10 replies to this topic

[wallystl]

Hello all.  Directing this 1st to Trey then to whoever else might have some insight.

I'm 2-1/2 years into CML, t315i for 1 year, on trial drug Ponatinib for 10 months with some success although the sides are difficult.

I have been resisting a BMT since I'm a single parent of a high school junior, trying to hold on till he is out of high school in 2013.  My best friend recently discovered that an employee of his has a wife who is a Dr. @ a leading NCI hospital.  As a favor he asked the woman some specific questions about my situation and my expected outcome if I go the BMT route.  The answers were frightening & unexpected although I guess the stats were available to me had I bothered to do the research.

In summary; I'm in chronic phase now with t315i.  If I wait till & if I ever progress to blast phase for a transplant the success rates are 80% at 3 months, 10 % at 12 months.  There also some issues with time between dx & BMT decreasing the chances for survival.These are not encouraging numbers for survival.

So of course my friend is of the mind that I should immediately begin plans for a BMT.  The debate is; how long can I maintain on the trial drug and avoid the BMT.  Also does the t315i change my chances for success with the BMT, also before blast phase there is the accelerated phase- what are the differences between these two phases?

So, what would you do?  Maintain the trial & hope for the best, move toward the BMT asap to get the best possible chance at survival?

Thanks to all and best of luck with the battle.

Wally

[Trey]

Overall, if a CML leukemia patient is responding to drug therapy, they should stay on drug therapy as long as it works and use transplant (BMT/SCT) as a last resort.  That would mean waiting to see the drug fail and the disease possibly progress to advanced phases.  Most patients would be able to see the failure occur in time to have a BMT before reaching Blast Phase.  The reason is that BMT success rates are relatively low (given the gravity of the issue) and can also lead to long term GVHD disabilities and/or organ damage.  Disease relapse rates are also higher than many would expect.  I wrote a post about BMT for leukemia including survival rates that cover many such issues:

http://community.lls.org/docs/DOC-1375

Regarding the success rates you quoted, it is very difficult to come up with specific rates, since many stats mix various types of leukemia and sometimes other blood diseases and various ages of the patients.  So the variables can be numerous.  But the 10% at 12 months is definitely too low.  For a patient in a situation like yours, the difference in probability of BMT success if done today or if the person somehow progressed to Blast Phase next year would not be significant enough to cause them to jump into a  BMT given the hazards of the procedure.  I do not know if you are currently responding well on Ponatinib, but unless a patient believes that drug failure is likely and no other drug would be available, a BMT is too risky to use as a prophylactic measure.  But BMT should be regarded as an arrow in the quiver of CML patients if truly needed; but if it were me, it would only be as a last resort.

[judyann]

Good Morning, Wally, 

I, too, am in chronic phase with t315i.  I was dx December 2006 and recently referred to a BMT center to "put everything in place in the event that a BMT becomes necessary".  I have failed Gleevec at 400, 600, and 800mg.  This week I am being bumped up to 140mg of Sprycel after failing 70 and 100mg.  My husband and I have had a consultation with the doctor who would be doing the BMT and there is so much to wrap your brain around and it does become overwhelming.  I would, however, recommend that you get a referral from your oncologist for a consultation with a bone marrow specialist.  That person can answer the questions you have.  And, be sure someone goes along with you because there is so much information and you can easily miss everything that will be told to you. Like you, one of my husband's and my concerns was the statistics for a BMT.  The statistics were not as bad as we had read but the process is rough . . .  very rough! 

I have not had my HLA typed and neither have my 5 siblings to see if any of us are a match.  Two of my siblings are already on the National Bone Marrow Donor registry.  The bone marrow transplant center wants me/siblings tested, however, the cost is $8000!  Yes! $8000!  The testing for me is $3000 and $1000 for each of my siblings . . .  and, even though I have an excellent health insurance plan, it does not cover HLA typing.  At the present time we are all in a "holding pattern" saving to cover the $8000 the lab fees/HLA typing.  My husband and I are also hoping that the Sprycel 140mg will bring my PCR-ABL numbers done again buying us some time.  Also, are you familiar with Ponatinib, a treatment for CML patients who have become resistant to available TKIs?  Ponatinib is very close to being approved by the FDA and the preliminary data is promising. 

I,too, will be anxious to read responses from others on this board, from CMLers who are resistant to the current TKIs available, and what steps are being taken by their oncologists.

