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Challenging pediatric CML case in USA

Jeff Lipton
Topic Author

1 month 3 weeks ago #2060 by Jeff Lipton

Replied by Jeff Lipton on topic Challenging pediatric CML case in USA

Normal hemopoiesis has not recovered for whatever reason and what does recover here is clonal. All the TKIs are good at shutting down the CML clonal hemopoiesis and switching will not change things. Persistent cytopenias is one of the very few indications for stem cell allografting and as I pointed out in a very recent commentary in Am J Hem, nothing will be gained by delaying and the risk of cytopenia related problems can only get worse. This is a young man and should have a good transplant outcome. I agree with Tim, but would not delay.

Henry D
Topic Author

1 month 3 weeks ago #2061 by Henry D

Replied by Henry D on topic Challenging pediatric CML case in USA

Thank you Prof for the comprehensive analysis of the situation. I am from Uganda and we face such challenges. However, I wish to be enlightened on why the first line treatment was dasatinib and at a high dose. I think we have not identified any ABL1 mutations in this young patient but what would be the role of other lines of treatment? I am asking this because in low income settings access to Allotransplants is not possible.

Henry

Delphine Rea
Topic Author

1 month 3 weeks ago #2062 by Delphine Rea

Replied by Delphine Rea on topic Challenging pediatric CML case in USA

Clear indication for an allogeneic stem cell transplantation.
Deficient underlying Ph neg hematopoiesis.

Anna Turkina
Topic Author

1 month 2 weeks ago - 1 month 2 weeks ago #2063 by Anna Turkina

Replied by Anna Turkina on topic Challenging pediatric CML case in USA

Dear Colleagues,

I don't have enough information to make a decision. To clarify the cause of cytopenia, it is necessary to perform a trepanobiopsy. In the era before TKI, myelofibrosis in the bone marrow was not rare (mainly in patients with hyperthrombocytosis).

My suggestions: if this patient is in a low-risk group, I would not rush to allo BMT. It may be recommended to prescribe dasatinib at a dose of 50 mg per day and eltrombopag 50 mg x2 times a day (increased dose). Evaluate the results of therapy after 3 and 6 months. When the platelet count has stabilized, eltrombopag is discontinued and increase the dose of dasatinib to 100 mg.

In a case of a high-risk patient with myelofibrosis, allo-BMT may be the optimal solution.

Kind regards - Professor Anna Turkina

Last edit: 1 month 2 weeks ago by arlene.

Jeff Lipton
Topic Author

1 month 2 weeks ago #2064 by Jeff Lipton

Replied by Jeff Lipton on topic Challenging pediatric CML case in USA

Marrow is empty and biopsy has been done. Although CML seems to have responded to DAS, normal hemopoiesis has not recovered.

This child is pancytopenic and at risk. G-CSF could be tried for a week or so to see if there is any response and this will give you an idea if there is any functioning reserve. I would not wait 3 months to see if platelets respond to eltrombopeg.

If no response to the G-CSF, I would not wait around for a problem. A lower dose of DAS or a drug switch will not allow recovery of what is not there. Proceed with an allograft. Waiting just increases the risk of infection, bleeding and/or platelet refractoriness developing. I continue to stand by the strategy that if SCT is on the table, waiting does not improve things and may make them worse

Michael Mauro
Topic Author

1 month 2 weeks ago #2065 by Michael Mauro

Replied by Michael Mauro on topic Challenging pediatric CML case in USA

Hi, i agree with others that this is an ominous combination of severe myelosuppression coupled with response albeit limited. I do not see ABL mutation testing, this may be informative for any therapy in the short or long term; it is intriguing there was stability followed by severe cytopenias, suggesting expansion of different clones. Historically transformation risk is high in these cases of severe myelosuppression and limited response, especially as therapy is difficult if at all and dose intensity drops. Growth factors may be tried to stabilize/bridge. Allografting seems the right path especially as disease burden is rising.

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