- 52% of children achieved a final serum ferritin level below 2500 µg/L.
- Adverse events included agranulocytosis (2.4%), arthropathy (50%, severe in 17%), and elevated ALT levels (43%).
Conclusions:
- Deferiprone is effective in reducing iron overload in pediatric patients.
- Severe arthropathy and agranulocytosis are potential risks that may necessitate treatment discontinuation.
Objective:
To establish efficacy and safety of deferiprone.
Design:
Prospective study.
Setting:
The Lady Ridgeway Hospital for Children, Colombo.
Patients:
Transfusion-dependent children in the age group 1 to 15 years.
Intervention:
Patients were given 75 mg/kg/day of deferiprone orally in divided doses.
Measurements:
Efficacy of deferiprone therapy was assessed by 4 to 6 monthly serum ferritin (SF) assays. Safety of therapy was assessed by 4-weekly white cell counts and serum alanine aminotransferase (ALT) levels. The Z-score was used to assess the significance of the difference between the mean initial and final SF level.
Results:
82 patients received deferiprone therapy for a mean duration of 30 +/- 14 months. Initial SF levels ranged from 1115 to 12,165 micrograms/l with a mean of 5156 +/- 2631 micrograms/l. Final SF levels ranged from 312 to 15,285 micrograms/l with a mean of 2809 +/- 2380 micrograms/l (Z score 5.99; p < 0.001). Two (2.4%) children developed agranulocytosis which reverted to normal on discontinuation of treatment. 41 (50%) developed arthropathy and in 17 this was severe enough to require discontinuation of therapy. Serum ALT levels were raised in 35 (43%) patients but reverted to pretreatment values or lower despite continuation of deferiprone therapy. There was one death in a 9-year old child who developed diabetes mellitus and heart failure despite deferiprone therapy for 3 years.
Conclusions:
A final SF level < 2500 micrograms/l was achieved in 52% children. Severe arthropathy and agranulocytosis may necessitate permanent discontinuation of therapy.