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Final Thoughts on the Treatment of Beta-Thalassemia

Panelists: Peter L. Salgo, MD, NewYork-Presbyterian Hospital; Maria Domenica-Cappellini, MD, University of Milan; Thomas D. Coates, MD, University of Southern California Keck School of Medicine; Farzana Sayani, MD, University of Pennsylvania; Sujit Sheth, MD, NewYork-Presbyterian Hospital/Weill Cornell Medical Center
Published: Friday, Mar 20, 2020



Transript: 

Peter L. Salgo, MD:
We have a little bit of time left. What I’d like to do is go across the table and give our viewers a sense of anything you’d like them to know—unmet needs, the way we’re going in the future, anything you’d like them to remember in about 30 seconds or so. Why don’t we start with you.

Thomas D. Coates, MD: To me the major issue with this disease, especially because it’s relatively rare, is that the patients should be connected with somebody who has substantial experience to manage them; the outcome can be very good. I’ve lived through part of this whole story—to see the advances have been amazing. I think that the overall outcome for these patients is terrific. But this isn’t something you can read from a list, a check-off list that you’re going to do in your office. It really requires expertise. Personally, I’ve known Nica for a number of years now, and the experience we gain from people who really are experts compared with us is invaluable. I really feel that, and I think that this is really critical. But the outcome, all that being said, with proper therapy is just really amazing.

Maria Domenica-Cappellini, MD: Definitely the story has changed. I still believe that we have to do some education in developing countries, where—as you said at the beginning—patients are not properly treated. And we have to be open to the new treatments. I just want to conclude that during the last 2 years, I was consulted by 20 couples for pregnancy, and 7 of them decided to have a child—affected children—because of the future.

Peter L. Salgo, MD: That’s amazing. You’re up.

Farzana Sayani, MD: I think that it’s a very, very exciting time. There are lots of new treatments, as we’ve all discussed. But for the community hematologist-oncologist, as an adult provider, it’s very important to continue to follow individuals with thalassemia intermedia—even if they’re not transfused—beyond just the hemoglobin. That was a key thing. There are lots of complications to monitor for, and I want to stress again reaching out to centers where there is a provider with experience in thalassemia. Even if it’s a once-a-year visit, still connecting because there are complications that increase as individuals get older. Sometimes it’s too late. So we really want to catch them early on. That’s what I would really stress.

Peter L. Salgo, MD: And you’ve got the last word.

Sujit Sheth, MD: Oh, I’m sure you have the last word.

Peter L. Salgo, MD: Don’t count on it.

Sujit Sheth, MD: I agree with what the other 3 experts have said. And it is really a truly exciting time. One of the things you asked about is, why do we need to think about all these newer things in development. Because we need to have as many options as possible. Because even though everybody seems to lump patients with the same disease into 1 cohort, these are individuals who have different needs in their lives, different approaches. Offering them varying options is really important. So it’s an exciting time, and it’s only going to get better.

Peter L. Salgo, MD: I just want to thank all of you for taking time out and joining us for today. What I get to see is a waterfront view of various medical specialties. And the takeaway is that this is an exciting time, whether it’s infectious disease and viral diseases or it’s beta-thalassemia, which—I never thought I would hear a respectable clinician say it—we have a cure for. I was pleased to be a member of a profession that’s made this kind of progress over such a brief period of time. I’m still in practice, and there’s more to come. Thank you so much for being here. On the behalf of the panel, I hope you found this Peer Exchange discussion to be useful and informative. I’m Dr Peter Salgo, and I’ll see you next time.


Transcript Edited for Clarity

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Transript: 

Peter L. Salgo, MD:
We have a little bit of time left. What I’d like to do is go across the table and give our viewers a sense of anything you’d like them to know—unmet needs, the way we’re going in the future, anything you’d like them to remember in about 30 seconds or so. Why don’t we start with you.

Thomas D. Coates, MD: To me the major issue with this disease, especially because it’s relatively rare, is that the patients should be connected with somebody who has substantial experience to manage them; the outcome can be very good. I’ve lived through part of this whole story—to see the advances have been amazing. I think that the overall outcome for these patients is terrific. But this isn’t something you can read from a list, a check-off list that you’re going to do in your office. It really requires expertise. Personally, I’ve known Nica for a number of years now, and the experience we gain from people who really are experts compared with us is invaluable. I really feel that, and I think that this is really critical. But the outcome, all that being said, with proper therapy is just really amazing.

Maria Domenica-Cappellini, MD: Definitely the story has changed. I still believe that we have to do some education in developing countries, where—as you said at the beginning—patients are not properly treated. And we have to be open to the new treatments. I just want to conclude that during the last 2 years, I was consulted by 20 couples for pregnancy, and 7 of them decided to have a child—affected children—because of the future.

Peter L. Salgo, MD: That’s amazing. You’re up.

Farzana Sayani, MD: I think that it’s a very, very exciting time. There are lots of new treatments, as we’ve all discussed. But for the community hematologist-oncologist, as an adult provider, it’s very important to continue to follow individuals with thalassemia intermedia—even if they’re not transfused—beyond just the hemoglobin. That was a key thing. There are lots of complications to monitor for, and I want to stress again reaching out to centers where there is a provider with experience in thalassemia. Even if it’s a once-a-year visit, still connecting because there are complications that increase as individuals get older. Sometimes it’s too late. So we really want to catch them early on. That’s what I would really stress.

Peter L. Salgo, MD: And you’ve got the last word.

Sujit Sheth, MD: Oh, I’m sure you have the last word.

Peter L. Salgo, MD: Don’t count on it.

Sujit Sheth, MD: I agree with what the other 3 experts have said. And it is really a truly exciting time. One of the things you asked about is, why do we need to think about all these newer things in development. Because we need to have as many options as possible. Because even though everybody seems to lump patients with the same disease into 1 cohort, these are individuals who have different needs in their lives, different approaches. Offering them varying options is really important. So it’s an exciting time, and it’s only going to get better.

Peter L. Salgo, MD: I just want to thank all of you for taking time out and joining us for today. What I get to see is a waterfront view of various medical specialties. And the takeaway is that this is an exciting time, whether it’s infectious disease and viral diseases or it’s beta-thalassemia, which—I never thought I would hear a respectable clinician say it—we have a cure for. I was pleased to be a member of a profession that’s made this kind of progress over such a brief period of time. I’m still in practice, and there’s more to come. Thank you so much for being here. On the behalf of the panel, I hope you found this Peer Exchange discussion to be useful and informative. I’m Dr Peter Salgo, and I’ll see you next time.


Transcript Edited for Clarity
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