My Child Was Diagnosed With an MPN: Now What?

Pediatric myeloproliferative neoplasms (MPNs) are extremely rare and as with any rare disease, it is important to find a treatment team that specializes in the condition and that the parent and caregivers can trust.

“With rare diseases, doctors usually like to contact other doctors if they don’t have a lot of expertise in something. And I think it’s also very important to understand why your doctor is doing something,” said Dr. Nicole Kucine, associate professor of clinical pediatrics at Weill Cornell Medicine, in an interview with CURE®.

To treat or not to treat?

MPN is a chronic condition with a high survival rate – especially in younger patients – so children can live with the disease for years to come. Treatment is not always necessary, so parents and their children should talk to health care providers about whether or not to start therapy, and if so, what the goals of treatment would be.

“There are definitely some kids that I think most people would agree with (who need to be treated). If they have a large blood clot, have terrible symptoms, or if something bad is going on, their disease needs to be brought under control,” Kucine said. “I think people feel more comfortable in that environment by giving the patient cytoreductive therapy.”

The goal should always be disease and symptom control to promote as normal a life as possible for the child with the MPN.

“Finding ways to manage symptoms, getting kids involved in activities they want to do, and finding ways to make that happen — that’s really important,” Kucine said. “So that kids can feel like kids and not feel like they’re sick all the time and can’t do what they want to do.”

Bone marrow transplant

Currently, there is only one treatment option that can potentially cure an MPN: bone marrow transplant. If a patient has a matched sibling donor who does not have the same MPN-causing mutation as the patient, that may be a viable option.

“If someone is completely healthy, they are less likely to go for a transplant. But I always say (to patients and their families) just make an appointment with the transplant team and hear about the risks and benefits. Get an idea of ​​how transplantation can really look like and what it can offer your child,” Kucine said.

Otherwise, healthy children are usually good candidates for transplants. Younger people are generally fitter and lack co-morbidities of older patients with MPNs that may make them ineligible for the procedure.

“You usually have better results if you have a healthier patient because you have fewer side effects from the drugs… If there are other co-morbidities, many drugs around transplantation can affect the liver or kidney or heart. If you already have perfectly healthy organs it is certainly easier to have a transplant than if you have medical problems.”

Change the way you see the disease

Part of promoting a normal life after an MPN diagnosis may also involve re-evaluating how the patient and family members view the disease.

MPNs are technically considered cancers — which can be a good thing, because that classification opens doors for research and funding money. However, it can be emotional and overwhelming to hear that your child has cancer.

“For someone who was diagnosed with an MPN at age 8, I don’t think it’s helpful to think of this as a blood cancer. I consider this to be chronic bone marrow disease,” Kucine said, stressing that an MPN diagnosis shouldn’t stop a child from doing what he loves or going to school and other activities. “I try to put it more in a long-term view. and really consider it a chronic (disease).”

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