Thanks for posting, Wally!  It is good to know others who are facing possible BMT/SCT. 

Judy

[CallMeLucky]

What is your response on ponatinib?  Have you achieved a complete cytogenetic response?

Do you have a matched sibling donor for BMT?  How old are you?  Do you have any other health issues?

I can say that for me, as long as I was stable on ponatinib, I would ride it out.  Ponatinib is for T315i what Gleevec was for regular CML 10-15 years ago.  There are many people who were at the end of the rope with CML and then started taking Gleevec and they are alive today.  There is a good chance that ponatinib will do the same for people with T315i.

BMT should always be the last option in my opinion.

Best of luck

[HPL]

I am also a Ponatinib patient, over 2 years into it, phase 1 patient. I have been Weak Positive for over a year now, stable. I had the T315I when I entered the trial. I agree with the comments above, I would use the BMT as a last resort, but I wouldn't wait too long after it's shown that I lost the response. It's never good to allow it to go to blast phase.

Judyann - Did your doctor talk to you about the compassionate use program from Ariad, the drug company that developed Ponatinib ? I know at least one person on a compassionate usage program. I have a contact at Ariad who I have talked to about the CU program, it's such a blessing since the Phase II trial is closed, yet there still exists a demand for it. I would strongly consider that route, given how effective the drug has proven for T315 patients.

Regards,

Hans

Zavie's Club # 1303

[hannibellemo]

Judyann,

Hans brought up the compassionate use of ponatinib and I wanted to make sure you had a contact for that. I know that you aren't new to CML but I just want to make sure you are aware that Jerry Mayfield has an excellent discussion board, too:  www.newcmldrug.com. He is using ponatinib under CU I believe and can tell you more about the process if you are interested. The process isn't simple from what I can tell but worth it for many. There has just been a recent discussion on the topic so if you aren't familiar you may want to check it out.

Good luck,

Pat

[BethG]

Hi Wally,

Sorry to hear you are having a tough time! Like others above, I am also on ponatinib due to the T315i mutation. It will be three years in June for me. I had challenges with side effects myself and I am doing well on 30mg...have you tried going to a lower dose? My PCR is essentially weakly postive and I'm generally doing well these days.

I would try to see what you can do with ponatinib before going for transplant. It is not for the faint of heart and a grueling procedure with no promises!

Good luck,

BethG

[susantheresa]

Hello Wally,

My name is Susan M and if you can get yourself to the post dated march 25 under Need SomeoneTo Talk To in the cmlblog, I wrote a bit about my transplant experience.  I to was a Cml patient when at the time Gleevac at 4, 6 and 800 mil. failed me.  There was no Spyracel or Tasigna out yet (this was 2004-2005) so I had no choice but to move toward transplant. I just have a couple of questions, How old are you and what state do you live in.  I would suggest that you meet with a Blood and Marrow Specialis.  My treating Hospital had several Blood Cancer Specialist with each heading up their own individual blood cancer.  My Dr. of coursewas the marrow specialist so that is who I met with.  Also, those comprehensive cancer centers all have transplant coordinators who take care of the insurance and  admissions.  They are ace's at navigating the insurance stuff. I am here to tell you that it was no walk in the park, but I had no choice, I moved out of chronic phase rapidly to accelerated and was told I would even go to blast sooner than later.  I do wish you the best in your decision, get the facts for your treatment because remember, although we have the same disease, how we respond to treatment is individual.  God Bless you on your journey.

[wallystl]

Hi everyone, thanks for the feedback.

This is the 2nd time I've faced this BMT delima and there seems to be a pattern of cancer centers trying to move patients to BMT & the cancer community trying to avoid BMT unless its the last possible option.  I agree with the answer here; I'm doing well on the trial drug and I'm going to continue that treatment until my condition changes, if ever.  Particularily helpful was the comment about how Gleevac was new 15 years ago & its saved many people from a BMT & they are still on Gleevac today.  So yes its possible that Ponatinib could be the Gleevac of 2012.  I'm being treated at a NCI center is St. Louis with an excellent transplant section.  So if and when the BMT becomes the last option I'll be in good hands.

Have a happy holiday weekend everyone!

Wally

[TeddyB]

Hope you are still responding well to Ponatinib and that your side effects are not to bad Wally.

Greetings from Norway.

[Happycat]

Hey, Wally,

Happy Father's Day to you!  (And all you other dads out there!)

Traci