Discussion Forums > Announcements

Update on the Cairo Conference

(1/4) > >>

Manal:
Dear all

Hope everyone is doing well. As you all know i attended the conference and it was great. There were 32 lectures covering all the issues related to thal and sickle cell aneimia in addition to the last session which was the doctor/parent session.

This is a chance to thank Prof. El Beshlawy (Chairman of ETA) for her huge efforts in organizing this confernce and giving us the chance to get to know all this information. The lectures are supposed to be uploaded on the ETA site later in this year, so i will  tell you as soon as they are there to have a look at them as they are really great. It is difficult to upload them on the site because of the size.

Some points regarding the lecturs:

Cardiac problems in Thalassemia ,Diagnosis, Prevention And Treatment .
John Porter, Professor and Consultant Haematology, University College London

This lecture was given by Dr. P.Telfer (UK) as Dr. Porter couldn't come on the last minute. It was a great one but the most imprtant point that caught my attention in this lecture is it showed the lack of correlation between liver and cardiac iron. In the lecture, many slides showed that many patients have iron in the heart and don't have iron over load in the liver and vice versa. So this means that heart examination (T2*) is important to check iron overload as it can exist without having ironoverload on the liver first.


Iron overload and chelation in Thalassemia intermedia
Ali Taher, MD
American University of Beirut Medical Center
Professor of Medicine
Hematology & Oncology


This lecture was interesting and annoying to me as Dr. Ali Taher said that serum ferritin in thal intermedia is not an accurate indicator of iron overload in patients and only Liver iron  concentration (LIC) is the important one whether through feriscan or liver biopsy.  This is not the case in majors as he showed different studies done in lebanon showing that exactly the same level of iron down by LIC methods done in both thal major and thal intermedia, but the serum ferritin was far less in intermeia than in major.


--- Quote ---The conclusion from both articles stressed the elevated concentrations of liver iron despite slight increase in serum ferritin in thalassemia intermedia.

Accordingly, we have set up a study to determine the extent of iron overload in 80 randomly selected TI patients in Lebanon. Following informed consent and extensive relevant medical history review, LIC using R2 MRI, SF, and other Iron markers were measured. Also Doppler echocardiography was done to detect pulmonary hypertension and left ventricular ejection fraction...

In comparison with data obtained from a randomly selected group of patients with TM treated at the centre, SF levels were seen to be significantly lower, while the mean LIC values were similar in both groups (Figure).   
--- End quote ---


I remember one of th examples in his study showing that a LIC showing 7.4mg in both TI & TM  patients and the serum Ferritin was 500 in the intermedia and above 1200 in thal major. So he is  proving that ferritin is not an accurate indicator  for ironoverload in TI especially who have undergone splenctomy.  His concern was that internal organs could be damaged and the patient is not aware.
His study is including NON transfused patients as he said that ''Iron accumulation in TI patients has been estimated to occur at a rate of 1-3.5 gm/yr and occurs mostly as a result of increased gut iron absorption secondary to chronic anemia. Increased iron burden is partly responsible for the occurrence of complications that occur in this disease such as liver fibrosis, heart failure and pulmonary hypertension; therefore, close and accurate monitoring of total body iron is necessary for ultimate proper chelation therapy.''

This may result in delayed chelation therapy, and expose patients to the morbidity and mortality risks associated with iron overload. Disease-specific management approaches are therefore required in patients with TI, which should include regular assessments of LIC, ideally by non-invasive R2* MRI.

When i asked him concerning my son he siad that an LIC should be done at the age of 10 if he remains non transfused.


 To be continued ......

manal

Kathy11:
Thank-you Manal.
You are one of a kind exceptionally loyal and faithfull to our cause "finding a cure for thal "research and learning.
We all are so blessed, because of your devotion.you make it sound effortless.May you be bless with all good things in life.
I hope you have a nice Mothers' day :flowers :flowers :flowers :flowers :flowers

I'm so glad we cross path :hugfriend :hugfriend :hugfriend :hugfriend :hugfriend

Much love Kathy

Manal:
... the rest of the comments:

Inducers of production of HbF and HbA in beta-thalassemia: what is new
Roberto Gambari
Department of Biochemistry and Molecular Biology, University of Ferrara

Actually this lecture was quiet technical and very scientific, but it mainly talked about 3 main points:
1-  induction of fetal HB through natural products like resveratrol, 5,7-dimethoxycoumarin and bergapten
2-  Induction of HB A through gene therapy and the importance if accompanying it with HB F inducers as their data suggest that a combination of HbF induction and restoration of HbA production following gene therapy could be beneficial.
3-  Functional correction of the beta°39-thalassemia mutation. ''This is a very interesting field of investigation. In beta°39-thalassemia the CAG (Gln) codon is mutated to the UAG stop codon, leading to premature translation termination and to mRNA destabilization. Interestingly, recent papers have described drugs designed and produced for suppressing premature termination, inducing a ribosomal read-through of premature, but not normal termination codons. We have demonstrated that treatment of erythroid precursor cells from beta°39-thalassemia patients with the aminoglycoside geneticin leads to production of adult beta-globin. This approach might be a new strategy to induce HbA in beta°39-thalassemia patients. ''



Later on that day, i had a talk with Professor Gambari and i asked him whether resveratrol increases the fetal HB on all patients or not necessarily. He answered me saying that he examines it on cells in his lab and found that it induces the fetal Hb in 70%.  I told him my findings regarding using it and the change in percentage of fetal Hb of my son before and after it and the duration of it.

