Question for Andy...

Did we met by chance at the conference of Asociatia Romana A Persoanelor cu Talasemie Major (ARPTM?) held in Sinai in November 2010?

I wasn't able to go to Sinaia then.....I didn't know that you went, but you met my friends there!

Thanx for answers!

Aha, it is still nice. I am sorry you could not made it. We have learnt a lot. I was with a friend who controls the thalassemia department within our organisation. We were the Dutch delegation. Will you be able to join your group to Turkey? I am not sure of they will come btw.

Your health is overall good?

Say hi from me to all! I've learnt a lot.

Hi Andy,

My son, Jayden, is 4 yrs. Old and he has Beta Thal Major. My husband and I are currently getting him treated at John Hopkins. We have chosen LA children's Hospital for his BMT. Do you think we have made the right choice in choosing that center? I also read that Texas Children's Hospital has done a number of BMTs  on Thal major's as well. I just want my son to go to the hospital that has the most experience.  Thank you for your time.

Lo

LA Childrens has one of the top thal centers in the US. I have complete confidence in them. They should be able to provide you with recent success rates on request. In recent years, the better centers are well over a 90% success rate. Check with both centers to compare the numbers of BMT's done for thals and the rate of success for each hospital. With that said, I would choose LA.

Hi Lo,

Good luck with the BMT, keep us posted.

Zaini.

My son is now 7 and a half years old. He had a ferriscan 3 years ago. The iron level around the organs were considered good at that time. The doctor is not too willing to have another ferriscan for a while. His ferritin level is around 1000. He is taking exjade solely. Never had any other type of chelation before. He started exjade at the  age of 3. Should he be doing more checks?

Annual tests are recommended. At some point, a T2* scan of the heart should also be done, as liver iron levels don't correlate with heart iron levels. Ferritin tests are good for regular monitoring, but organ iron has to be evaluated, also.

http://www.thalassemia.org/updates/R2T2.pdf

How often should these tests be performed?
Ideally, these tests should be performed yearly, as part of a
thalassemia patient’s annual comprehensive care
examination.

Hi Andy...just a quick update on my younger son's reaction to Exjade. The rash completely disappeared in exactly 1 week. He was covered in the rash from head to toe but continued with Exjade and also took Piriton 3 x daily. when it came it spread quickly all over about after 1 week of starting on the medication with slight swelling on his face and lips and was really itchy. And when it started to disappear, it was a bit weird, it first went away from the face then neck then chest, arms working its way down the whole body.
He was taking 1500mg (split dose) but now he takes 2000mg daily.
.......Now he has started wearing braces.... Is it okay to drink the Exjade solution as normal even though it comes into contact with the metal in the mouth or to use straws?

Hi Emby,

The original warnings about metal and Exjade were out of concern over a reaction, but it turned out that the concerns were unwarranted. Don't be concerned at all about the brief contact with the braces. I'm glad to hear that the rash cleared up as expected.

Thank you Andy...he will be happy to bin the straws and just swig it down 

Andy...though my younger son has split dose, having read somewhere here that Exjade does not last in the system for longer than 18 hours (one place stated 16 hours and one, 18) and that the effect of it in the body is higher during the first few hours of taking Exjade, it becomes less effective during the hours after. Also taking split dose would mean true chelation for the full 24(plus) hours. So I was thinking that wouldn't this be putting more pressure on the kidneys especially as Exjade can have more of an impact on kidneys as the split dose is overlapping the 24 hour period?. Maybe the 6 hours of not having Exjade in the system is to give the kidneys a rest because of it's effect on the kidneys.   

Emby,

It has been found that the opposite may be true, because when we talk about the effectiveness of exjade we talk about its effect on free iron.  The kidneys are involved with removing exjade from the system.  Administering lower divided doses decreases the side effects on the body as it is likely more harmful to hit the body with large doses of the drug.  It is much easier on the kidneys to deal with smaller quantities of exjade over time rather than one large dose.  This is true also for the stomach, the liver as well as the kidneys.  The lower likelihood of irregularities in liver function and kidney function provide evidence of this. 

Hello Sharmin...nice to hear from you..hope you are well and especially that special young man you have in your life..hope he is doing well.
I hear what you say but once the body is used to Exjade it shouldn't have major side effects and split dose might still be making the kidneys work harder...and with that we have no long term evidence on impact on kidneys for this. I am asking about this because Desferal can be given full dose and works best given over a period of 12 hours and the full dose for Ferriprox is split 3x daily working best taken at 8 hour intervals covering 24 hours (and not more than 24 hours) and the both of these chelators are hugely successful. I don't think people stick to taking them strictly at these hours so they have a few hours a day the meds are not having the supposed effect in the body yet most still take these meds religously and have been successful managing their iron levels and maintaining healthy organs. The Pharmaceuticals must know this and may have advised taking Exjade as a single dose to work for 18 hours for better balanced management of iron long term and especially with the kidneys as it's working and resting the kidneys for 24 hours without the 24 hours bleeding into the next 24 hours. I'm sorry if I don't make sense.....but I'm just trying to undestand something here  

....maybe good to split then after a while maintain with single dose?..

The once daily dosing for Exjade has far more to do with marketing than science. Once a day sounds good and it is what Novartis felt patients wanted most. Oakland has endorsed the split dose method. The reports we hear about split dose are universally positive.