In Need Of Your Advise Andy

Dear Andy,

Hope you are keeping young, well, fit and bouncy  !!
Andy i need to bug you about something. My young son had his T2 scan which revealed heart iron 17.8 (mild iron) liver 4.8 (mild iron).  He was taking 3 1/2 vials of desferal (500 mg) 5 nights a week.  His Doc has told him to discontinue with desferal and to take ferriprox 3 tabs 3x daily. He weighs 59.2kg.  Is this okay? or do you think he could do better on combination of the two?  His ferritin is 517.
 
Also the Doc has told us that from now the boys will be having transfusion worked out by desired hb of 15 (Hb 15 - actual Hb x body weight x 3).  Our older sons Hb has been below 8.9 pre transfusion so i feel it might be okay for him.  But the little ones Hb has only dropped the last 2 times pre transfusion.  Do you think this is a good formula for BOTH of them ?  Do you think that it be okay to go with this formula short term because I'm worried that too much blood annually is not good regarding iron levels although theirs is low? 
Also is it safe to be transfused regularly with 5 units of blood averaging 250mls?

One last thing...our younger one has a slightly enlarged spleen...what does this mean?  Would it be a bad thing to have increased blood transfusion? Can the size of the spleen be reduced?  How could the size of the spleen be maintained without it getting bigger?  He has been well but I'm worried about it.

Please, please, please help.  I know that I have asked too many questions but i would really, really appreciate your help and valuable advise.

Take care and Big Hug
Emby       

Hello Emby,

Andy might not be able to answer right away as he is in NY for a conference on Thalassemia. Such a good thing for all.

My young son had his T2 scan which revealed heart iron 17.8 (mild iron) liver 4.8 (mild iron).  He was taking 3 1/2 vials of desferal (500 mg) 5 nights a week.  His Doc has told him to discontinue with desferal and to take ferriprox 3 tabs 3x daily. He weighs 59.2kg.  Is this okay? or do you think he could do better on combination of the two?  His ferritin is 517

I think as Ferriprox is well known to remove Iron from the heart, it is a good option to start with it. For the dose, I think it is best to start with a lower dose for the body to get adjusted to the new medicine. The optimal dose is 75 mg/kg, so depending on the body weight the child should take the medicine, but I have heard to start with a lower dose and gradually increase it. As his ferritin is not high, i think it would be ok to discontinue desferal for now. That also gives him a break from dialy needles.

Hello Narendra,

Andy has gone to drink from the 'Fountain Of Thalasseamic Knowledge' ? He is the best.  Hope he brings back some fantastic news  !

Thank you for getting back so soon.  Our younger son has been put on the lower dose and I'm praying he stays well with it. He will have to go for weekly blood checks which might be worse than having desferal all weekdays because his veins just never want to bleed back   . 

Anticipating Andys return.
Emby
 

Hi Emby,

It really is a marvelous conference and an incredible group of doctors and researchers who have been gathered here by the Cooley's Anemia Foundation (a big pat on the back to everyone from CAF).

Narendra has steered you right. Ferriprox is far more effective at removing heart iron so this is the correct move. You sons should have a T2* over 20 and with work, over 30, so there is some work to be done. I agree about starting on Ferriprox because gastrointestinal complaints are the most common side effect, and starting with a low dose and working up to the required dose can help to minimize this. If possible, see if the oral Ferriprox solution is available in the UK and get that because the incidence of gastro problems has been greatly reduced in the liquid version of Ferriprox. If I run into Gerry from Apo Pharma again tomorrow, I will find out if the UK is one of the places the liquid is on the market.

I want to include a comment from one of the top doctors in the world in regards to Ferriprox. At the end of his presentation which showed once again that Ferriprox is the superior chelator for removing heart iron, the doctor said that he just cannot understand why this drug is not being made available to patients in every country. We're getting closer to approval in the US but once again, the FDA is taking their sweet time with the process.

I'm not sure I understand the question about blood. Are you saying 5 bags of 250 ml at once? I do believe that a higher Hb may be of some help with the enlarged spleen, but don't worry too much because slightly enlarged is not a big concern.

