Deferasirox for managing iron overload in people with myelodysplastic syndrome

Review question

We aimed to review the evidence about the effects of deferasirox (an oral therapeutic option) on reducing iron overload in people with myelodysplastic syndrome (MDS), which is a diverse group of haematopoietic stem cell disorders.

Background

Repeated red blood cell transfusions can lead to clinically relevant secondary (i.e. due to the transfusions) iron overload in some people with MDS, particularly of lower risk groups over the course of their disease. Since the human body has no natural means of removing excess iron, drugs to remove the excess iron (iron chelation therapy) might be indicated to prevent organ complications. Since the newer oral iron chelator deferasirox has become available, iron chelation therapy is offered more widely to people with MDS.

We wanted to assess whether administering deferasirox is beneficial in people with MDS.

Key results

We searched the available literature up to 03 April 2014. We could not include any data in this review that answered our question. However, we found three ongoing trials and one completed trial investigating deferasirox in people with MDS of lower risk groups (low and intermediate-1 risk MDS). As the completed trial has only been reported in insufficient detail (in abstract format), we were unable to definitively decide on inclusion of this study or draw any conclusions from this. Once available, these results will be important to inform physicians and patients on the comparative advantages and disadvantages of this treatment option.

Authors' conclusions: 

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.

Read the full abstract...
Background: 

The myelodysplastic syndrome (MDS) comprises a diverse group of haematopoietic stem cell disorders. Due to symptomatic anaemia, most people with MDS require supportive therapy including repeated red blood cell (RBC) transfusions. In combination with increased iron absorption, this contributes to the accumulation of iron resulting in secondary iron overload and the risk of organ dysfunction and reduced life expectancy. Since the human body has no natural means of removing excess iron, iron chelation therapy, i.e. the pharmacological treatment of iron overload, is usually recommended. However, it is unclear whether or not the newer oral chelator deferasirox leads to relevant benefit.

Objectives: 

To evaluate the effectiveness and safety of oral deferasirox for managing iron overload in people with myelodysplastic syndrome (MDS).

Search strategy: 

We searched the following databases up to 03 April 2014: MEDLINE, EMBASE, The Cochrane Library, Biosis Previews, Web of Science, Derwent Drug File and four trial registries: Current Controlled Trials (www.controlled-trials.com), ClinicalTrials.gov (www.clinicaltrials.gov), ICTRP (www.who.int./ictrp/en/), and German Clinical Trial Register (www.drks.de).

Selection criteria: 

Randomised controlled trials (RCTs) comparing deferasirox with no therapy, placebo or with another iron-chelating treatment schedule.

Data collection and analysis: 

We did not identify any trials eligible for inclusion in this review.

Main results: 

No trials met our inclusion criteria. However, we identified three ongoing and one completed trial (published as an abstract only and in insufficient detail to permit us to decide on inclusion) comparing deferasirox with deferoxamine, placebo or no treatment.

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