New oral drug could make Thalassemia treatment less painful for children

Courtesy by DailyTimes.Com.Pk!

* Ferriprox tablets can be used as iron chelation treatment instead of Desferal injections

By Tanveer Sher

KARACHI: Any child who has Thalassemia, a blood disorder that requires a lifetime of transfusions, will tell you that five to seven days a week they have to undergo the painful treatment of receiving injections for eight to 12 hours in order to flush out the iron buildup in their system.

According to hematologist Dr Tahir Shamsi, children with Thalassemia major need to receive blood transfusions to overcome their shortage of red blood cells, which otherwise makes them pale and tired. Most children with Thalassemia major need blood transfusions every four to six weeks for the rest of their lives but with regular transfusions, these children grow normally and are able to do all the things other children do.

But as blood contains iron, which the body cannot get rid of naturally, the multiple transfusions result in a build up of the element. After ten or more years, the presence of such levels of iron may cause complications, which can result in poor growth and development, liver and heart disease, and even death.

According to Shamsi, in order to prevent these problems, children with Thalassemia major needed regular treatment to remove the excess iron from their bodies for which Desferal (R) (deferoxamine mesylate) (Desferal (R) is a trademark of Novartis Inc.) has been administered for decades.

Iron chelation is the only effective therapy for removing excess iron. Until recently, the only chelator available was Desferal, a drug that must be injected under the skin for 8-12 hours, for 5-7 nights per week. This treatment is exceedingly painful for the young patients and many parents are reluctant to put them through the agony. Unfortunately, this puts their child’s health at risk.

But now, a tablet can replace these painful injections, Shamsi says. Ferriprox (TM) (deferiprone), (Ferriprox (TM) is a trademark of Apotex Inc.) the first iron chelator which can be given by mouth, is currently approved in 48 countries, including the European Union, for the treatment of iron overload in patients with Thalassemia major for whom deferoxamine therapy is contra-indicated or who present serious toxicity with deferoxamine therapy.

Ferriprox is approved by the American Food and Drug Agency (FDA). The new medicine is used once a day by the patient and helps remove the accumulated iron from the body through urine and stool.

So far, more than 50 children have been given the new medicine, Dr Shamsi said while referring to treatment at the Bismillah Taqi Blood Disease Center.

He said that every year more than 5,000 new cases of Thalassemia major are detected in the country and currently 55,000 patients are registered with the Pakistan Thalassemia Federation.

Hematologists have identified the marriage of two Thalassemia minor people as the major cause of the spread of the illness as their newborn in most of the cases was found to be a patient of Thalassemia major.

There are more than 10 million Thalassemia minor patients in the country and unless the government introduces a law banning the union of two Thalassemia minor people, more children are likely to be born with the disease. (Ed’s note: Kindly consult a physician or your doctor before you make any decision about the medications mentioned above.)

2 thoughts on “New oral drug could make Thalassemia treatment less painful for children”

  1. If you only knew how many times I have looked for decent content about \”oral chelation therapy\”. Well done – I really appreciate your site. Thank you.

  2. i ve sum information that i wud like to share
    ferriprox otherwise known as L1 is not FDA approved as a first line drug and plus it has got a no of side effect which all the pt using it ll tell u that they r not comfortable wid it. also the best way to use ferriprox is to use it wid desferal inf so the agony and pain of the desferal is still there. how ever there is a new drug that is available in pakistan is by the name of asunra or interanaionally known as exjade. it is the only and very recent oral drug that removes iron wid a once a day oral dose and that 2 effective as desferal and less damaging than ferriprox. this drug is available only in pakistan thalasemia society and in jamila sultana foundation. if anyone wants to know furhter plz contact there institutoins.there consultant ll tell u all there is to know

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