What about T2 iron liver? Isn't this (4.8) too low? In the liver iron must be above 19 (T2*) and in heart above 22 (T2*). So Ferriprox is o.k but together with desferal or exjade that remove iron from the liver, because ferriprox has little effect on liver.(If I understood correctly 4.8 is T2* for  liver iron.)


Lena.

The liver iron measurement is normally an R2* which is an inverse, so the lower the score, the better. 7 is considered the upper optimal limit. 15 is an indicator that there is a high danger of iron overload in that patient. Informational note: Heart iron was also once expressed as an R2 score but is now more commonly measured as T2*. R2* is the inverse of T2*. R2* (1/T2*). So, a low liver iron score is the target, while a high heart T2* is the target. (This was just discussed at the conference today). I cannot vouch that every country is using the same system of measurement.

 That's why I was so perplexed and didn't know if I had to post--I did finally. We measure T2* here, but two years ago we started measuring R2* only for liver---btw mine is 1,0 and I keep on chelating with ferriprox and desferal both.

Lena.

Lena,

You are such an inspiration for all thals and to be honest, to me too. 

Hiya Andy,

In my minds eye I feel you have gone on a 'Great Adventure' to some far away land equiped with your shiny armour, with a gleaming crested sword and sheild.....oops...should really be equiped with a note pad and pencil....or would a laptop/netbook be more accurate!....Anyway, in my minds eye, we are the ordinary folk waiting back home for some fantastic lotions and potions and all manner of cures... !!
But really Andy, I thank you for taking time out again and thinking of others (you give advise to loads of other people with all sorts of conditions, not just us thalasseamics) and I know that what you bring back will be extremely valuable to all.

Ferriprox is available in syrup form in the UK, however I am a bit reluctant to give the boys this because of one young boys bad experience with it....maybe in the future.  I keep thinking tablets are better because they release over a longer period in the body and liquids might not be effective enough.....

About the blood.....I do mean 5 bags (averaging 250mls) all at once.  is that a bad thing? And only when you have the time to, can you please give me imformation about the spleen.

Keep well Andy, Big Hug
Emby       

Hello Lena,

I completely agree with you about combination therapy. I think Ferriprox & Desferal work together brilliantly in reducing excessive iron in the heart and liver. I feel alot of people on here have benefited from it as well as our son.  All medication have side affects and some medications are more suitable for some than for others. Personally, I prefer this combination and our boys are okay with it too. Presently, with there low levels of ferritin they are enjoying having a complete break from the Desferal.
I can see from your photo that you are looking very well and healthy.  You seem to have a natural beautiful glow about you. I pray that you always keep well.

Take care.
Emby     

Hi Emby,

The liquid Ferriprox does not taste good even though it is cherry flavored, so that can be a factor in willingness to comply. It is equally effective and has a lower rate of stomach discomfort but with kids, the taste can affect compliance, so as long as they have a problem with it and the pills don't bother their stomach, I agree with that it's best to use the tabs for now. Believe me, Apo Pharma is well aware of the taste issue and it was even compared to taking a medicine like NyQuil 3 times a day every day, so I can understand why it may not be a pleasant experience.

As far as transfusing 5 units at once, it seems like a large volume that could cause some stress to the heart (it's a high volume of fluid to add in one sitting), but I would prefer if some of our veteran thals can advise us on this. I've never heard any thal tell me that they got more than 3 units at one sitting. Usually, if there is a major correction needed in Hb, it will be done with weekly transfusions until the desired level is reached. Can we get some input from experienced members?

I believe the idea with the spleen is that if you can keep the Hb higher, this shuts off most production of the inferior thal red blood cells in the body, so they are no longer being filtered out by the spleen, causing enlargement, but the transfused cells are also removed by the spleen, but the hope is that there will be a net benefit and a reduction in spleen size. One important note, is that a slightly enlarged spleen isn't really any danger.

Hi Andy,

Our boys are both having upto 4 units of blood. On average they would need 1000mls every four weeks.  I am thinking that this is the most they could have regularly and less stressful for the heart.  However on occasions they may require  the 5th unit but not the full amount and it would be in one sitting.  they usually run the transfusion at 200mls per hour. Do you think this is safe?

