How to Approach the Conversation with Your Child’s Hematologist
By Nicole Kucine, MD, MS
It can be difficult to know what to expect when your child is being evaluated for an MPN. There is limited guidance on the internet, which can make specialist visits overwhelming to families. As parents, you can expect that you and your child will be asked many questions about symptoms he or she might be having, including headache, abdominal symptoms, rashes, and itching. Your child will undergo a number of blood tests, including some genetic tests, as well as a bone marrow examination. Children who are having symptoms specific to a certain body area may need radiologic tests (such as an ultrasound or MRI.) You should feel free to ask anything you want, and make sure if you are searching the internet you look at reliable sources (such as the MPN Research Foundation, MPN Advocacy Education International, or cancer.gov.) Some of the questions I am often asked include the following:
Does my child have cancer?
This is a tricky question, and others may disagree, but I do not think of children with MPN as having cancer. In adults, the World Health Organization criteria considers MPN to be chronic forms of leukemia. We are still evaluating MPN in children, and at this point it is not clear that they are all the same disorders in kids as they are in adults. I view the classical MPN in children as chronic bone marrow disorders, and while they have the potential to transform to acute leukemia, this is not something that has been reported in the literature. By envisioning these as chronic illnesses, I think it helps to set the expectations for long-term follow-up and aiming for keeping day to day activities as normal for your child as possible.
Does my child need the bone marrow evaluation?
The answer to this is definitely yes. The bone marrow exam is an extremely important part of the diagnostic process. It can provide a lot of information about what is going on with your child’s blood cells at the source. Things like storage iron, fibrosis, and the appearance of the precursor blood cells are studied. The procedure itself is performed with anesthesia to make sure your child is asleep during the bone marrow test and doesn’t remember it. The pain following a bone marrow test is generally very mild, and children may not require any pain medicine or might require a dose of Tylenol.
What type of MPN does my child have?
The diagnostic criteria for the various types of MPN are based on years of study and data on adult patients. They include features like appearance of bone marrow cells, genetic findings, and lab criteria. Making the appropriate classification for adult patients is important for discussions of prognosis and treatment. We do not yet know if we can directly apply these criteria to children, and knowing the exact type of MPN each child has may not be possible. While it is important to gather all of this information in children, it may not be as important to specifically name the exact type of MPN, and “MPN, unclassifiable” is an appropriate diagnosis for a number of children. The decisions about how to counsel families, what treatments may be recommended, and what follow-up is needed, will be made based on a variety of findings.
Does my child require treatment for his or her MPN?
The answer to this question varies, as I do not believe treatment is required for all children with MPN. I usually determine the need for treatment based on an individual child’s symptoms and lab findings. I generally do not recommend treatment for children who are asymptomatic and have reassuring labs. Children with mild symptoms in the setting of a high platelet count can often benefit from low-dose aspirin, as long as they are not showing evidence of bleeding or acquired vonWillebrand disease. Children with high red blood cell counts can also benefit from low-dose aspirin or phlebotomy. When a child has a severe clinical event such as a blood clot, or does not have improvement of symptoms with initial therapy, then cytoreductive therapy is appropriate. Which medication is used should be decided based on a conversation with the family and the treating doctor. I have been asked about what is my “cutoff” for high platelet or red cell counts for treatment, and there isn’t a standard cutoff. For example, I don’t think an asymptomatic child with a platelet count of 1.3 million necessitates treatment if they are feeling well and otherwise healthy. If you asked 10 different hematologists, you would probably get 10 different opinions on when to treat, so I think it’s important to have a conversation with your hematologist about the risks and benefits of different treatments.
While it can be frustrating to be facing a rare disease, there is ongoing research to help us better understand these conditions in children and adolescents. Keeping an open mind and making sure you have good communication with your child’s hematologist is the most important thing you can do.