DESFERAL SUMMARY
Desferal, deferoxamine mesylate USP, is an iron-chelating agent, available in vials for intramuscular, subcutaneous, and intravenous administration.
Desferal is indicated for the treatment of acute iron intoxication and of chronic iron overload due to transfusion-dependent anemias.
Desferal is an adjunct to, and not a substitute for, standard measures used in treating acute iron intoxication, which may include the following: induction of emesis with syrup of ipecac; gastric lavage; suction and maintenance of a clear airway; control of shock with intravenous fluids, blood, oxygen, and vasopressors; and correction of acidosis.
Desferal can promote iron excretion in patients with secondary iron overload from multiple transfusions (as may occur in the treatment of some chronic anemias, including thalassemia). Long-term therapy with Desferal slows accumulation of hepatic iron and retards or eliminates progression of hepatic fibrosis.
Iron mobilization with Desferal is relatively poor in patients under the age of 3 years with relatively little iron overload. The drug should ordinarily not be given to such patients unless significant iron mobilization (e.g., 1 mg or more of iron per day) can be demonstrated.
Desferal is not indicated for the treatment of primary hemochromatosis, since phlebotomy is the method of choice for removing excess iron in this disorder.
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NEWS HIGHLIGHTS
Published Studies Related to Desferal (Deferoxamine)
Long-term sequential deferiprone-deferoxamine versus deferiprone alone for thalassaemia major patients: a randomized clinical trial. [2009.04]
A multicentre randomized open-label trial was designed to assess the effectiveness of long-term sequential deferiprone-deferoxamine (DFO-DFP) versus DFP alone to treat thalassaemia major (TM). DFP at 75 mg/kg, divided into three oral daily doses, for 4 d/week and DFO by subcutaneous infusion (8-12 h) at 50 mg/kg per day for the remaining 3 d/week was compared with DFP alone at 75 mg/kg, administered 7 d/week during a 5-year follow-up...
Inflammation and oxidant-stress in beta-thalassemia patients treated with iron chelators deferasirox (ICL670) or deferoxamine: an ancillary study of the Novartis CICL670A0107 trial. [2008.06]
BACKGROUND: We assessed whether oxidant-stress and inflammation in beta-thalassemia could be controlled by the novel oral iron chelator deferasirox as effectively as by deferoxamine... CONCLUSIONS: Iron chelation therapy with deferoxamine or with deferasirox was equally effective in decreasing iron burden and malondialdehyde. The possible differential effect of the two chelators on inflammation warrants further investigation.
Patient-reported outcomes of deferasirox (Exjade, ICL670) versus deferoxamine in sickle cell disease patients with transfusional hemosiderosis. Substudy of a randomized open-label phase II trial. [2008]
BACKGROUND/AIMS: There is increasing evidence demonstrating the value of transfusions in sickle cell disease (SCD). However, resultant iron overload can be life threatening if untreated. Chelation therapy with deferoxamine requires parenteral infusions that can negatively impact quality of life and adherence to treatment... CONCLUSIONS: Patients with SCD are therefore more satisfied with deferasirox, which has a lower impact on daily activities than deferoxamine. Given the high levels of satisfaction, it is likely that quality of life will be improved. These results also suggest that treatment adherence with deferasirox may be better than with deferoxamine, which should lead to improved long-term outcomes. 2008 S. Karger AG, Basel.
Prospective evaluation of patient-reported outcomes during treatment with deferasirox or deferoxamine for iron overload in patients with beta-thalassemia. [2007.05]
BACKGROUND: Iron chelation therapy (ICT) with deferoxamine (DFO), the current standard for the treatment of iron overload in patients with transfusion-dependent disorders such as beta-thalassemia, requires regular subcutaneous or intravenous infusions. This can lead to reduced quality of life and poor adherence, resulting in increased morbidity and mortality in iron-overloaded patients with beta-thalassemia. Deferasirox is an orally administered iron chelator that has been approved for use in the United States, Switzerland, and other countries. OBJECTIVE: This analysis was conducted to compare patient-reported outcomes (PROs) during receipt of DFO infusions or once-daily oral therapy with deferasirox (ICL670)... CONCLUSIONS: In this study, patient-reported satisfaction and convenience were significantly higher for the once-daily, oral ICT deferasirox than for DFO infusions. Among patients who had received DFO before the study, the majority indicated a preference for deferasirox over DFO. Most patients receiving deferasirox indicated that they would be willing to continue taking it. These results suggest that deferasirox had a positive impact on patients' daily lives.
A randomized, placebo-controlled, double-blind trial of the effect of combined therapy with deferoxamine and deferiprone on myocardial iron in thalassemia major using cardiovascular magnetic resonance. [2007.04.10]
CONCLUSIONS: In comparison to the standard chelation monotherapy of deferoxamine, combination treatment with additional deferiprone reduced myocardial iron and improved the ejection fraction and endothelial function in thalassemia major patients with mild to moderate cardiac iron loading.
