Notes from the Egyptian and Syrian conferences

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Offline Manal

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Notes from the Egyptian and Syrian conferences
« on: May 18, 2009, 12:58:34 AM »
Find below some notes taken from both conferences,

*In the lecture under the name ''Gene therapy: is it a reality??'' by Dr. Farid Boulad (working with Dr. Sadelain team) medical director of the pediatric day hospital at Memorial Sloan-Kettering Cancer Center. As a clinician, Boulad will be involved in treating patients. Though the lecture was so technical but at the end he was so optimistic by the future trail and his answer was ''Yes'' it is a reality.

One of the biggest problems they are facing to start the trails is the unavailability of volenteers. In the USA the population is around 300 million and thal patients are around 1000 only. He said that they have one now from the USA and they are bringing two more patients from Sicilia and will arrive the USA on June. He said that the trail is open for patients above 15 years old.

* Professor El Beshlawy gave a really perfect lecture about the usage of L-carnitine in reversing the PHT. It was supported by a clinical study.

She also gave a comprehensive lecture suming up all the HB inducers (natural or chemical) . I will try to get these two lectures and upload them on the site here for reference

* Many studies showed that after usage of hydroxurea, the caridac functions have improved and also the PHT has decreased. Dr, Wood commented that this findings have caught his attention and furthur studies should be made to test  the effect hydroxurea from the cardiology point of view rather than its effect on HB

* A debate went on between hematologist and cardiologist (Dr. Wood/ Dr. Ali Taher/Dr. Aessopos) concerning the diagnosis of PHT. Ususally when the echo showed a increase in the Pulmonary preassure, the patient is diagnoised with having PHT but Dr. Wood commented that he PHT has to be diagnoised by using the Tricuspid regurge jet velocity (TRJV) togather with the Pulmonary Pressure and not only the Pulmonary pressure only because it can be increased in a patient with a normal TRJV and in this case he is not diagnoised as a PHT patient.

*Non-transferrin-bound iron (NTBI) is for sure an indication for iron toxicity, but not a good marker for iron overload

* In the lecture of  Dr. Farroukh Shah, she said that there is a study (still on animals) that showed that the splitting of the exjade dose twice a day has a great improvement on the cardio and liver profiles better than taking the dose for once. But this is off course not recommended yet

*Thal patients who have Hepatits B virus will have the same treatment as those who are non thals while thal patients who have hepatitis C virus and using Ribavirin, shouldn't use it at all with a Hb under 8.5 and therefore adjustment in trasfusion regimes has to be done

*Studies of the usage of hydroxyurea in the Iranian populatons showed that it has a really good effect in eliminating transfusion in thal major patients ,it was up to 50% of the patients enrolled in the study. But comments that this has to do with the kind of mutations of this particular populations and shouldn't be generalised

* Also the Iranian doctors had a really good study about the side effects of using the hydroxyurea. Their study showed that the best dose given is 10mg/kg and the safest dose would be a maxiumum of 20mg/kilo. Their studies showed that after 10 years some dermatologic and neurologic side effects has appeared and only one patient has got Lukemia,butit was related to other factord too and can not be proven to be related to hydroxyurea.

This study was debated by Dr. El Beshlawy cause from her expierence, egyptian patients start responding to the hydroxurea from the dose of 20 to 25 mg/kg and sometimes more dose is given and proven to be safe overthe years, So i guess that this is too related to kind of mutations or may e other factors inthe body

* Unreliable readings of ferritin in Thal intermedia were also discussed and how same ferritin has a different LIC in thal major and thal intermedia patients. I was able to copy the readings and you will find a great difference in the LIC range

Ferritin                         LIC mgFe/dry weight TM         LIC mgFe/dry weight TI
500                                       2.7                                 5.8
1000                                     5.4                                    11.6
1500                                       8.2                                 17.4
2000                                       10.9                                 23.2

These numbers really shows that we never depend on serum ferritin in TI for detecting iron overload

Also increased LIC is correlated with PHT in thal intermedia patients


* As a parent i was always questioning the infertility associated with theuse of hydrea, Dr, Porter has answered me saying that in a study doen in rates, it showed that infertility is an outcome of this usage but in reality and with patients who have Sickle cell disease, it was found that their sperm counts were already low before trying the hydroxurea in the first place and some times is is more lowered by the usage of hydrea. But actually i got aquainted to a lot of SCD patients who are parents and have been on hydrea all their life.

*The usage of EPO with hydrea is sometimes effective ( though it is not effective cost wise) but sometimes EPO has a risk of causing extra medullary hematopoesiss and therefore lead to an increase in iron absorption

*I got to know that COLIA 1 gene is the marker for Osteoprosis

* i got to know too tht there is a swiss chelator called Ironix (brand name that could be a generic),some doctors  used it for clinical trails and it was said to have a real good effects, but actually i can not find anything about it online

* I always wondered why each country has to do a study comparing all the chelators, i thought that that this is a waste of time since one or two studies are enough but one of the doctors told me  that different populations have different reactions because of the nature of mutations and they found for example that the majority of egyptian enrolled in studying the effect of L1/Kelfer are in most cases suffering from severe drop in the white blood cells ( can not recall the scientifc name) so it is somehow to know how the same medicne affectdifferent ethnic groups

I hope i will be able to recall more notes

manal

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Offline Sharmin

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Re: Notes from the Egyptian and Syrian conferences
« Reply #1 on: May 18, 2009, 04:49:53 AM »
Manal,

Thank you so much for all of this information it is incredible.  I am very interested to see how all of this pans out in the future.  I also hope that you see many benefits with hydrea for Ahmad.  I can see that his hg has increased from the 60s to the 70s.  I hope that over time you will see a further increase. 

