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Challenging pediatric CML case in USA

  • Sarah Harney
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2 days 22 hours ago #2066 by Sarah Harney
Replied by Sarah Harney on topic Challenging pediatric CML case in USA
Thank you for your response.

Patient fortunately has good donor options and is a good candidate for transplant, so we are moving forward with allo BMT.

We trialed GCSF 5 mcg/kg for 2 weeks with no response (ANC remained 40-60 throughout this time) so have now stopped it.

I repeated a bone marrow 2 weeks ago which showed cellularity <10%. His BCR ABL PCR is interestingly down quite a bit- only 5.474% (major breakpoint e13a2, with 0.011% minor breakpoint e1a2) compared to 24% (0.020% minor breakpoint) in his marrow back in May. Dasatinib has been on hold since early April.

Myeloid NGS panel and BCR ABL mutation analysis were sent from most recent marrow, and are still pending.

My one question at this time is whether to start asciminib to try to get any more disease control prior to BMT, or to keep him off TKI so as to not further suppress any chance of possible delayed spontaneous recovery.

Thank you!
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  • Sarah Harney
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2 days 22 hours ago #2067 by Sarah Harney
Replied by Sarah Harney on topic Challenging pediatric CML case in USA
Thank you so much for your thoughts and recommendations.

We are moving forward with allo HCT as soon as possible. Trialed GCSF for 14 days with no response. Marrow still <10% cellularity, predominantly erythroid precursors. BCR ABL PCR down to 5% without any other intervention since last marrow 1 month prior (off TKI >2 months now).

Any thoughts on utility of starting asciminib to try to get as much disease control as possible prior to transplant? I see both pros and cons.
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2 days 22 hours ago #2068 by Sarah Harney
Replied by Sarah Harney on topic Challenging pediatric CML case in USA
Hi there, I opted to start with dasatinib as first line TKI for this patient as he was an older teenager. Since dasatinib may be associated with faster and deeper molecular response than imatinib, I figured it may have a better chance at TFR later down the line. And dasatinib 100mg is standard FDA approved dosing (BSA 1.8)
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2 days 22 hours ago #2069 by Sarah Harney
Replied by Sarah Harney on topic Challenging pediatric CML case in USA
Mild reticulin fibrosis (MF-1) without collagen fibrosis in both his diagnostic marrow and his most recent marrow. I have not been comfortable restarting any TKI at this point because of his persistent profound cytopenias (Plt <10, ANC <100 for 10+ weeks).
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