Dear David,

We had a case that was almost identical to your case. This is a teenage boy with serious compliance and social issues. The treating physician tried imatinib, dasatinib and nilotinib, but he was not compliant, then tried omacetaxine. He is stable on it now. The difference in his case is that he does not have a good donor and refused transplant.

For your patient, I would be inclining to transplant having the fact that she has a good donor and better outcome with transplant in children/adolescents in general. She has another 60-70 years of life expectancy. Compliance with post-transplant medication may be an issue, but it is not indefinite like TKI.

Nobuko

Nobuko Hijiya, M.D.
Pediatric Hematology/Oncology
Lurie Children's Hospital
Northwestern University

Hello, I am a CML patient advocate (President of The CML Society of Canada) as well as a Masters level Trainer on coaching patients with chronic illnesses in the area of self management. The Self-Management course is 6 week course for patients that was developed at the Stanford University School of Medicine and offered at the McGill University Health Centre, here in Montreal.

As such, I wish to comment on the patient adherence issues of this case. It might be a good idea to have either the patient or the patient's mother, ideally both, reach out to Mr. Greg Stephens of The National CML Society in the US - 1-877-431-2573 (I believe you are located there?). He may be able to help pair the patient and their family with a fellow CML patient mentor who can help this patient see the incredibly hopeful future for CML patients. Particularly young patients.

Recently, CML Patient Advocates worldwide met at our annual New Horizons meeting where we learned about the great success of the stopping drug trials. This young patient may very well be a candidate for such a trial in the future. Thereby allowing her to lead a very normal life, choose an educational path, career and even have a family. She needs to realize that the path to this 'normal' life starts with strict adherence to therapy and accepting guidance on self-management techniques and how to manage side effects of treatment.

She sounds like she and the family are in great despair and could be helped with counselling, mentoring and support. Otherwise in spite of all your best efforts and the best drugs available, she may never be able to comply with the necessary treatment.

Thank you for all the great work you do.

I agree with Prof Saglio. She is on the right pathway and I would endorse a better family's enviroment. Psychological support is important during adolescence especially fighting against cancer. transplant choice should chase up if the case but not to obtain a better compliance.

As she is showing a continous trend towards reduction of BCR-ABL level and she has now reached a value compatible with CCyR although not yet MMR, I would not recommend for the moment to move to SCT. I would try to reach the maximum of adherence to therapy even reducing, just in case, the dasatinib dosage again to 100 mg and maybe also to 80 mg per day if the lack of adherence is due to side effects.

Trouble is, it doesn't matter how good the drug is, if the patient does not take it...
I would continue with dasatinib and see how she does. The trend is in the right direction albeit slow likely because of compliance. I think what we forget is that compliance is just as if not more important post allograft and I have memories of people in this age group who did not take their immunosuppressives or supportive medications and did not do well.

Although she has not reached MMR by 18 months she is showing slow reduction in levels. I am not sure I would go to transplant here unless your concerns of compliance and losing her to follow up are driving this decision more than the PCR response to treatment.