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Deferiprone-associated myelotoxicity
F N al-Refaie1, B Wonke, A V Hoffbrand
1Department of Haematology, Royal Free Hospital, School of Medicine, London, UK.
European Journal of Haematology
|November 1, 1994
Summary
Deferiprone (L1), an oral iron chelator, can cause agranulocytosis and neutropenia in myelodysplasia patients. Early detection and intervention, like G-CSF, are crucial for neutrophil recovery.
Area of Science:
- Hematology
- Pharmacology
Background:
- Myelodysplastic syndromes (MDS) are a group of clonal hematopoietic stem cell disorders.
- Iron overload is a common complication in MDS patients requiring chronic transfusions.
- Deferiprone (L1) is an oral iron chelator used to manage iron overload.
Observation:
- A 63-year-old patient with myelodysplasia developed agranulocytosis (neutrophils 0 x 10(9)/l) 6 weeks after starting deferiprone (79 mg/kg/day).
- This was the third case of agranulocytosis observed during clinical trials of L1 at the Royal Free Hospital.
- Neutrophil count recovered within 7 days after discontinuing L1 and initiating G-CSF (300 micrograms/day).
Findings:
- Four patients in total experienced neutropenia or agranulocytosis during L1 trials.
- The incidence of agranulocytosis associated with deferiprone is approximately 1.6% based on worldwide clinical trials.
- The incidence of neutropenia is approximately 2%.
Implications:
- Deferiprone treatment requires careful monitoring for hematological adverse events, particularly neutropenia and agranulocytosis.
- Prompt recognition and management of these adverse events, including drug cessation and G-CSF administration, can lead to neutrophil count recovery.
- These findings highlight the importance of risk-benefit assessment in patients with myelodysplasia receiving oral iron chelation therapy.