Deferasirox for managing iron overload in people with myelodysplastic syndrome

Abstract

We planned to report evidence from RCTs that evaluated the effectiveness of deferasirox compared to either placebo, no treatment or other chelating regimens, such as deferoxamine, in people with MDS. However, we did not identify any completed RCTs addressing this question.We found three ongoing and one completed RCT (published as an abstract only and in insufficient detail) comparing deferasirox with deferoxamine, placebo or no treatment and data will hopefully be available soon. These results will be important to inform physicians and patients on the advantages and disadvantages of this treatment option.

Deferasirox for transfusion‐dependent patients with myelodysplastic syndromes: safety, efficacy, and beyond (GIMEMA MDS0306 Trial)

“…Based on this understanding and the impressive benefits seen in thalassemia populations, the US Food and Drug Administration (FDA) and the European Medicines Agency (EMA) approved the oral iron chelator deferasirox in 2005 for the treatment of ‘chronic iron overload due to blood transfusions’, inclusive of transfusion‐dependent patients with MDS. Since its approval, the body of evidence on the efficacy and safety of deferasirox in patients with MDS has been slowly expanding , although data from randomized, controlled clinical trials are still lacking . Until such data becomes available, further evidence from prospective trials evaluating and confirming the efficacy and safety of deferasirox therapy in MDS remains essential; especially considering the advanced age and high prevalence of comorbidity in this patient population compared with other congenital or acquired anemias.…”

Chelation efficacy and erythroid response during deferasirox treatment in patients with myeloproliferative neoplasms in fibrotic phase

“…In the last few years, several controlled clinical studies have shown that DFX is the preferred treatment for chelation therapy in patients with TS or MDS and transfusion iron overload, with a safety profile that is acceptable and manageable even in elderly subjects . As a consequence, this medication is now commonly employed in the real‐life management of these patients.…”

“…Deferasirox (DFX) is an oral iron chelator widely employed in the treatment of iron overload during thalassemic syndromes (TS) and MDS . The efficacy and safety of DFX in these contexts have been reported in a number of published prospective clinical trials . In addition to the chelation effect, DFX has been associated with an unexpected hematological improvement, which was initially demonstrated in some case reports and subsequently confirmed in about 15–20% of patients in controlled clinical trials .…”

Deferasirox in a refractory anemia after other treatment options: case report and literature review

“…More biological and clinical studies are necessary to better define mechanisms of action of deferasirox and predicting factors of response . Association of iron chelation therapy with other MDS modifying drugs or support strategies may increase rates and duration of response, but accurate assessment of toxicities and a longer follow‐up are mandatory due to moderate or elevate discontinuation rate (40–80%), drug adverse events reported in these patients of over 60 years of age (gastrointestinal and renal episodes mainly): Table .…”