Exjade for transfusion iron overload and thalassemia – pro

Iron overload is a cumulative, potentially life-threatening, consequence of frequent blood transfusions or conditions such as thalassemia. Iron starts to build up in the body after as few as 10 transfusions because the body cannot remove it on its own. Iron chelation is the only effective drug treatment for transfusion-related iron overload. In thalassemia, ineffective erytrhopoieisis causes iron overload as well. In iron chelation, an agent binds to iron in the body and tissues and helps remove it through the urine and/or feces.

Exjade (deferasirox) is indicated for the treatment of chronic iron overload due to blood transfusions (transfusional hemosiderosis) in patients 2 years of age and older. The FDA does not provide criteria for iorn overload but increased deposition in the organs is generally accepted to be iron overload. MRI si more senstive in identifying iron overload than iron studies.

Exjade is administered once-daily as a drink. Exjade was developed specifically to meet the high unmet medical need for iron chelation despite the availability of deferoxamine, the standard iron chelator used around the world. While effective, deferoxamine requires nightly infusions by needle and pump, often lasting eight to 12 hours per night for five to seven nights a week as long as the patient continues to receive blood transfusions or has excess iron within the body. As a result, many patients may have stopped or avoided iron chelation therapy, thus risking the toxic effects of iron overload. Due to the burdensome administration of deferoxamine, compliance with standard chelation therapy is poor. Previous studies of patients with thalassemia have shown that good compliance with deferoxamine improves survival and quality of life.

Iron overload secondary to thalassemia also responds to Exjade. A phase III trial in thalassemia major patients demonstrated non-inferiority to deferoxamine at doses of over 20 mg/kg/day. Non-inferiority at lower doses of deferasirox was not established but this may have been solely due to study design. Therefore, in thalassemia, the optimal dose of deferasirox still need to be clarified.
A randomized, open-label, Phase III trial evaluated Patient-Reported Outcomes (PROs) at the end of one year and found that significantly more patients on deferasirox as compared to those on deferoxamine reported treatment satisfaction (89% vs. 41%, respectively) and treatment convenience (93% vs. 11%).65 Of those previously treated with deferoxamine, 97% of those in the deferasirox arm indicated a preference for deferasirox and 86% indicated a willingness to continue treatment as compared to 14% of those assigned to the deferoxamine group. These findings suggest that patients are generally compliant with Exjade therapy.

Another large study, was THALASSA, the first pivotal placebo-controlled studythat examined the benefit of iron chelation with Exjade® (deferasirox) in patients with non-transfusion-dependent thalassemia (NTDT), and it demonstrated that Exjade can significantly reduce iron overload.

Jadenu is the same drug as Exjade. indicated for treatment of chronic iron overload caused by nontransfusion-dependent thalassemia syndromes and with a liver iron (Fe) concentration (LIC) of at least 5 mg Fe per gram of dry weight (dw) and a serum ferritin >300 mcg/L

Vichinsky E, Bernaudin F, Forni GL, Gardner R, Hassell K, Heeney MM, Inusa B, Kutlar A, Lane P, Mathias L, Porter J, Tebbi C, Wilson F, Griffel L, Deng W, Giannone V, Coates T.Long-term safety and efficacy of deferasirox (Exjade) for up to 5 years in transfusional iron-overloaded patients with sickle cell disease.Br J Haematol. 2011 Aug;154(3):387-97. doi: 10.1111/j.1365-2141.2011

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