Clinical Trials Related to Desferal (Deferoxamine)
Thrombolysis and Deferoxamine in Middle Cerebral Artery Occlusion [Recruiting]
Iron overload has been associated with greater brain injury in ischemia/reperfusion
experimental stroke models and ischemic stroke patients, especially in those treated with
thrombolytic treatment. Deferoxamine administration, an iron chelator, offers a
neuroprotective action in ischemia/reperfusion animal models.
Primary objective: To evaluate the security and tolerability of deferoxamine endovenous
treatment in acute ischemic stroke patients treated with iv. tPA.
Secondary objectives: To study pharmacokinetics of deferoxamine given by endovenous bolus
(10 mg/Kg) followed by 72-hour continuous intravenous infusion (20, 40 o 60 mg/Kg). To
evaluate the deferoxamine effect in clinical outcome, infarct volume and hemorrhagic
transformation and brain edema development.
Methodology: Double-blind, randomized, placebo controlled, dose-finding phase II clinical
trial. Study stages: 1st: bolus+20 mg/Kg/day vs. Placebo (n=15: 5); 2nd: bolus+40 mg/Kg/day
vs. Placebo (n=15: 5); 3rd: bolus+60 mg/Kg/day vs placebo (n=15: 5). These doses will be
increased according to security results of the previous stage. Patients will be continuously
monitored in stroke units. Laboratory parameters will be measured at baseline, 24h, 72h and
30 days to evaluate adverse events related to the drug. Serum deferoxamine and feroxamine
concentrations will be measured along time after the injection in a subgroup of patients to
the pharmacokinetics study. CT scan will be performed at 24-36h to assess hemorrhagic
transformation and brain edema. The NIH Stroke Scale will be evaluated during
hospitalization, and the Rankin score at discharge and 3 months.
If deferoxamine demonstrate to be secure and well tolerated treatment in acute stroke
patients, it may be a new therapy option to lower the brain injury after ischemia and
reperfusion.
Iron Balance Study of Deferasirox, Deferoxamine and the Combination of Both [Recruiting]
Subjects with thalassemia major require regular transfusion therapy to sustain life. The
iron present in the transfused blood remains in the body where it can cause a variety of
organ dysfunctions. Lifelong iron chelation therapy is needed to maintain iron balance but
its effectiveness varies greatly. Like that of deferoxamine (Desferal, DFO) the mainstay of
chelation therapy for 30 years, the effectiveness of deferasirox (Exjade, ICL670), the newly
approved, orally effective iron chelating drug, is not satisfactory in all subjects. Even
with good compliance, the iron excretion induced by a given drug exhibits wide
subject-to-subject variability. There is often persistent iron overload of extra hepatic
tissues such as the heart and pancreas leading to cardiac disease and diabetes. Combining
the drugs may be a better approach in those subjects at increased risk. The iron balance
studies proposed will permit an assessment of the potential of such a combination to place
subjects in net negative iron balance and the relative effectiveness of the combination in
relation to that of the individual drugs, an additive effect being expected. With such
information, physicians will be able to design individualized chelation regimens that
maximize effectiveness while minimizing side effects by adjusting the ratio and/or the
dosing schedule of the two drugs.
Safety & Efficacy of ICL670 Vs. Deferoxamine in Beta-Thalassemia Patients With Iron Overload Due to Blood Transfusions [Completed]
The purpose of this study is to deterimine if the new orally active iron chelator, ICL670, is
as effective and as safe as deferoxamine in preventing accumulation of iron in the body while
a patient is undergoing repeated blood transfusions.
Evaluating Use of Deferasirox as Compared to Deferoxamine in Treating Cardiac Iron Overload [Recruiting]
This is a clinical research study in patients who have iron overload in the heart due to
chronic blood transfusions.
The study will have 2 treatment groups and will compare the safety and efficacy of chelation
therapy with a medicine called deferasirox (ICL670) with another medicine called
deferoxamine (DFO). The study is aimed at finding out which of the two medicines is the best
for treating iron overload in the heart.
Patients will be treated for 12 months (core study phase). Patients who complete the core
study phase will be offered to continue their study treatment in a 12 months extension
phase. During the core and extension, the effects of treatment on iron overload in the heart
and the liver will be evaluated using specific magnetic resonance imaging (MRI) assessments.
Combo Chelation Trial [Recruiting]
This is a small pilot study looking at the safety of giving combination chelation with
Exjade and Desferal to 15 patients. The hypothesis is that combination chelation is safe in
decreasing overall iron in patients with thalassemia.
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