First of all, I want to thank everyone for input.

To answer Pankaj’s question;
1. What was the Sokal score at presentation?
He was in intermediate by using Sokal score. However, it is very important for us to remember that Sokal score or other scoring systems have never been validated in pediatric population. I hope we will have data to validate or modify it in the near future.

2. When and why was the mutation testing done?
Mutation analysis was done a couple of times. First, at 3 months when cytogenetic response was not favorable and second time at 15 months when bcr-abl PCR increased a little from 12 months (two times).

3. How's the support of family members?
They are great. They are very intelligent and highly capable people. I have no doubt about their compliance.

4. What is the age of HLA matched sister?
Two years younger than the patient.

Not only the issue of scoring systems I mentioned above, there are many pediatric specific issues in CML that we need to work on. That includes children’s much longer life expectancy, better tolerability to HSCT and unique morbidities. Suppression of bcr-abl for 10 years as done in 70 year old patients may not be the best approach for children. We are getting more and more data on growth failure by long-term use of TKIs.

We are about to open a CML study in Children’s Oncology Group (COG). Although the number of children with CML is much smaller than adults, this study will provide important information. Also important is establishing standard care for children with CML.

We are also working on a retrospective study using a large dataset from CIBMTR to evaluate long-term outcome of HSCT.

We are working hard to improve the outcome in children with CML. Thanks again.

Nobuko

Thanks Nobuko for clarifications. I think the debate about treatment in this kid is on the following parameters

1. Long-term adverse effects (known and unknown) of TKI vs risk and long-term adverse effects of allogeneic stem cell transplantation.

2. Applicability of adult data on pediatric patients (as quoted by Dr. Tim and Dr. Jorge)

I would put my humble bet on close molecular monitoring of the kid and keep option of transplantation in the reserve.

Regards

P

Dept. of Internal Medicine
PGIMER, Chandigarh, India.