Looking forward to hearing about what you have learnt at the conferance.  I believe that with every sickness God has made a cure for it.  Untill He Wills to reveal it we have a duty to care for our bodies inside and out. Like you say, care for thalasseamia has come a long way and God willing it would only get better.

Take care, Big Hug
Emby   

Andy and Emby,

thank you so much for your kind words. Sorry my reply comes late-I was away from Athens and just returned.
 
I would like to comment on the transfusion frequency.

The only time I got 3 bags of blood was 30 years ago when I was in Cardiff, Wales, for some tests and that was when I first got my pump there from the University Hospital of Wales in Cardiff. They had me transfused overnight then with 3 bags of blood and that took so many hours. Is it a habit in England to be transfused that way? I do not know.

Since then I never repeated that and to tell you the truth, as I see it now, it was a bit dangerous as they transfused me with hb 7 (due to some tests they let me without transfusion for a long period) and heart would have suffered a little receiving 3 blood bags at one stroke.
 
It is many years now that I have adopted the "one-bloodbag-every-15-days" scheme. I think this is much too good for the heart and everything, as you manage to keep your hb at approximately the same level and always get fresh blood. I even disagree with the two-bloodbags-every-20- days scheme, which is widely done here in Greece, but only due to thals not wanting to be to the hospital so often. For me, my health comes first and do what I consider best. That's why I adopted this scheme, although I have to travel for one hour to get to the hospital which is outside Athens-in another city (Korinthos).

There is one other thing I want to stress out. Every blood bag I get is around 300-400grams and the transfusion takes almost 3 hours to be completed.

Emby, you are talking about 5 bags amounting to 250mls(how many grams is this?), or is it 250mls each one of them? Because this makes a lot of difference. If it is 5X250mls, I will agree with Andy it is a very large volume of blood for one sitting. Why have the doctors adopted that scheme?

Lena.

Emby,

Refer to the link : http://www.thalassemia.com/documents/thalhandbook2008.final.pdf 
I have copied over the transfusion section below for ease. This is published by Children's Hospital Oakland

If possible, print it out and keep the complete handbook with you for reference. It has complete details for care of thalssemics.

4.3.3 Target hemoglobin and frequency of transfusions
The amount of blood received on transfusion day is determined by pre-transfusion hemoglobin levels. The target is to maintain the pre-transfusion hemoglobin level between 9 and 10 g/dL. Attempts to maintain pretransfusion hemoglobin at above 10 g/dL increase transfusion requirements and the rate of iron loading. Transfusions should be given in an outpatient setting with an experienced transfusion team that uses proper safety precautions (patient/blood identification bracelets). Blood should be transfused at 5 mL/kg per hour, and the post-transfusion hemoglobin should not exceed 14 g/dL.

In patients with severe anemia (hemoglobin less than 5  g/dL) or cardiac compromise, the rate of transfusion should be reduced to 2 mL/kg per hour to avoid fluid overload. Diuretics such as furosemide (1 to 2 mg/kg) may be necessary for some patients. If cardiac insufficiency is present, higher pre-transfusion hemoglobin levels (10 to 12 g/dL) should be maintained with smaller volume transfusions given every one to two weeks.

The patient’s weight and pre-transfusion hemoglobin and the volume of transfusion should be recorded at each visit. These values should be periodically reviewed to assess the volume of blood required to maintain the desired pre-transfusion hemoglobin level. Annual blood transfusion requirement in patients without hypersplenism is usually below 200 mL packed red blood cells/kg per year.

Hi Lena....thanks Narendra,

In one of my post I wrote about our older sons Hb dropping to 8.0 and maintaining at that sort of level pre transfusion.  We were told by his Dr. that because of growth spurts he would probably need to come for transfusion a week early for a few months and then go back to once a month after his pre transfusion Hb maintains at 9.5.  He doesn't want to come to the hospital so often so the Dr. has raised his desired Hb to 15 after transfusion.  This works out with him receiving about 200mls more each time.  We do not want him to stay with this formula all the time.  We would like to look at what levels his Hb stays at pre transfusion and then God Willing, come back Hb 14 formula.

Both our boys need about 900/1000mls of blood every 4 weeks and they have it in one sitting for a period of 5-6 hours.

Emby