Efficacy and safety of administration of oral iron chelator deferiprone in patients with early myelodysplastic syndrome

Hemoglobin. 2011;35(3):217-27. doi: 10.3109/03630269.2011.578515.

Abstract

Forty-eight patients with early myelodysplastic syndrome (MDS) without excess of blasts, with average initial serum ferritin levels of 2739.5 μg/L (range 825-11287 μg/L), were treated with deferiprone (L1) in a daily dose of 40-90 mg/kg. Median duration of chelation treatment was 10.9 months (range 4-24 months). Chelation was effective (maintained or decreased iron stores) in 16 out of 22 patients (73%) with serum ferritin levels <2000 μg/L in contrast to only 12 out of 26 patients with serum ferritin levels >2000 μg/L. Combination of L1 with recombinant human erythropoietin (rHuEPO) (30-40 kU/week) resulted in effective chelation in five additional patients with serum ferritin levels >3000 μg/L. Incidence of adverse effects was comparable to that in thalassemic patients. Gastrointestinal symptoms represented the most frequent adverse effect of L1 therapy (37.5% of patients) that limited an effective escalation of the daily dose of the drug and led to discontinuation of the treatment for six patients. A decreased number of granulocytes was observed in five (13%) patients and agranulocytosis occurred in two patients (4%). Granulocyte counts were restored after cessation of L1 treatment and administration of granulocyte colony stimulating factor (G-CSF) in all but one patient. Administration of L1 in a daily dose of at least 75 mg/kg may represent an alternative approach in treatment of mild and moderate iron overload in MDS patients who cannot be treated with deferasirox (DFRA) or deferoxamine (DFO).

Publication types

  • Clinical Trial

MeSH terms

  • Adult
  • Aged
  • Aged, 80 and over
  • Agranulocytosis / chemically induced
  • Agranulocytosis / drug therapy
  • Deferiprone
  • Drug Therapy, Combination
  • Drug-Related Side Effects and Adverse Reactions
  • Erythropoietin / therapeutic use
  • Female
  • Ferritins / blood
  • Gastrointestinal Diseases / chemically induced
  • Granulocyte Colony-Stimulating Factor / therapeutic use
  • Humans
  • Iron Chelating Agents / therapeutic use*
  • Iron Overload / drug therapy
  • Iron Overload / prevention & control*
  • Male
  • Middle Aged
  • Myelodysplastic Syndromes / complications*
  • Myelodysplastic Syndromes / drug therapy*
  • Pyridones / administration & dosage
  • Pyridones / adverse effects
  • Pyridones / therapeutic use*
  • Recombinant Proteins
  • Treatment Outcome

Substances

  • Iron Chelating Agents
  • Pyridones
  • Recombinant Proteins
  • Erythropoietin
  • Granulocyte Colony-Stimulating Factor
  • Deferiprone
  • Ferritins