Hi, after a consultation with the consultant yesturday my daughters cml is not being controlled by the second TKI. She is still at 99% detectable. We are 9 months into our journey. There was talk of trying a 3rd alsongside planning a transplant just incase it doesnt work either. What are the chances of the 3rd tki working? Worried sick about a transplant. Shes only 12!
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Second drug to fail... worried mum
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Jenny:
So sorry to hear about your daughter - life's certainly not fair. Could you perhaps give us some more info like the first 2 TKI's and the dose, whether or not her doctors have done additional mutation testing, has she achieved CHR, what is her blast count? Hard to say if a third TKI would be more effective, but since they all work a bit differently, there's no harm in trying. If a transplant is the ultimate answer, she has age in her favor. And when it is successful, no more TKI!
Sending prayers your way
First was imatinib at 400mg.
Second was disatinib at 70mg.
Third is possibly nilotinib at a dose to be confirmed when they have spoken to an adult team.
I dont know what the numbers are or if theyve done additional testing for mutations i just know her fbc's are normally good with the exception of her platelets which fluctuate up and down regularly. Shes still in the chronic phase which is good. Marrows are being taken every 6-8weeks and are still detectable at 99%. They have just confirmed she has fibrosis at grade 3 which im told is pretty bad meaning marrow samples are not the best.
She needs a mutation test right away. She may have the t315i mutation or variant which only a few drugs control. There is no reason for her doctors to keep guessing. Demand the mutation test.
In the mean time, as long as her blast count is low (below a few percent), she has time. Blast cell crisis is what kills in CML - without blast cells, CML is chronic and slow. One thing to minimize blast cells is to make sure her blood vitamin D level is around 50 or higher (but less than 100). Low vitamin D enables blast cells to rapidly divide without differentiating including leukemic blast cells. Vitamin D is needed for blast cell differentiation. A little known fact most doctors have no clue about.
I second Scubas comment 100%. Mutation testing should be done. If T315I is detected Ponatinib should be started as it is the only TKI that can control T315I and has shown effective in doing so. Even if they are starting to have transplant conversation and the other two TKI did nothing for your daughter only Ponatinib makes sense IMHO. It has shown to return Accelerated and even to a lesser extent Blast phase back to Chronic phase.
Of course T315I is not the only mutation which is why testing for them is necessary.
My doctor took a guess when I switched to Nilotinib but my first labs after switch were favorable. I have no doubt he would have said mutation testing even after only three months had my numbers continued to climb.
Praying they test her soon and find the right treatment to help her get this under control and moving on with life.
Theres been no mention about a growing number of blast cells. They have mentioned the vitamin d to me and she now takes a supliment daily. I didnt understand the full extent of why it was important so thankyou. Do you know what a mutation test involves? Im hoping now that they may have done one and not told me. We see the consultant again on thursday so i will be sure to ask.
It's interesting some doctors are now more aware of vitamin D's role in the immune system and blood development. Bone and blood go together. They are from the same embryonic cells driven through evolution by the sun. Imagine that (https://www.labroots.com/trending/genetics-and-genomics/11786/blood-cell...). This is why vitamin D is so important for blood health!
I can't stress it enough. Play in the sun.
(I wonder how many childhood leukemia's could have been prevented if our children played in the sun without sunscreen)
https://scitechdaily.com/strong-link-between-vitamin-d-deficiency-and-va...
Nilotinib is likely a waste of time and effort. The drug is very similar to Imatinib but binds more tightly. If Gleevec and Sprycel showed no response, Nilotinib is not likely to work - it could....just not likely. This is why a mutation test is SO IMPORTANT. It seems your daughter has other things going on in her bone marrow (fibrosis) which they need to examine very closely. CML may not be the only issue. Her doctors don't seem to be in an urgent mode, so likely blasts are not a major problem. This may give you time which is good.
In terms of transplant - that should be an absolutely last resort. But should it be necessary, data shows that children are much stronger to survive transplant in comparison to adults. That is in her favor. Hopefully she won't need a transplant.
Some additional thoughts - consider supplementing with selenium (a couple of brazil nuts a day), Curcumin (C3 complex) as well as vitamin D3. Have her vitamin D level tested. It needs to be around 50-70 ng/ml for maximum blood/cancer benefit.
