Our 3 year old is starting Exjade

Dear friends
Our daughter has Beta Thal Intermedia. She has received 6 transfusions since her first in June 2010. She is growing well and is a happy well functioning girl. She is transfused every 7-8 weeks, pre-transfusion Hb 9-9,5.

It is time to start iron chelation. She is 12 kg, her ferritin approx 700 before last transfusion. We will start carefully with 125 mg Exjade daily. It will be taken extended blood samples this week before she starts, then every 14 days, thereafter every month so on…

Dear friends, do you have advices to share with us?  We are quite nervous about this. She is only 3 years old but very brave. We are excited to see if it works the way it is supposed to, if she will drink it, if she will have side effects. When is it the best time to give it, and how is best way to give it?

Check out the article and comments I posted in the Facebook group about using Exjade. It has been found that taking it with various foods works fine, so don't limit yourself to liquids when starting your child on Exjade. Also, if nausea is a problem, the dose can be split in half and taken twice daily to reduce the nausea.

Thank you Andy! I had trouble reading the file, until I found the pdf. It is really good news that it can be mixed with food, it will probably make it a lot easier.

Hello,

Good luck with Exjade

A few things to keep in mind are,always start with a low dose,not the full dose,and increase the dose slowly and gradually,giving the body time to get used to it,her dose would be and should be according to her body weight,and initially you'll need to do SGPT and creatinine tests every two weeks,until you are satisfied that exjade is not affecting liver or kidney functions,then you can monitor these monthly.

Don't worry,kids mostly do well with exjade,they may take a bit to get adjusted,but usually they do

Zaini .

Thank you Zaini! 

Hi
You are lucky that you have been given exjade as I have been told that I should start on desferal on my 2 1/2 year old Katia in a few months. I am absolutely dreading this as little katia absolutely hates needles and every time we get a transfusion she is stressed out with very distressed and traumatised by the canula. We have been told that here in Australia they do not subsidise Exjade for under 6's unless for special circumstances. I do not know what to do. Do you know that pro's and cons of exjade vs desferal. My little girl is growing really well, she is already 95cm tall and weighs 16kg but I do not want to put here through trauma every night with desferal.
If anyone has any suggestions of what I can do, or say to the doctor I would love to here from you as I have been so upset and stressed by the situation at the moment.

Hi!
Yes are happy that we could start with exjade right away, as we earlier were told that we had to start with Desferal. We startet Sunday (3 days ago), and it seems to be working fine. We mix the tablet with orange juice and give it to her in the morning (on an empty stomach 30 minutes before breakfast).

We think the main reason that we were told to start with Desferal is that it is less expensive than Exjade. We do not accept that. It is such a huge difference for a little child to drink one glass of orange juice in the morning compared to what she has to go through with the pump. We have actually not got the approval for cost coverage yet, but we hope to get it soon

Besides the cost issue we were told that the reason for starting with desferal is that this product has been on the market longer, and therefore considered safer for small children.

I do really understand that you are upset and stressed about the situation.  Maybe you should try to talk to the doctor once more. Hopefully others can give you more suggestions and advices.
Good luck!!

Happy to hear that things are going good with Exjade and Thanks, for the advice. This is exactly what they said to me "about the product being safer for younger children" and the doctor knows that I am not happy about the desferal. I will hopefully get to speak to Dr soon in regards to this because there is no way I can give desferal to her.

Hi Andy,
              My 2 years old daughter just started anusra 300 MG per day last month. Her weight is 12 KG. Her ferrating before starting this medicine was 1314 and now after one month of start of this is 1570 . I do'nt understand such alarming increase in the ferratin level . Also in the urine routine test Epitelial cells 2-3, RBC 5-7 and pus cells (15-17) have been detected . Doctor has asked for urine culture to rule out any urine infection .

I am not sure if this is beacuse of start of this medicine or due to some other reason . Also she is taking 300 MG in one go and we have not yet splitted medicine .

Pls suggest what should be done at this point . I have doubts whether this medince is working or not or doing something wrong by overloading body with increase in ferratin .

regards
MA

Hi MA,

Has the serum creatinine level also been tested? This is most important when using the deferasirox based drug and should be monitored monthly. It is very important in this case because it could be an infection or caused by asunra. If there is no rise in creatinine, it is most likely an infection. If the child is having symptoms of a urinary infection, asunra should be temporarily stopped. Please don't worry about the ferritin level. It is not significant. If any infection is present, even the sniffles, ferritin level will rise as a defense mechanism by the body. This has absolutely nothing to do with iron level in the body. Bacteria and viruses thrive on iron, so when the body has any infection, iron is sequestered in serum ferritin to prevent the infection from feeding off iron stores. This is a common reason for a sudden spike in ferritin levels. Also, the levels are not considered to be an accurate gauge of iron stores in the body, but are more useful for comparing over a period of time. There is also one other very common possibility for the ferritin increase, even if no infection is present, when using deferasirox. We have heard this same thing often from patients and parents after first starting Exjade. Exjade's chelation effects are often not seen right away because it is removing iron stores in tissue and free iron in the blood first. This is most important because the free iron and iron stores in the body are what pose the real risk. Iron stored in ferritin can do no harm, so you actually do want the drug to be attacking the iron that can do harm. It varies on how long before you may see a drop in ferritin, but for a two year old, it shouldn't be too long. Patients with a high iron load sometimes don't see a ferritin drop for a year or more, but the drug is working, which can be more accurately seen by liver and heart scans, but these will come later than this young age.

I would suggest splitting the dose in two and taking half in the morning and half in the evening. Recent research shows that deferasirox can be crushed and mixed in various foods. One thing that is very important with all chelators but even more so with deferasirox is staying well hydrated. Make sure your child gets plenty of fluids, especially water. Do not allow this to be neglected. This drug can have side effects in the kidneys and liver and keeping the kidneys well irrigated is very important to helping prevent side effects.

deferasirox=asunra=exjade=desirox

Andy,
         Thanks andy for Nice detailed response . Well Her S Cretinine value is 0.30 Mg/DL. Her urine culture report comes positive with E Coli infection . In LFT only SGOT is .58 rest all paramters are OK . For urine infections any natural way to treat this ??

Last thing would you advice to wait for Gene therapy to arrive in another 5-6 years or should we go for BMT . We are bit concerned even if we have sibling HLA matched the risk of BMT are high and also at tha same time we are hearining gene therapy is also not succeeded as of now with certain over expressions of gene in certain test so look like it might take years as each thal patient is having different genes and mutations combinations . Pls advice

regards
MA

Hi Andy,
             Waiting for your expert opnion on my last email.

regards
MA

Hi MA,

Cranberry juice is the only natural remedy for urinary infections that I hear about, but one has to guard against overuse because it can cause a secondary issue if used too much. There may be other natural methods of which our members might be aware. Has there been any improvement yet?

I expect it will be some time before gene therapy is readily available and the initial cost will be high. Rigorous testing through trials will have to show no long term health issues that would severely impact quality of life before it will get official approval. A cord blood transplant from a matched sibling would be the ideal method in my opinion. We may see progress in other areas that will make matters less urgent. There is a program starting up in India that will try to provide as many BMT's as inexpensively as possible, through our friends in Italy, led by Dr Lucarelli and Dr Sodani. A recently made contact through Facebook will be keeping me abreast of this development. Their website is now functional and you can see it at http://www.curethalassemia.org/