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Diamond–Blackfan Anemia
Published in Dongyou Liu, Handbook of Tumor Syndromes, 2020
Early treatment of iron overload in DBA patients who fail to respond to steroid therapy and require RBC transfusions helps improve the prognosis. However, deferiprone may cause neutropenia, and should be avoided as a treatment of iron overload in persons with DBA.
Neoliberalist approaches to healthcare and the transnational pharmaceutical corporations
Published in Théodore H MacDonald, Removing the Barriers to Global Health Equity, 2018
Deferiprone helps clear iron from the blood which builds up in patients with thalassaemia and can prove fatal. At first the trial went well and Dr Olivieri published promising results in the New England Journal of Medicine. Then she noticed worrying liver changes in some of her patients. She raised her concerns with the company and tried to find a way of adapting the trial. But she was unprepared for the response of the company, whose potential million-dollar drug she was now questioning.
Case 40
Published in Atul B. Mehta, Keith Gomez, Clinical Haematology, 2017
Iron chelation therapy is usually administered in the form of parenteral desferrioxamine (DFO, typically given by subcutaneous infusion over 12 hours, five nights each week, with oral vitamin C). Side effects of DFO include sensory changes (hearing loss, visual defects), and bone and cartilage abnormalities. The necessity of parenteral administration contributes toward poor compliance. Deferasirox is a once-daily oral iron chelator which can cause renal and gastrointestinal (GI) disturbances but is generally well tolerated. Deferiprone is an oral iron chelator which seems to be particularly effective at removing cardiac iron. Side effects include agranulocytosis (1%), arthralgia and GI disturbances. Cardiac iron is best measured by magnetic resonance imaging (MRI). Haemopoietic stem cell or umbilical cord stem cell transplantation is curative for selected patients.
An evaluation of deferiprone as twice-a-day tablets or in combination therapy for the treatment of transfusional iron overload in thalassemia syndromes
Published in Expert Review of Hematology, 2023
Richa Shah, Aashaka Shah, Sherif M. Badawy
Although uncommon, the most serious side effect associated with deferiprone use is agranulocytosis [74]. Most studies in patients on DFP treatment define agranulocytosis to be an absolute neutrophil count <0.5 × 109/L [74]. Most recent definitions suggest that agranulocytosis should be defined as an absolute neutrophil count of less than 100 [84]. This suggests that although agranulocytosis is a significant side effect that patients should be monitored for while on DFP treatment, the incidence of serious agranulocytosis is possibly lower than published in the initial literature. Furthermore, the use of DFP in other hematologic conditions aside from thalassemia has not shown an increase in the risk of agranulocytosis [85]. It is also worth noting that the risk for neutropenia or agranulocytosis is not dose-dependent and that the risk is usually lower after the first 6 months of DFP therapy.
Drug safety in thalassemia: lessons from the present and directions for the future
Published in Expert Opinion on Drug Safety, 2021
Laura Grech, Janet Sultana, Karen Borg, Joseph Borg
Deferiprone (Ferriprox® or Kelfer®) was the first oral bidentate iron chelator approved in Europe in the 1990s while in the US, it was approved in 2011. Deferiprone is usually given to TDT patients when deferoxamine therapy is contraindicated or inadequate. Deferiprone binds to ferric ions and forms a 3:1 (deferiprone:iron) stable complex that is then eliminated in urine. Deferiprone is usually given at a dose of 75–100 mg/kg/day divided over 3 doses [28]. In β-thalassemia patients, deferiprone was found to improve liver iron concentration and cardiac iron load and sometimes the treatment is combined with deferoxamine [7]. A number of ADRs, such as gastrointestinal (GI) disturbances, increased liver enzyme levels, and arthropathy, have been linked to deferiprone, with agranulocytosis and neutropenia being the most serious adverse events (AE) [29–32].
Emerging therapies in Friedreich’s Ataxia
Published in Expert Review of Neurotherapeutics, 2020
Theresa A. Zesiewicz, Joshua Hancock, Shaila D. Ghanekar, Sheng-Han Kuo, Carlos A. Dohse, Joshua Vega
FRDA causes a marked reduction of frataxin, leading to oxidative damage and subsequent neurodegeneration [54]. An important role of frataxin is to assist in the formation of mitochondrial iron-sulfur (Fe-S) clusters (ISC) [55], which are vital to cellular respiration and energy production [56]. Iron accumulation has been found in the cell models and FRDA patient tissues; therefore, iron chelators might be an appealing strategy for FRDA. Deferiprone is an orally administered iron chelator with good permeability that has been tested as a treatment of FRDA. One double-blind, placebo-controlled study randomized 72 FRDA patients to receive deferiprone 20, 40, or 60 mg/kg/day or placebo for 6 months [57]. Deferiprone was generally well tolerated at the lowest dose of 20 mg/kg/day, but two patients experienced worsening ataxia taking the highest dose of 60 mg/kg/day. An additional patient experienced reversible neutropenia while taking 20 mg/kg/day. There were no significant changes in the FARS in the 20 mg/kg/day group, and worsening was observed in those patients who received 40 mg/kg/day. However, patients in all dose groups had a decrease in cardiac hypertrophy [57]. The future of iron chelators as a treatment for FRDA remains unclear at present.