Discussion Forums > Thalassemia Major

AP Biology Student Looking for Insight

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faitdavid:
Okay, thank you Zaini!

patel.1005:
Wow an average day huh, well its pretty average...with a twist. I do everything everyone else does. Only thing different about me is that every now and then I have to go and refuel ;) and I have to take desferal infusions every night. I'm 22, so I go to school, I go out, I have a life and a part of that is transfusions and chelations. My doctors where always hesitant about letting me play team sports, so they would never sign the physical forms in middle and high school, but it didn't make a difference to me, I still played with friends and stuff just as hard as any other kid.

Physically- ya when its time for a transfusions, I generally get frequent headaches, I'm more tired through out the day,  and when its been a REALLY long time (sometimes I push transfusions of b/c I want to go on vaca or I cant schedule it the week its supposed to be) i can get short of breath sometimes (really probably only been twice in my whole life). I have found that working out, doing cardio, lifting weights generally helps me maintain an energy level through the month and has actually caused my hemoglobin levels to be higher that month (its actually something that really interests me, I keep looking for studies associating exercise and hemoglobin levels but I haven't found anything yet).

Transfusions- I go every 3 weeks. Biggest problem I have with them is that on one hand they give me life (lol my brother calls me his little leech ;) ) and on the other they cause side effects that could potentially kill me (iron). I hope and pray every day that the gene therapy trials that are currently going on an thalassemia patients are successful and sometime soon I can get the therapy.

Chelation- I didnt start until about 8 years ago, which really is late for thalassemia patients. Generally you should be monitoring iron from infancy and maintaining a relatively normal level. My parents, however, couldn't handle administering the daily infusions so I didnt start until I turned 16 and could do it on my own. I started on desferal but I was at the age where I didnt really know the consequences of not taking my medication and rebelled. I wouldn't take it all the time. Eventually I moved to Exjade because it was an easier regimen but my compliance was even worse because I couldn't handle the side effects. Then I hit a point around 18 or 19 where I got pissed at the world and everything because of the thal. it was my why me? phase. I rebelled, went completely off the chelators. I didn't know much about thal and I had never bothered to learn about it so when someone asked me about it i just said its a blood disorder that require transfusions. Eventually I started getting random liver and heart pains. I started looking up more information about thal and iron overload and realized that I was slowly killing myself. I immediately started back up on the chelation, but the damage was done. My levels were through the roof! its taken me 2 years to bring my iron down to about 7000. Its going to be a major accomplishment when I finally have it complete under control. The desferal is a pain in the butt (soft tissue if you want to be more accurate) but its keeping my iron in check so I take it religiously now and can only hope for a better pill form to be developed that I dont react as much to.

So your big question, what do thal patients want to be able to do easier?
I dont know how to answer this, its pretty much a toss up between the chelator and the transfusions. A safer, more efficient chelator would be welcome, but a cure would be ideal.

Hope this helps, if you have specific questions dont hesitant to message me!

Archana

faitdavid:
Thanks for the response Archana! That's interestig how they disnt want you to play sports before and now you're finding that exercise helps keep your hemoglobin up. That is late for chelation! Now I think they start when ferritin hits 2000, and it was probably much higher at that point. Wow, 7000 is way up there! Good luck!
I was talking to a representative from Pfizer today (his som goes to my school) and he said that they're working on a stronger oral chelator that could work independently of desferals, so that's great. I think it's in phase 2 or 3 testing, and then it has to get approved by the FDA, which puts it around 3-5 years from the market...

David Fait

Andy Battaglia:
David,

The oral chelator mentioned is probably FerroKin, which is moving along in trials. New developments in chelation are necessary because there are various problems associated with every chelator currently being used. Another chelator that showed great promise is S-DFO (starch desferal), a chelator that would be administered for about one hour per week by IV. Not having to use chelation daily is quite attractive to patients who have to deal with one side effect or another on a daily basis. This drug could also supplement another chelator in patients with a high iron load. It would guarantee that an almost constant chelation takes place, which is the ultimate goal of iron chelation. Unfortunately, this drug has been stalled by a lack of funding. In my opinion, this drug would greatly improve chelation compliance among those bothered by side effects of the current chelators. I would really like to see it revived with new funding.

faitdavid:
Yes Andy, I believe that was it. Like exjade causing liver and kidney problems, right? I just looked up the S-DFO, and it seems really good, but that's a shame about funding. Couldn't we get some anemia foundations like CAF and the Sickle Cell Foundation among others to contribute, since many blood disorders require frequent transfusion, and as a result, people with these conditions have high iron, and need chelation. I mean, I'm sure there would be a gooddeal of interest, but maybe not the amount of money needed for a large undertaking. Developing a drug costs almost a billion dollars, 12 years, and almost 1000 failed other drugs...

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