Transfusion Independence

This seems to be another article about transfusion requirement going DOWN by chelation of iron.

IronJustice, be careful about what you say. Firstly, I believe you must change the title because it makes us all way too happy
Secondly, do realize that some people need more blood during their way to get deironed! These are side effects for the medications. And since most journeys to deironing take several years, you will notice that your transfusion scheme can be with longer intervals.

Although these articles are very ineresting, I do not believe in a cure for thal nor for pk def. yet. Remember that scd, but especially mds and AA are very different diseases that ours.

I know it smells like bake frogs, but that's how it is. There is indeed more happening in thal world than in mine. That's absolutely a fact, and for thal a very good one!

Dori

First, I just want to reply to Dore. I do believe we will see gene therapy cures for Thalassemia, Sickle Cell disease and Pyruvate kinase deficiency. I say this because each one of these has been cured in animal trials and it is only a matter of time before all of these have been trialed in humans. I realize that cures available to the public are still years away but they are coming. I also think that many of the advances in treatment may make a cure less meaningful, as the disorders will become easier to manage as new developments are introduced.

I also want to get back to the discussion about transfusion requirements at low ferritin levels. Lena has not seen any difference. Umair said he has had a longer gap between transfusions. The reason I brought this up, in addition to the material presented here by ironjustice, is that I have indeed heard that in  infants who have never been allowed to have any iron load, that less destruction of red blood cells has been observed. There is some thought that by chelating as soon as transfusions begin, that this may work towards lower blood requirements.

I realize that any evidence presented here by patients is anecdotal, but I would like to hear from more patients. Has anyone else noticed a difference in blood requirements once their iron load has dropped to the close-to-normal range? I don't think we'll hear from anyone regarding infants, as this is very new and I don't know if this is actually being tried yet, since most doctors prefer to wait until the child is older before introducing chelation drugs. It may be that the earlier chelation begins, the better off the patient will be in ways that we previously did not consider.

Quote: is that I have indeed heard that in  infants who have never been allowed to have any iron load, that less destruction of red blood cells has been observed.

THAT is precisely what I am talking about. 
I didn't know that someone somewhere had actually shown evidence of that.
I've held that it is iron loading from BIRTH and the treatment 'should be' phlebotomy AT birth and a low iron intake lifetime.

At the very least, this has doctors and researchers questioning when chelation should start. More and more, we are seeing a trend towards earlier chelation and attempts to minimize iron loading right from the first transfusion. Can we get some research going in the area of developing early use chelators based on natural antioxidant chelators like IP6 and green tea extract? Concentrated forms of these and other natural substances may help to prevent that early iron buildup that occurs before chelation drugs are deemed safe for the age of the infant.

Quote: Deemed safe for the infant.
Answer: THAT is what is happening right now in the NIH Diabetes Iron Reduction Trial.
It seems the doctors are STOPPING iron removal at a **predetermined point** .
Previous studies show by removing iron DOWN to BELOW what they 'think' is safe leads to improved recovery.
It seems they have designed the study to fail IF one takes into account previous studies.
In Jehovah Witnesses they do not recommend transfusion until the hemoglobin is 5.5.
IF that type of anemia doesn't kill the Witnesses one might not think it will kill anyone else.

Hmmmm, I thought that JW never want to have blood transfusions. So this sound odd to me. The level of 5.5 makes me smile because thats the level I usually get transfused but then we talk in mmol/l

The other thing is not related to this topic. It is just that I dont dare to guess anymore what my hgb can be. I lost any feeling in refer to that. I feel pathetic. No feeling at all. The same for my ferritin.

I absolutely believe that chelation should be start the minute newborns get transfused.

5.5 gl/dl is 3.4 mmol/l. It might nog kill you immediately, but in the long term it is not good. Ironjustice, how do you feel by a hgb of 3.4? I would feel like a fish outside water! In the long term you will developed complications like skeletical changes and poor bones. (I would be very very cold and depressed too!)

Quote: Can we get some research going in the area of developing early use chelators based on natural antioxidant chelators like IP6 and green tea extract?

Answer: One might think this would be EASILY tested in anyone with a known iron overload ? IF one were to get some luminol the same luminol they use in CSI ?
One could buy get the substance / food / chelator to be tested and do a urine test on yourself and see if iron begins to show in your urine ?
"Luminol will also detect the small amounts of blood present in urine"
http://en.wikipedia.org/wiki/Luminol

Explanation - translation, please
 

One figures out exactly what amount of luminol is required to illuminate the iron in your urine. THEN you get 'whatever' you are testing as an iron chelator and you eat it. You THEN test your urine FOR 'extra' iron that in theory you SHOULD be urinating out.
To put it plain and simple. 

I think I miss something. What is the logical of that?  What the use of knowing?

Yet deferiprone is still not approved by the FDA. Of course, Apo Pharma is nowhere the size of its giant competitor, Novartis, so one quickly understands how this can be. The FDA is a joke and is getting worse every year.

The availability of L1 would be of no consequence IF one can find out WHY maltol isn't being promoted as an alternative to L1 ?
L1 / deferiprone is simply a 'ripoff' of the sugar maltol ?
Maltol is used as a template to MAKE deferiprone but I cannot find ANYTHING about the iron REMOVAL 'properties' OF maltol.
The best I've really been able to find is that maltol is SAFER than the iron chelators used now and it is used in a bubble gum to reduce smoking.
IF one WERE to find the studies which SHOW the rate of removal of iron by maltol then one can move from there since a person can get it for almost nothing out of China.
I have been down to the diabetic bakery here and they have no clue about maltol being used in foods and so I've been stymied there.
I think maltol could be used in a caramel with vegetable lecithin to make a VERY functional food for those with iron excess for sure but for a slew of other problems.
"In a structure-function investigation by Hider and co-workers , in which the yardstick of choice was radical scavenging ability, deferiprone was completely ineffective, maltol was somewhat effective, and 3,2-HOPO was the best of the representative hydroxypyrones and hydroxypyridinones examined. Desferrioxamine was by far the most effective in this regard."

I understand it a bit more.
I think it must be more than 10 years ago since I started taking deferiprone for the first time. Then we or the pharmacy bought it from a buidling contrictive market and the pharmacy made pills of it. And it was much, much cheaper then it is today.

Quote: And it was much, much cheaper then it is today
Answer: Which PROVES what we have all suspected all along. The drug makers are NOT 'in it' for US they are in it for the cash. It is evidenced in the drug thalidomide that drug that made kids to be born with flippers. It is NOW worth $4000 a month for cancer patients in Canada and the US because they 'feel like' charging that much but one can get it in Mexico and India for $12 dollars.
THAT is the why I think I cannot find the studies about maltol and how it would be effective and the same with phytic acid. Safe natural substances which won't even be looked at by the FDA for some reason. Phytic acid they refused to even consider it. Refused ? I think maltol should be pushed to the forefront due to its already PROVEN ability to remove iron IE: template FOR other iron chelators and shown to be LESS dangerous than the synthetic chelators and therefore EVEN more DESIRED by anyone with a brain. IF a doctor is shown a substance is as effective AND safer and REFUSES to use it then he shown be drawn and quartered .. imho.