The Carao Trial - How effective is it & who benefits most etc.

Andy,

What i don't get is that while her hb was normal,why was she feeling so tired and down in the last week,and she was looking so pale that i was assuming that her hb must be low,She was so down that she kept saying "mama something is happening inside me",and this scared me.She couldn't explain what exactly was wrong but i felt like she was feeling shortness of breath.I hope carao will keep helping her and we can increase her transfusion intervals,may be a couple of transfusions can help us make sure about it.

Zaini.

Hi Lloyd,

From where should i order more bottles of carao? My daughter is taking 4 spoons per day and she is on her third bottle.

Zaini.

Zaini,

Paleness and shortness of breath are both signs of hemolysis taking place. Of course, it could be something else unrelated to thal, but a rise in reticulocyte count would be related to hemolysis, also. And this brings up a question for thals. Can you tell when hemolysis is taking place? Is there something inside that you can feel?

Zaini,

What you describe has also occurred with my son on couple of occassions.  He says that he cannot express what is going on, and he also seems to be out of breath.  His doctor however says that his lungs are perfectly clear, his O2 is 100%, and his lung capacity is not only normal but it is above average.  I am also confused by this and it also scared me.  He described this feeling last week when his hg was 10.4.  Maybe as Andy says the kids are experiencing the symptoms of hemolysis - and maybe the feel it more as they get closer to a hemoglobin of 10. 

Have other thals ever noticed this? 

Sharmin

I am so much interested in getting an answer to your question Andy ? Maybe older thal can help us in this. Does hemolysis takes place daily with the same rate???


I know Zaini and Sharmin how frightening this sentence can be, i wish you will never have this situation again. 

manal

Hi,

I am sure it must be hemolysis ,to tell you the truth when ever my hb is low i also feel short ness of breath and a burden on chest.

Andy,

I think you were right about her dosage of ferriprox and desferal as her ferritin came almost similar,few months back it was 1232 and now it's 1259,so i think we need to re check her doses as she has also gained a bit of weight,her weight is 28 kg now,unfortunately i didn't go to hospital for some reasons today,so i have asked my husband to throughly discuss it with the doctor,but what do you say what her dosage should be?.

Manal,

Thanks a lot 

Zaini.

The formula for Ferriprox (L1) is 75 mg/kg daily, taken over 3 doses. 75x28=2100, so 2000 would be the normal dose. The doctor monitoring Zainab should be the one to approve this. If she is tolerating it well and has no drop in white cells, the doctor will probably adjust the dosage as she grows.

Something of interest I learned about at the Apo Pharma booth at the conference was that they have released Ferriprox Oral Solution. It is a liquid form of deferiprone (L1) and has tested for far fewer instances of gastric complaints. It is available in a few countries and is going through the approval process in others. Hopes are for a late 2009 release in Pakistan. This should be much easier for children to take.

Andy,

Once the hg drops to 95 - 90 the body begins to retic.  Blood tests at this level often reveal some reticulocytes.  When the marrow is higher the bone marrow is usually suppressed, I wonder if the children feel this way as their hg is falling to 10 because this is when the marrow begins to get signals to "kick in".   If this is the case, children may actually be aware of a strange sensation in their bodies - perhaps the adult patients would be better at describing it.  I think our children are now old enough to realize something is going on, but not old enough to know how to explain it to us.  Maybe they have always experience this, but they were not as 'aware' as they are now becoming that changes are occurring in their bodies. The breathlessness is the worst symptom, I believe I have felt what you describe too Zaini. 

Andy, is this a possible cause of the strange feeling during the last week before tx?

Sharmin

Well, The ward doctor didn't surprise me by saying that everything is fine and we don't need to change anything, according to him 1259 is the lowest ferritin level he has ever seen in any chronically transfused patient. I am going to talk to her hemo,but to be very honest she is not much help either,i'll be the one to remind her that the L1 dosage is 75 mg/kg and we should chnage the dose according to the weight and quantity of blood,and then she'll just sign it to make it official  ,or may be she'll say the same thing, who knows? some times doctors annoy me so much i wanna yell at them.

Zaini.

Zaini,

What you said here is common. In many countries, doctors don't base their idea of what an acceptable ferritin level is on what is best, but what has been practical for patients in that country to achieve. This has been greatly influenced by the cost of chelation and the problem getting patients to comply. I have commonly heard that doctors tell patients that a ferritin level below 3000 is acceptable. We know that long term this is not true. But it has been the best doctors could hope for in patients so they have accepted it. As chelation has become more available and easier to comply with, doctors need to adjust their thinking. We all know that getting below 1000 is the goal of chelation and that the work involved getting there is well worth it.

Patients and parents all have a basic right to know everything about the medical conditions that face them, regardless of what some doctors or even governments, such as in Singapore, may believe. Many doctors find it annoying and a challenge to their egos when patients know more than they do, and try to deter patients from becoming informed. Don't be deterred. Dr Marwaha was very disturbed that patients were not given access to medical talks in Singapore and he said that the parents know more about thalassemia than the doctors. If only more doctors could be like him. Parents have to be firm and demand the best for their kids. Ferritin readings can be misleading and we know that working to keep it consistently low is very important. Insist that your child can do even better. Maybe you can convince the doctor that this is how all patients should feel.

