Progressive organdysfunction is the consequence of iron-deposition caused by multiple transfusions in patients with transfusion dependend anaemia's like homozygous β-thalassaemia or Diamond-Blackfan anaemia (DBA). Regular chelation with deferioxamine diminishes irondeposition, stops liverfibrosis and stabilises myocardial damage. Problems with deferioxamine-treatment are well-known: local irritation at injectionsites, noncompliance, neurotoxicity and costs.
One of the oral iron-chelators, 1,2 dimethyl-3-hydroxy-pyrid-4-one (L1 or Deferiprone) has proven efficacy in Fe-excretion in urine. In the eighties studies in adults were started in several countries. Since 01.01.1993 a dutch national study was started for children for whom deferioxamine was not feasible.
At 01.01.1996 12 children entered the protocol, treated in four university pediatric departments; β-thalassaemia 4, DBA 4, HbSS 2, MDS 1, PK-deficiency 1. Minimal duration of treatment 6 months, maximal 3 years. Number of transfusions 50 to > 300, age 7-14 years. Initial Ferritin-values 750-5570 μg/l. Dosage of L1 was 50 mg/kg/24 hrs in 3 doses, in average. Effectivity was measured as 24 hours Fe-excretion in urine; for 2 patients the NMR liver/muscle ratio was used for follow-up.
Results: Fe-excretion measured from 1 to 22 mg/day with great intra-individual variation. In 3 cases negative iron-balance was reached. Ferritin-values showed a rise in 2, a decline in 2, 8 remained stable. NMR follow-up showed a decline in one patient. Adverse effects: Joint-pain in knees: 2, agranulocytosis: 1.
Conclusions: Deferiprone is an effective oral iron chelator, with acceptable side-effects. Only 25% of treated patients reached negative ironbalance; the effect seems to be dose-dependent.
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van Weel-Sipman, M. THE DUTCH EXPERIENCE WITH THE ORAL CHELATOR DEFERIPRONE 156. Pediatr Res 41, 777 (1997). https://doi.org/10.1203/00006450-199705000-00175
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DOI: https://doi.org/10.1203/00006450-199705000-00175