Study of Exjade (defrasirox) taken with food

Standard is 500 in Toronto. Standard practice in Toronto, exjade is stopped at 500 and resumes at 1000. We had asked multiple times the reason for a break. The answer we got that it is safe practice for children, you do not want to overburden their bodies, levels below 500 for longer period of times (in children) will chelate essential minerals from children. Infact time and again it is proven that children are different than adults, who can continue to take exjade at levels below 500.

@ Zara

I am confused as well, your son should continue with exjade for lower levels. Levels at 1450 at no point is low enough to stop exjade. Second, why take desferal if exjade is available. There is no added benefit to use desferal over exjade. You should speak to your doctor and tell him that you do not see any advantage of desferal over exjade, and what is the level he is trageting.

I agree completely with Canadian Family. Ferritin of 1450 is not high and is not a reason to switch chelators. Be patient and continue the Exjade, making sure that the dose is raised as the child grows, so that the 30 mg/kg dosage is maintained.

Thanks for ur replies.
My son's serum ferritin reports of last 3 months are:
June: 1650
July: 1100
August: 1450
 In June when Dr saw that ferritin is 1650 he said that it is really high and that means asunra is not working on him bcz we are giving it with food    (they don't believe it works sane with food) and we should immediately start desferal. We were really worried so we repeated the test next month and the result was 1100. We were really happy but again next month it was 1450.
This month Dr said that we should not depend on the reports bcz they don't show the exact result. He is getting regular transfusions and that means his ferritin is increasing day by day. His organs will start to damage and we should get the level below 1000. He told us to start desferal but we didn't agree. So he said increase asunra dose to 500 for 2 months and give it the proper way like on empty stomach in the morning with water or juice and then repeat the test.
From last 1 and a half month I am giving him 400 asunra with juice just one time. I tried to give him 500 a week before but he stared diarrhea. So I stopped it for 2 days and then again started 400.
Plz suggest me what should I do? Me and my husband are really confused and worried. We don't want to give him desferal.
I will be grateful if you do reply in detail.

Zara,

At this early age, it is more important to get the child accustomed to the chelation drug than is the actual ferritin level. This must be accomplished first. If the child doesn't get sick when asunra is taken with food and does have problems when taking on an empty, stomach, then it should be taken with food. It does work and many patients take it right after a meal. Just because a doctor is not familiar with something doesn't mean it doesn't work. The trials using Exjade with food were done at some of the best thal centers on earth, with Novartis overseeing the results. They found no difference in effect of the drug when taken with food. When we consider this in practical terms, the only method that should be used is what allows the child to chelate 100%. With small children, it can take some time to get used to it. As long as liver and kidney values stay within range, there is no reason to stop the drug.

Right now, just focus on finding whichever method of taking asunra agrees with the child. Once the child can take it without getting sick, we can talk about what needs to be done about the ferritin. He probably needs a higher dose, but that can't be done until he is taking the drug without issues. Have some patience. As long as he is taking the drug, he will be removing iron.

Thanks alot ANDY.
I totally agree with you that's why I gave asunra mixed with food for almost 3 months. But our Dr's don't agree and this time he really scared us that he is not chelating well with asunra and his organs will start to damage. We don't know what to do bcz if something happens to my son the Dr's are not responsible for that and they even don't care.
I want to start 500 now but his stomach is not accepting that much dose. I am giving him milk thistle for last 3 months and his ALT has also got down with that. This month his ALT was 68. Thanks for ur advice Andy. May God give you all the happiness in life.

Scare tactics is the oldest trick in the book, your doctor needs to update himself/herself. I agree with Andy when he said "Just because a doctor is not familiar with something doesn't mean it doesn't work."

As long as your child is taking exjade, he is chelating iron. The changes in ferritn levels are normal, infact I would be satisfied to see the ferritn levels increased from July to August. This is an indicator that exjade is working and iron is chelated from organs and is now in blood stream for removal.

I forgot how old is your child and what is his weight, however, split the dose (half morning, half evening) if you could and take it with food.

Thanks Canadian family for ur reply.
My son is 2 years old and his weight is 14.5 kg. I have given him asunra in split dose and mixed with food for 3 months but our Dr's don't believe that the medicine is working that way. So I stopped splitting the dose and now I am giving him 400 asunra with juice on empty stomach in the morning.

Zara, 3 months is not enough to show a decrease in Ferritin as your child just started chelating. Follow Andy's suggestion and you will see a change in 6 months to an year. When my child first started chelating, we didnot see much change in Ferritin for almost a year but then suddenly it started dropping and now we see changes in ferritin with the dose (her ferritin is around 500) and we micromanage the dosage month to month to keep ferritin close to 500.
Also, my child always took exjade with food as she could not tolerate taking it in empty stomach and it did not affect chelation. So, use it with food and try variety of food to see what he likes the most. Taking it is the most important thing so it better taste good so that he will not miss any dosage .