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Thalassemia Patients and Friends and thalpal Ā© A. Battaglia 2017





54213 Posts in 5702 Topics by 5802 Members
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A message for all  parents who are thals. Keeping your iron load under control is an absolute obligation to your children.
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Author Topic: Phase 3 Luspatercept Trial  (Read 19586 times)
Andy
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« Reply #45 on: January 14, 2017, 06:24:22 PM »

The goal is raising total Hb, so that's what gets reported. They may be following other values, as well, but in the context of trials, the goal is specific. I am curious if this would raise MCV levels. I imagine it would.
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Andy

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sofear
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« Reply #46 on: March 06, 2017, 02:30:53 PM »

I am curious if this would raise MCV levels. I imagine it would.

I feel like that probably won't be the case. I just had a look at some older power points of Luspatercept (like this one) and it seems that the increase in hemoglobin always happens with quite a large increase in the RBC count. Why is no one talking about the MCV that always seems to be low in thalassemia patients? We have multiple options to increase the RBC count, that is nothing new.

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sofear
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« Reply #47 on: May 03, 2017, 01:01:25 PM »

I'm not sure whether it has yet been posted, but I recently stumbled upon a nice animation of the working mechanism of Luspatercept:

https://www.youtube.com/watch?v=oPfig2aOIYw

(I'm not sure how accurate it is, though, but it's pretty cool to watch.)
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Manal
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« Reply #48 on: May 03, 2017, 01:45:44 PM »

I met Dr. Piga last November in Jordan ( pan middle east conference ) and he said that he expects Laucpatercept to be available in the market in 3 years and he added that in few months they will start the pediatric clinical trails.

Manal
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sofear
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« Reply #49 on: May 05, 2017, 06:42:46 AM »

After I saw the animation of the mechanism of action of Luspatercept, I tried to understand it a bit better and went through a lot of videos and articles about basic biological topics but also thalassemia-specific ones.

Although I'm not able to judge the quality of the content, I later found a site with articles about thalassemias of which some were really interesting to read:

http://cursoenarm.net/UPTODATE/contents/search.htm?search=thalassemia&menu=0&submitType=Search

One part of an article (http://cursoenarm.net/UPTODATE/contents/mobipreview.htm?29/32/30208) had the following passage:

Quote
Normal hemoglobin biosynthesis requires an intact structural gene and the structural and spatial integrity of silencers, enhancers, promoters, and LCR sequences. Despite considerable effort, we do not fully understand how the sequences and factors regulating globin gene expression interact with one another to ensure high levels of expression of the proper globins at the proper developmental stage and during the proper steps of erythroid differentiation.

This information has been last updated in 2011, so I'm not sure how up-to-date it still is, but the question that came to my mind was:

How are the processes of red blood cell differentiation and maturation, which Luspatercept interacts with, actually linked to the expression of the globin genes?

« Last Edit: May 05, 2017, 08:00:16 AM by sofear » Logged
Hafiz Akhtar
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« Reply #50 on: May 10, 2017, 12:44:10 AM »

Andy Sir what is ur opinion in this regard. can v say that it will b approved & commercialized within 3 years and our children 2 some extent will be free from transfusion. i mean to say burden of transfusion will b reduced. perhaps our dream will come true.
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