Deferiprone.
Deferiprone (DFP) has been evaluated in a wide range of disorders, but most data come from transfusion-dependent thalassemia. The safety and tolerability profile includes gastrointestinal complaints, liver enzymes elevation, weight gain, arthropathy, neutropenia, and agranulocytosis. The last requires close monitoring of blood count and precludes the use of DFP in conditions with bone marrow abnormalities. The efficacy profile is similar among the three available chelators. For DFP, the choice of dosage is crucial to optimize the effect on liver iron concentration, according to the iron load degree and transfusional iron input. Growing evidence indicates that DFP, alone or in combination with deferoxamine, is effective in removing cardiac iron and preventing cardiac iron load. The available data consolidate an important role of DFP in the management of iron overload. There is a need to compare directly the relative value of the available chelators in the long-term prevention of iron toxicity by well-designed randomized controlled trials.
Piga
Antonio
A
Thalassemia Center, University of Turin, Orbassano, Italy. antonio.piga@unito.it
Roggero
Simona
S
Salussolia
Ilaria
I
Massano
Davide
D
Serra
Melania
M
Longo
Filomena
F
D000077543:Deferiprone; D003676:Deferoxamine / Q000627:therapeutic use; D006801:Humans; D007501:Iron / Q000378:metabolism; D007502:Iron Chelating Agents / Q000627:therapeutic use*; D008099:Liver / Q000378:metabolism; D011728:Pyridones / Q000627:therapeutic use*; D016032:Randomized Controlled Trials as Topic; D013789:Thalassemia / Q000188:drug therapy*; D065227:Transfusion Reaction
D007502:Iron Chelating Agents; D011728:Pyridones; D000077543:Deferiprone; D007501:Iron; D003676:Deferoxamine