I even mentioned that i have many friends on this site who are using it or are willing to use it. He seemed very interested and he gave me his contact number and email and requested that i can  ask the people who use it on this site to work togather with him as he need more information about us.

He wants me to make a list of the patients using it  including my son with all their medical details and we report to him our findings. He said that he really does not have any time to join the site but he is interested in our expierence. He said that we can exchnage benefits. We report to him our findings and he can also set controls since anyway we are using it and since it is an antioxidant any way.  He can help us by sending him our blood samples by DHL for example at his own expense so he can work on our cells directly on his lab to see what he finds what works better on them.

Actually i find this very interesting since some of us are using resveratrol in all cases. So if any one is interested in this PM me all the details of the hB f levels of his case before and after using it and if you are transfusion dependent or not and any other details  as i told Professor Gambari that i will email him back in a day or two. So i will wait for your feedback on this issue.



'Retrodifferentiation; A Process of an Autologous Somatic Cell Reprogramming to a Pluripotent Stem Cell State and its Application in Haematological diseases'
Dr. Ilham Saleh Abuljadayel

In this lecture, Dr. elham dicussed her findings on aplastic anemia patients. After this procedure and all along the follow up blood tests, she recognized that fetal Hb was switched on in an accumaltive trend. This trend stopped at the 6th month after the treatment.  From this she said that it helps in thal major patients as they don't transfuse for 6 months. Though this is not permenat but she counts it as positive thing because transfusing every six month is better than monthly.  The last aplastic anemia patient that has gone through this procedure was in 2004 and he is doing fine till now.

So concerning thal major, it is up to six month with no transfusion and in thal intermedia patients, she has worked in two and they have been free from transfusion for the last two years and she does not know until when it will last. 

Concerning whether the repeated treatment could prolong the time of stablising of Hb , she does not know because untill now she did not repeat the treatment for the same person who has thal.

In the lecture she showed slides of patients before and after treatment  who suffered from differnet skin diseases, diabetic foot, pictures of herself showing eye wrinkles as she has gone through this process personally because she had neutropenia and others issues.

She claims that here are no side effects and that the only people who can not  do this process are the ones that has  protein S or C that cause cogulation.


After the lecture i personaly talked to her and asked her why these stem cells aren't taken from prepheral blood cells instead of the rtodiffernation process of WBCs and whether these stem cells are programed to target the gamma genes in particular or they are just released in the blood circulation?  Anywy she said that she uses the WBCs ecause usually she needs billions of stem cellls and second she commented that the stem cells that is infused have a tremendous positive effect on all the body including growth, osteioporosis, iron overload, cardio,..... but when i asked her about the hb level which is the most important thing to me, she said that it could be maintained between 7 and 8. 

Actually i was disappointed as i thought that the levels could be much higher. Actually there was a mother who has a 4 year non transfused thal intermedia child who already is stablising at 7.5 and she wanted so much to go through this procedure,but now she finds it useless for her, but the doctr told her that it still can help him in his growth or any other complications.  But i don't think that this is actually the only thing we are looking for.

Later next day, in the parent/ doctor session, it was announced that three Egyptian children were has gone through retrodiffernation ( two girls and one boy). One of the girls attended. She is 11 years old, but look much younger. She is thal intermedia but transfusing when her HB drops to 5 or 6. She has done splenctomy, has virus C and osteoperosis. She has done it from exactly 45 days. Now they say that she has increased a little bit in height, don't remember how much exactly, the osteoprosis is much better and the HB is stablising at 7.9

My personal opinion is that this case can really benefit from this procedure as at least she won't transfuse for a while and had some positive effect on growth but this wouldn't be the case for every case. Anyway i have got her contact so i can follow up as they say it is recently done and more positive things could happen later.

Dr. Elham mentioned that she is very much interested in doing this procedure using the stem cells of the mother or the father ( haploidentical transplant ) as this should lead to a complete cure but she will not use a chemotherpy at all and when i asked her about the GVHD, she said that the chemo is the thing the worsen everything because it kills all body,( actually i don't get this part.

She is waiting to try this on any desperate patient who has no other solution except this and is willing to do it, again we are lab rats!!!!!!!!!!!

Anyway, this procedure is done In Jordan and Saudi Arabia with a cost of  $15000 (by the way, last year it was for $ 6000 and she said the more there are cases the lower will be the price!!!!!!)
It take sjust one day at the hospital.


Time for rest

To be continued..........

manal

Manal:
Thank you so much dear Kathy. You know when i go to conferences and understand anything, that is because of this site . The sharing of every member here opens new prespectives and different issues and keep us updated.  I  don't know how life could be or how i can benefit my son if you were not part of my life. Thank you :hugfriend :hugfriend
manal

maha:
Hi Manal
This is really good. Were you jotting down everything or you have it recorded on tape or do you just have a terrific memory. A big thankyou for your efforts and waiting for more :biggrin
Maha

Navigation

[0] Message Index

[#] Next page