Andy,

What i don't get is that while her hb was normal,why was she feeling so tired and down in the last week,and she was looking so pale that i was assuming that her hb must be low,

Zaini.

Zaini,

And this brings up a question for thals. Can you tell when hemolysis is taking place? Is there something inside that you can feel?

Zaini,

What you describe has also occurred with my son on couple of occassions.  He says that he cannot express what is going on, and he also seems to be out of breath.  His doctor however says that his lungs are perfectly clear, his O2 is 100%, and his lung capacity is not only normal but it is above average.  I am also confused by this and it also scared me.  He described this feeling last week when his hg was 10.4.  Maybe as Andy says the kids are experiencing the symptoms of hemolysis - and maybe the feel it more as they get closer to a hemoglobin of 10. 

Have other thals ever noticed this? 

Sharmin

Hi Zaini, Andy and Sharmin

I feel this way on several occassions, even though i can't understand why too.. Even in the last transfusion i had, I was feeling extremely tired and out of breath when i climbed stairs and walked about too much.. I thought my hb must be something like 7 now... (it was nearly 3 and half weeks by then since i transfused..)... I even stayed in bed one whole day, just dint have the energy to get up...

But when i went to the haemotologist and got hb checked, it was almost 10. (9.8 to be exact). Dr. Alan Teh saw how pale and tired i was, but both of us were surprised by the results... he gave transfusion anyway... He trusted my instincts when i said i was feeling that way...

this is not the first time it happened to me.. and i donno how to explain it... sometimes i ask if its in my mind? to imagine that since its near to my transfusion my hb must be this low 

but then, how would zainab and little A imagine that, right? so i guess we do know when we are experiencing hemolyis...

any other thals? whats your experience?

Maako,

thank you for that explanation, which our little ones are not able to provide for us.  Maybe it is infact the feeling you get when the bone marrow is beginning to become active. 

Whether a non thal individual has a hg 110 or 100 it doesn't matter because the marrow is always active.  In a transfused thal major the hg is suppressed when it is 115 (lets say) but perhaps it gets messages to become active closer to a hg of 10 - and that is when thals can feel the hemolysis.  I am guessing that this is a possiblitity.  Maybe also near the end of the transfusion the red cells are getting old (because thals don't replace them on their own) and their oxygen carrying capacity is reduced somewhat... 

It is difficult to understand, but I know that some doctors here recommend having smaller tx every two weeks rather than a normal tx every 4 weeks.  They say that it is optimal for growth, I wonder if this is the reason.   

I understand that red blood cells are replaced in the human body about every 28 days, then they are broken down and new red cells are made. 

Does this make sense to anyone else?  It makes me wonder if the small tx every 2 weeks is in fact best - BUT it can be a big lifestyle change for many. 

Sharmin

.. and i donno how to explain it... sometimes i ask if its in my mind? to imagine that since its near to my transfusion my hb must be this low  

Maako,I think that sometimes this could be right. Before knowing that i am myself  has anemia, i never complained from anything but after my knowledge i just some times feel tired from things i used to do without any problem and i refer them to the phrase '' because i have low Hb''

For this i am would want to raise my child knowing that he is just different NOT diseased. I think that uncoinciously (spelling....) when you know that you are diseased even if you are leading a positive life, you tend to put limitations to your abilities. I don't know if i am right in this thinking or not or even it is my still hidden thoughts of in accepting but all what i am sure of is wishing to give my child the very normal life

But again young children wouldn't realize this as you questioned Maako,so there could be definetly symptoms for hymolysis???

manal

Thanks Andy,

For your support,actually the ward doctors meet a lot of thals,and sadly most of them have very high levels of iron,at least that's the situation here in my country,sometimes i wonder if i should start a campaign to convince patients to comply with the chelation and bring down their ferritin levels? At least in the hospital where we are going?  but the thing is every body is not able to afford every chelator,so they try to use what ever they can get.
What i don't get is why doctors give them a sign that it's ok to have iron overload while you are chronically transfused,i,ve seen patients with such high iron levels that it horrifies me,most parents doesn't have the basic knowledge,and unfortunately they accept that their kid is going to die anyway,so why try? This is pathetic,i've seen well off rich people with the same approach,A few months back i saw a little girl,younger then my daughter in the hospital,i don't know what her ferritin level was at that time,but when after a few months i saw her again,she was com[pletely changed,with the darker skin,and the tell tale signs of thal major,that means that her parents weren't complying with the chelation,since she was very young to be compliant by herself,and i know that she belongs to a well off family.Often when i meet moms,educated moms in the hospital,i literally force them to join this forum,but sadly i haven't seen a single one of them here,well the least i can do is keep reminding them,
I'll ask my daughter's hemo about the dosage,if she doesn't approve it,i am gonna change it anyway.

Zaini.

Here is the update on the Carao Use and results for my niece

1. Confirmation of delivery of the carao – Yes
2. Date when carao is begun – 8th July 2008
3. Dose given – 2 Teaspoon per day
4. Age and diagnosis of patient – 3 and half years (Thal Major)
5. Average hb before carao was begun (the last several blood test results, if available) – Between 9 and 10
6. Results of all hb tests done during or soon after the period of use of carao. Of course, the date of the test(s) must be included – 9.4 (18th Oct 2008)
7. Whether or not any other treatments were started during the period that carao was given - No
8. Any extra observations, such as observations on energy level and effects on the overall health of the patient – Nothing visible