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Facts to Know While Undergoing Therapy for CML

Leukemia Leukemia Treatment

CML Treatment: Medication or Transplantation?

Summary & Participants

Mounting evidence from clinical trials shows that patients with chronic myeloid leukemia (CML) may have long-term treatment success forgoing the only known cure for their disease. Watch and listen as CML experts explain how a promising drug now complicates the decision facing many patients-whether to seek a bone marrow transplant.

Medically Reviewed On: June 26, 2006

Webcast Transcript

ANNOUNCER: There are several effective medical therapies for treating Chronic Myeloid Leukemia. But there's only one proven cure.

STEPHEN O'BRIEN, MD: Well, clearly the most desirable treatment goal is to cure the patient with Chronic Myeloid Leukemia. At the moment, the only know curative therapy is a bone marrow transplant.

ANNOUNCER: But a transplant carries significant risk, and doctors say a new drug may offer another path toward long-term survival. It's called imatinib, or Gleevec.

STEVE MACKINNON, MD: And those patients may not require a transplant in the future. Clearly we don't know that yet, but the whole appears that that the treatment could postpone either temporarily or indefinitely bone marrow transplantation for many patients.

ANNOUNCER: In a bone marrow transplant, doctors use chemotherapy and radiation to kill most of a patient's marrow, to destroy diseased, leukemia cells. Then doctors replace the marrow, with a transplant from a donor.

Success is most likely if the transplanted marrow is a good match, especially if the donor is a sibling, or other close relative.

Results are also best if the patient is young, and otherwise in good health. Even so, there is significant risk.

STEPHEN O'BRIEN, MD: If you were to embark on bone marrow transplantation, in the best centers with the best matched donors, you might be looking at a chance of something seriously going wrong that could life threatening, of around about 15 or 20 percent.

ANNOUNCER: Those odds explain why any improvement in medical therapy draws attention.

In clinical trials with CML patients, a standard test shows Gleevec is more effective than more traditional therapies in reducing the number blood cells with genetic damage.

GWEN NICHOLS, MD: There are a large number, maybe 40 percent of patients, who get a chromosome response to Gleevec. We don't know how long those will last, but some of them seem to be quite stable, and without evidence of the chromosome coming back.

ANNOUNCER: And while that's not a cure, studies with other drugs show this so-called "cytogenetic response" corresponds to longer survival. But data on Gleevec is preliminary, and doctors are still debating how and when it's best used.

GWEN NICHOLS, MD: The tough question is, if we start with that therapy or transplantation.

STEPHEN O'BRIEN, MD: It's fairly clear if you're at the extremes of the spectrum. So for example, if you're 20 and you had a related donor that matched very well, and you were fit and healthy, I suspect myself and most other doctors would recommend a bone marrow transplant.

If you're 60 and whether or not you have a donor that age, I think the risks of transplant are excessive perhaps, and then drug therapy would clearly indicated.

For the middle ground, I think one has to discuss the options with the patients and provide as much data as one can, and then it's a very personal choice for the physician and the patient in their particular circumstances.

ANNOUNCER: Another difficult decision is what a patient should do who is taking Gleevec, and who shows a good response.

STEVE MACKINNON, MD: My own opinion, and I'm a bone marrow transplant physician, is that if I have a patient who's on Gleevec. I would want them to continue on the drug. On the other hand, for patients who don't have good responses to Gleevec, if they're young enough, those patients can then proceed with the transplant.

But one of the great unknowns nowadays days is will prior Gleevec therapy effect the results of transplant subsequently? And that could happen in a number of ways.

ANNOUNCER: One concern is that delaying a transplant might increase risk. It's possible that the drug itself might lead to complications. And a patient who fails medical therapy, like Gleevec, might face a transplant with more advanced disease.

But it's also possible that using Gleevec may improve results in later transplants.

STEVE MACKINNON, MD: And the last thing to bear in mind is that Gleevec may put patients into remission prior to transplantation. And that has a possibility of actually improving the results of transplantation.

ANNOUNCER: No doctor is ready to call Gleevec a cure for CML. But if research shows Gleevec prolongs survival, doctors say many CML patients may enjoy long, symptom-free lives, without the trauma and risk of a bone marrow transplant.

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