First 9 months of chelation. Results. Also, is Desferal the best?

Hello folks,

It’s been a while but -as you all know only too well- life happens around us even as we're dealing with thal. 

Our wonderful daughter Sophia will turn 4 this September and despite quite a few issues (which we've shared with all of you here- thank you) we've more or less stabilized over the last few months.

She's now transfused every 14 days, keeping her Hb at quite good levels but still having to take 2mg of prednisone daily.
Sophia has been transfusing for two and a half years but we started effectively chelating last September. Approximately 9 months now.
She weighs 15 kg and takes 250mg of desferal every two days.
Desferal seems to be working, inasmuch as we quite obviously see it affect her stool and urine.
Her ferittin however hasn't changed. Sophia started off at a high 4400 -which was to have been expected as she had a very large number of transfusions when dealing with a hemolytic episode at a younger age.
It’s still at 4400.
The medical team- which I'd like to stress is devoted to Sophia and never have given us the impression of taking anything lightly-  feel that this ferittin level is not a huge concern at this stage and have made a very incremental increase in her dosage (effectively we went from 13 to 15 doses per month).
Sophia has had her yearly checkups which haven't shown any issues of concern.

My question is two-fold:
A) Should we be discussing dosages, frequencies etc.? We feel that the medical team's current wait and see approach seems to make sense in light of only 9 months passing but I'd like to hear from people's experience.

B) Alternatives to Desferal: We've gathered from discussions that our medical team prefers desferal to the other alternatives. They feel it is the safer, effective drug. As Sophia deals with the pump splendidly well (we make a big family night event around it) we're not concerned about alternatives because of compliance. We would like to be informed however about efficacy:
We've been reading that dual chelating therapy seems to be more effective. Are this Exjade+Ferriprox or is Desferal one of the two components? Are there age issues?

Sorry to pile so much stuff into one post, the only mitigating factor being is that I suspect that a lot of people have the same questions.

Thanks again to everyone and God Bless you all.

Hi InGreece ,

Wonderful to hear from you and also wonderful to read that life happens. Thal is something to deal with but it is not life and should never be any obstacle to having a life. This attitude has obviously rubbed off on Sophia and we see that in her ready acceptance of chelation. It's something she has to tend to but it's only one thing in her life, which I am sure is full of wonder at her age. I hope all parents can understand how important this distinction is for thals. In fact, there are times I hate using the word thals but we have to use something to indicate that a person has thal in terms of our discussions, but in truth, they are not thals. They are people just like everyone else.

I think the cautious approach to chelation has merit because observation of any side effects as the dosage slowly rises is extremely important. It is far better to be in a position to nip a problem in the bud, rather than try to correct a problem after it has occurred. At Sophia's age there is nothing wrong with taking it slow and making sure that desferal does indeed suit her.

I think as she gets older you may want to investigate other possibilities. One would be to switch to Exjade to make life a bit easier, since desferal will be required 5-6 nights per week as she gets older. The other possibility is adding Ferriprox to the mix with desferal when she is a bit older. There is now an oral Ferriprox solution that is much easier to use and has been shown to cause a much lower rate of stomach discomfort. If this route is chosen, she may be able to take desferal twice weekly and Ferriprox daily, or even no desferal if her iron load remains low. As far as efficacy, no chelator has yet been shown to be as effective as Ferriprox at removing iron from the heart. However, this may change. A bit of anecdotal evidence. Aston Dialo, who is one of my best buddies that I have made through this group, has been on Exjade for awhile. His ferritin is now in the 200's and his T2* results was a magnificent 36! This tells me that Exjade is indeed clearing iron from his heart. I have to mention that he has been very compliant with every chelator he has used, so even though he is in his mid 30's now, he has not had a high iron load to deal with at any point, so this may be a factor in his current status. But it does give us some idea of the efficacy of Exjade.

Each patient is different, so we do need different chelators. In fact, I do expect to see more chelators marketed in the coming years. By the time Sophia reaches adulthood, we will be talking about completely different possibilities with chelation.

Hello Ingreece,

My daughter is 3 yrs old and she also started chelation with desferal last september with ferritin of ~ 1800. We started with 500mg (1 vial) 20 days in a month. However her ferritin was not coming down ...hovered around 1400 to 1600 for the last 8 months. Last month, Dr. Neufeld increased number of days to 5 days/week (i.e. 20 days within 28 days in a month) and dose was increased to 750 mg.

For Sophie, 15 days/month and the dose of desferal seem a bit low as her ferritin reading is ~ 4400. Again, this may be due to patient to patient difference and how each child reacts to the medicine.

Also, are you using VitC with desferal. We have been dilligently giving 100mg of VitC gummy bear to our daughter after starting desferal. Thanks to Andy, we found the gummy bear that my daughter wouldn't miss for a day.

Thank you both for your replies. 

Andy:

Sophia will take part in her first theatrical production this Monday. It's the end of season play for her music playgroup. She has one line -which she nails during rehearsals, and as she's the youngest she's something of a mascot. I saw the rehearsal yesterday with her singing loudly with all the other children.

Life is beautiful.

So we have to chelate. We'll chelate. Thank God we can.

On the dosage situation: Thank you for the information. We suspected as much but it was good to hear that your perspective was similar to our own. We'll wait a few a months and then see how the ferritin is coming along.

 Bostonian_04:

We've seen similar doses to what you're describing given to children around Sophia's age. Seeing younger children given higher dosages at shorter frequencies was one of the reasons I made the original post.

But I think it is about patient differences and the cautious approach Andy suggested our medical team might be taking.

We'll stay on top of it but we'll give it time and try not to be overly concerned until we see how it's going.

You are right in reminding about the vitamin C. We only started doing that systematically last month and, I think, it makes a discernible difference.

Time will tell.

All the best with your daughter who I assume is every bit as precious as our Sophia.

Dads and their daughters.

In the meantime we have a show on Monday 

In Greece,

Best of luck to our little precious Sophia for her performance,your attitude towards life is an inspiration,and i'd like to see more parents with this attitude,on this forum we have so many wonderful parents,but in personal life i know other people who think thal is the end of this world and now life has nothing to offer to their child,i get so disappointed when i meet such people,they destroy their child's life with their own hands.

On the other hand ,your posts filled me with joy,indeed thal is not whole life,its just a part of life please keep visiting and posting,may be your attitude will become a guiding light for any desperate soul out there.

Zaini.

Totally agree
 
manal