Sharmin
Sharmin

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Offline Manal

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Re: Notes from the Egyptian and Syrian conferences
« Reply #2 on: May 18, 2009, 09:01:36 AM »
I too hope the same Sharmin :hugfriend, anyway you are welcomed dear

manal

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Offline Zaini

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Re: Notes from the Egyptian and Syrian conferences
« Reply #3 on: May 18, 2009, 11:23:58 AM »
Manal,

Splitting the dose of exjade,i think some one on our forum has done it,i can't recall the name right now.

Zaini.
^*^Xaini^*^

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Offline Manal

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Re: Notes from the Egyptian and Syrian conferences
« Reply #4 on: May 18, 2009, 12:52:55 PM »
Zaini, but this is a trail on animals and not yet recommended either by doctors or Novartis.

manal

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Offline maha

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Re: Notes from the Egyptian and Syrian conferences
« Reply #5 on: May 18, 2009, 01:18:42 PM »
Hi Manal
When little Miss A was having continuos nausea and vomitting Sharmin had suggested splitting the dose. I think she had got this info from oakland.

maha

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Offline Sharmin

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Re: Notes from the Egyptian and Syrian conferences
« Reply #6 on: May 18, 2009, 05:24:30 PM »
You are right Maha,
Oakland had suggested this to me when I was in Oakland - in case lil A had nausea - so I passed the information on to lil Miss A.  I think that they are pioneers in Oakland and they already have experience with splitting the dose out there. 

Sharmin
Sharmin

Re: Notes from the Egyptian and Syrian conferences
« Reply #7 on: May 18, 2009, 09:03:45 PM »
Hi Maha and Sharmin,

Little Miss A started the dose in the evening rather than in the morning to help with her nausea and vomiting. I vaguely recall the talk of splitting the dose but it was never implemented.

Not sure if other patients are doing it in HSC, Toronto.
Regards.

Re: Notes from the Egyptian and Syrian conferences
« Reply #8 on: May 19, 2009, 01:11:07 AM »
thanx for ALLLL these infos. u r an encyclopedia!!!!!!
but did dr farruk shah attended the conference in egypt? if so, i am very sad that i was ill and did not attend they confrence :wah

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Offline Manal

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Re: Notes from the Egyptian and Syrian conferences
« Reply #9 on: May 19, 2009, 09:49:53 AM »
I am sorry Mohamed to know that you were ill, i hope you feel better now. :getwell :getwell

 As for Dr. Shah,she attended and gave very good lectures. I hope i can have access to the conference lectures so we can upload them on the site here

manal

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Offline nice friend

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Re: Notes from the Egyptian and Syrian conferences
« Reply #10 on: May 19, 2009, 12:58:51 PM »
 :getwell Mohammad , i hope u r doing fine now ...

Best Regards
Umair
Sometimes , God breaks our spirit to save our soul.
Sometimes , He breaks our heart to make us whole.
Sometimes , He sends us pain so we can be stronger.
Sometimes , He sends us failure so we can be humble.
Sometimes , He sends us illness so we can take better care of our selves.
Sometimes , He takes everything away from us so we can learn the value of everything we have.

===========
Umair

Re: Notes from the Egyptian and Syrian conferences
« Reply #11 on: May 23, 2009, 03:05:08 AM »
thanx alot friend :love :love :love :thankyou2

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Offline nice friend

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Re: Notes from the Egyptian and Syrian conferences
« Reply #12 on: May 23, 2009, 08:22:57 PM »
Your Welcome  :biggrin
Sometimes , God breaks our spirit to save our soul.
Sometimes , He breaks our heart to make us whole.
Sometimes , He sends us pain so we can be stronger.
Sometimes , He sends us failure so we can be humble.
Sometimes , He sends us illness so we can take better care of our selves.
Sometimes , He takes everything away from us so we can learn the value of everything we have.

===========
Umair

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Offline Manal

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Re: Notes from the Egyptian and Syrian conferences
« Reply #13 on: October 01, 2009, 11:24:28 AM »
I found on the TIF site some of the lectures that i attended in Syria. Many are very informative especially the one of Dr. El Beshlawy that is suming up all the fetal HB inducers (natural and non natural) with a stress of the usage of hydroxyurea on thal major and intermedia

http://www.thalassaemia.org.cy/pdf/syria/day%202/Day%202%208.pdf

Also you will find a lecture about gene therapy by Dr. Boulad ( participating in Dr. Michel Sadelain Thalagen)
http://www.thalassaemia.org.cy/pdf/syria/day%202/day%202%209.pdf

You can not miss exjade update by Dr. John Porter and also lectures of Dr. John Wood. Also management of HCV and HBV.
For all the above plus others, check this link
http://www.thalassaemia.org.cy/pan_me.html

manal


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Offline nice friend

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Re: Notes from the Egyptian and Syrian conferences
« Reply #14 on: October 03, 2009, 08:29:55 AM »
Thanx , Manal  for sharing these links .. i dont have ADobe Acrobat reader right now , but i will soon to view these files , all 3 links r on top of  my topic of interests ... :thankyou2 ...

Best Regards
Umair
Sometimes , God breaks our spirit to save our soul.
Sometimes , He breaks our heart to make us whole.
Sometimes , He sends us pain so we can be stronger.
Sometimes , He sends us failure so we can be humble.
Sometimes , He sends us illness so we can take better care of our selves.
Sometimes , He takes everything away from us so we can learn the value of everything we have.

===========
Umair

 

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