Phase 3 Luspatercept Trial

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.

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.

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.)

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

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:

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?

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.

Is there any update on luspatercept trials?

Only comments from patients in the trials. Some are doing very well. I don't anticipate any official findings until trials conclude.

LUSPATERCEPT is a success! Phase 3 primary and secondary outcomes were positive as reported on July 9. FDA submission will be during first half of 2019 so we could potentially be using Luspatercept in Summer/Early Fall 2019.  good news! Congrats and best wishes to all

Great News..thanks for sharing

We have gotten two very positive reports about Luspatercept in the past two weeks. The fact that they are seeking  approval demonstrates that they believe it works well enough to be a significant factor in treating thalassemia.