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A 19 yo on asciminib as 4L

  • Nobuko Hijiya
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5 months 1 week ago - 4 months 3 weeks ago #1930 by Nobuko Hijiya
A 19 yo on asciminib as 4L was created by Nobuko Hijiya
The patient is a 19 yo male.  He was initially diagnosed in 2017.  He came to us from another center after he failed imatinib, dasatinib and nilotinib.    There is no doubt about his compliance.    He has responded to each TKI initially, then, the level went up.   He did not have TKD mutations.   When he was referred to our BMT team, I suggested asciminib as he was18 yo. (Asciminib has not been approved for children.)  He started standard dose 40 mg BID in April 2022 and achieved molecular remission.   He achieved MR3 in 4 months but there are some up and downs up to 0.5%4/12/22       10.3% (prior to asciminib)5/31/22       3.4%8/19/22       0.13%11/18/22     0.039% (MR3)3/9/23         0.065%6/27/23       0.58% (lost MR3)7/10/23       0.375%7/28/23       0.098% (regained MR3)8/29/23      0.028%  (the lowest so far)9/25/23      0.108%10/25/23    0.21%The best donor option is haplo.   What do you recommend?
I appreciate your thoughts.  

Nobuko Hijiya, MD
Pediatric Oncology
Columbia University Irving Medical Center
Last edit: 4 months 3 weeks ago by arlene.
  • Delphine Rea
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4 months 3 weeks ago - 4 months 3 weeks ago #1932 by Delphine Rea
Replied by Delphine Rea on topic A 19 yo on asciminib as 4L
Dear Nobuko,

I suggest to maintain him on asciminb, simplify at 80mg QD and from time to time if fluctuation stable, ask for KD mutation analysis.

Best regards,

Delphine Rea
Last edit: 4 months 3 weeks ago by arlene.
  • Tim Hughes
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4 months 3 weeks ago - 4 months 3 weeks ago #1933 by Tim Hughes
Replied by Tim Hughes on topic A 19 yo on asciminib as 4L
Hi Nobuko,

Very difficult case. It looks like he will not sustain a good molecular response to asciminib but I would probably watch for a few more months to be sure. If he is losing response to asciminib, which looks likely, and given the less than ideal donor, I would be thinking about ponatinib (45 mg/day with reduction to 15mg/day if/when he achieves MR2) as a last chance before considering the transplant option. Before stopping the asciminib it would be important to screen for kinase domain and myristoyl site mutations. Any single mutation should not represent a problem for ponatinib but if he has compound mutations, ponatinib may not be a good option – depending on the actual mutations.

Cheers - Tim
Last edit: 4 months 3 weeks ago by arlene.
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