Deferiprone versus Deferoxamine in Sickle Cell Disease: Results from a 5-year long-term Italian multi-center randomized clinical trial

Calvaruso, Giusi; Vitrano, Angela; Maggio, Rosario Di; Ballas, Samir K.; Steinberg, Martin H.; Rigano, Paolo; Sacco, Massimiliano; Telfer, Paul; Renda, Disma; Barone, Rita; Maggio, Aurelio

doi:10.1016/j.bcmd.2014.04.004

Cited by 19 publications

(22 citation statements)

References 29 publications

Supporting

Mentioning

Contrasting

Order By: Relevance

“…The blood chemistry levels indicated that DFX administered for 1 week did not cause any kidney or liver toxicity as compared to control animals (untreated). Safety of DFX administration has also been previously reported in the literature [77–81]. …”

Section: Methodsmentioning

confidence: 72%

Neuroinflammation, oxidative stress, and blood-brain barrier (BBB) disruption in acute Utah electrode array implants and the effect of deferoxamine as an iron chelator on acute foreign body response

et al. 2019

View full text Add to dashboard Cite

The use of intracortical microelectrode arrays has gained significant attention in being able to help restore function in paralysis patients and study the brain in various neurological disorders. Electrode implantation in the cortex causes vasculature or blood-brain barrier (BBB) disruption and thus elicits a foreign body response (FBR) that results in chronic inflammation and may lead to poor electrode performance. In this study, a comprehensive insight into the acute molecular mechanisms occurring at the Utah electrode array-tissue interface is provided to understand the oxidative stress, neuroinflammation, and neurovascular unit (astrocytes, pericytes, and endothelial cells) disruption that occurs following microelectrode implantation. Quantitative real time polymerase chain reaction (qRT-PCR) was used to quantify the gene expression at acute time-points of 48-hr, 72-hr, and 7-days for factors mediating oxidative stress, inflammation, and BBB disruption in rats implanted with a non-functional 4×4 Utah array in the somatosensory cortex. During vascular disruption, free iron released into the brain parenchyma can exacerbate the FBR, leading to oxidative stress and thus further contributing to BBB degradation. To reduce the free iron released into the brain tissue, the effects of an iron chelator, deferoxamine mesylate (DFX), was also evaluated.

show abstract

Section: Methodsmentioning

confidence: 72%

Neuroinflammation, oxidative stress, and blood-brain barrier (BBB) disruption in acute Utah electrode array implants and the effect of deferoxamine as an iron chelator on acute foreign body response

et al. 2019

View full text Add to dashboard Cite

show abstract

“…The only randomized controlled study evaluating ICT compared deferiprone and deferoxamine . Over the course of the 5‐year study, both agents substantially reduced serum ferritin levels, with the number of patients with serum ferritin levels <400 ng/mL being significantly higher in the deferiprone group compared with the deferoxamine group (36.6% vs. 3.3%; P = .002).…”

Section: Resultsmentioning

confidence: 87%

“…The 3 selected studies were published between 2001 and 2014 (Table ) and included 294 patients with characteristics relevant to the study . Only 1 of the 3 studies was designed to compare outcomes of ICT, a 5‐year randomized controlled trial comparing deferiprone and deferoxamine . The other 2 studies were observational, longitudinal, natural history studies, neither of which formally investigated ICT.…”

Section: Resultsmentioning

confidence: 99%

“…The failure to show a statistically significant improvement in survival in this study was likely due to the small sample size, with just 10 patients per arm; there were 6 deaths in the control arm compared with 1 death in the deferoxamine arm. More recent randomized controlled studies that met our entry criteria suggest that there is little difference in efficacy, safety, and tolerability between ICTs . As with the earlier randomized trial of ICT, the failure to observe differences between deferoxamine and newer treatment approaches in these trials in terms of outcomes and tolerability is likely, at least in part, due to the small size of the trials.…”

Section: Discussionmentioning

confidence: 99%

See 1 more Smart Citation

The effect of iron chelation therapy on overall survival in sickle cell disease and β‐thalassemia: A systematic review

et al. 2018

View full text Add to dashboard Cite

Red blood cell transfusions have become standard of care for the prevention of life-threatening anemia in patients with β-thalassemia and sickle cell disease (SCD). However, frequent transfusions can lead to accumulation of iron that can result in liver cirrhosis, diabetes mellitus, arthritis, arrhythmias, cardiomyopathy, heart failure, and hypogonadotropic hypogonadism. Iron chelation therapy has been shown to reduce serum ferritin levels and liver iron content, but limitations of trial design have prevented any demonstration of improved survival. The objective of this systematic review was to investigate the impact of iron chelation therapy on overall and event-free survival in patients with β-thalassemia and SCD. Eighteen articles discussing survival in β-thalassemia and 3 in SCD were identified. Overall iron chelation therapy resulted in better overall survival, especially if it is instituted early and compliance is maintained. Comparative studies did not show any significant differences between available iron chelation agents, although there is evidence that deferiprone is better tolerated than deferoxamine and that compliance is more readily maintained with the newer oral drugs, deferiprone and deferasirox. Iron chelation therapy, particularly the second-generation oral agents, appears to be associated with improved overall and event-free survival in transfusion-dependent patients with β-thalassemia and patients with SCD.

show abstract

“…These agents along with their properties are listed in Table II. There are decades of experience with these agents, showing that they are effective at reducing iron in both SCD and thalassaemia patients (Aydinok, et al 2015, Calvaruso, et al 2014, Hoffbrand, et al 2012, Maggio, et al 2011, Piga, et al 2013, Tsouana, et al 2015, Vichinsky, et al 2011c). There is substantial evidence that these agents improve the clinical outcome in thalassaemia(Modell, et al 2008).…”

Section: Principles Of Iron Overload Management In Scdmentioning

confidence: 99%

How we manage iron overload in sickle cell patients

Coates

Wood

2017

Br J Haematol

View full text Add to dashboard Cite

Summary Blood transfusion plays a prominent role in the management of patients with sickle cell disease (SCD), but causes significant iron overload. As transfusions are used to treat the severe complications of SCD, it remains difficult to distinguish whether organ damage is a consequence of iron overload or is due to the complications treated by transfusion. Better management has resulted in increased survival, but prolonged exposure to iron puts SCD patients at greater risk for iron-related complications that should be treated. The success of chelation therapy is dominated by patient adherence to prescribed treatment; thus, adjustment of drug regimens to increase adherence to treatment is critical. This review will discuss the current biology of iron homeostasis in patients with SCD and how this informs our clinical approach to treatment. We will present the clinical approach to treatment of iron overload at our centre using serial assessment of organ iron by magnetic resonance imaging.

show abstract

Deferiprone versus Deferoxamine in Sickle Cell Disease: Results from a 5-year long-term Italian multi-center randomized clinical trial

Cited by 19 publications

References 29 publications

Neuroinflammation, oxidative stress, and blood-brain barrier (BBB) disruption in acute Utah electrode array implants and the effect of deferoxamine as an iron chelator on acute foreign body response

Neuroinflammation, oxidative stress, and blood-brain barrier (BBB) disruption in acute Utah electrode array implants and the effect of deferoxamine as an iron chelator on acute foreign body response

The effect of iron chelation therapy on overall survival in sickle cell disease and β‐thalassemia: A systematic review

How we manage iron overload in sickle cell patients

Contact Info

Product

Resources

About