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Resistant Pediatric CML

  • Nirmalya Roy Moulik
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3 days 19 hours ago #2131 by Nirmalya Roy Moulik
Replied by Nirmalya Roy Moulik on topic Resistant Pediatric CML
My response wouldn't differ much from the adult experts- I would continue the asciminib for a longer period before making any changes immediately. Of course, BMT should be considered, as this child has a high chance of treatment failure over time.
Also, the importance of the ASXL1 mutation in children with CML is less clear than in adults.
Hope this helps.
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  • Nobuko Hijiya
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1 day 16 hours ago #2132 by Nobuko Hijiya
Replied by Nobuko Hijiya on topic Resistant Pediatric CML
Having the pending result from 7 months, I would give her a little more time with asciminib, once daily. Asciminib decreased the FISH from 4 months to 6 months.
It would be a good idea to at least consult BMT service and look at the mutation analysis. But I would give her a little more time, a couple of months, as long as she is in CP and BCR::ABL1 is decreasing. The best timing of BMT is not very clear. As long as the patient is in CP, I think it is OK. ASXL1 mutation is known to have less favourable outcome in adults. We do not have data in peds yet.
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  • Jeff Lipton
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1 day 16 hours ago #2133 by Jeff Lipton
Replied by Jeff Lipton on topic Resistant Pediatric CML
For whatever reason, this young woman is resistant with very little response - this may be a mutation or clonal progression, but that does not matter. The longer that is waited, there is a chance that she will lose the only treatment that will likely benefit her in the long run, ie an allograft. Playing around with TKIs in combination or not runs the risk that she will blast off. Best result with an allograft is NOW, if a donor is available. Debulking with a TKI will not improve things. Hydroxyurea if necessary and transplant. I am concerned that she was on the cusp of what we used to call accelerated disease at diagnosis - basophils worry me here. Waiting here is a ticking clock.
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