Young Adult and Pediatric Patients With MPNs Should Remain Hopeful, Expert Says

Better survival outcomes were seen in young adults patients with myeloproliferative neoplasms (MPNs) compared with adult patients with MPNS, according to a paper, recently published in theCurrent Hematologic Malignancy Reports. Investigators led by Nicole Kucine, MD, MS also found that pediatric patients demonstrated lower mutations than adult patients, potentially improving their chance of survival and limiting disease transformation to myelofibrosis and acute leukemia.

Diagnosis of MPNs in pediatric and young adult patients remains rare, although it has been observed more frequently in recent years. The majority of data in MPNs, however, is largely based on older patients, which makes managing these younger patients more challenging.

Kucine et al reviewed available literature on pediatric and young adult patients with MPNs to gain more insights into the clinical care of these patients. Although the data were mostly retrospective, she found that pediatric patients had lower rates of common mutations, thrombotic events, and disease transformations to either myelofibrosis (MF) or acute myeloid leukemia (AML) than found in adults with MPNs.

It is typically more challenging to enroll pediatric patients than young adults to clinical trials due to missing the age cutoff in most clinical trials, which is usually 18 years of age. Because of this and that it is such a rare diagnosis, worldwide collaboration is needed to further evaluate this patient population and potential treatments that could be more beneficial. Currently, these patients are generally treated with hydroxyurea, interferon, or anagrelide.

In an interview withTargeted Oncology, Kucine, Pediatric Hematology-Oncology at Weill Cornell Medicine, discussed the findings from her review of the literature on children, adolescents, and young adults with MPNs. She highlighted what next steps are needed in this space in terms of research and what a community oncologist should do should for younger patients with MPNs.

TARGETED ONCOLOGY: What was the purpose of conducting this research?

Kucine:The paper was a review to try and summarize the available literature that is out there on pediatric and young adult patients with MPNs. We wanted to try and think about the important questions and directions for future research and clinical care for our younger patients with MPNs.

TARGETED ONCOLOGY: What are the treatment options for these younger patients with MPNs?

Kucine:For children and pediatric patients under the age of 18 or 21 years of age, depending on the center as most places are different in their age cut-offs, the things that have been used in the past are hydroxyurea, interferon, and anabralide. Those are the main drugs that are used. The number of pediatric patients who have been treated with ruxolitinib (Jakafi) are very low, and it has mostly been used in the setting of clinical trials. For young adults, they are usually eligible for most clinical trials that are open. A lot of the newer agents that are being studied in MPNs are potentially available for young adult patients as well. Usually, the age cut-off is 18 years old.

TARGETED ONCOLOGY: What were your findings in the study?

Kucine:The major findings were positive in thinking that young patients tend to do better than older adult patients with MPNs. This is probably for a variety of reasons, but specifically, in children with MPNs, these patients have very low rates of transformation to MF; there were only 1 or 2 case reports of patients diagnosed in childhood with an MPN and at some point transforming to AML. It is not really clear that this happens in childhood as opposed to happening when they are adults.

We found the same thing for young adult patients. Even though they do have a decent number of thrombotic events, the transformation rates appear lower and outcomes are good. That is reassuring because I imagine young adult patients and parents of those children being diagnosed with MPNs wonder what the outcomes are and the longer-term implications. Even though the data out there isn’t a large amount of prospective data, I do believe we need more study overtime of these patients to see what happens because having a disease for 30 to 40 years is probably very different than having it for 20 to 30 years if you are diagnosed when you are older. From what we are seeing so far in the retrospective studies, the data at this point is reassuring.

TARGETED ONCOLOGY: What is important to take away from these data?

Kucine:Young patients and families of these children diagnosed with MPNs should be hopeful and remain positive. We are seeing lower rates of the worst complications and good prognosis. They should be hopeful about future clinical trials, and I think it is important for young patients, if they are being diagnosed with an MPN, to either seek out opinions from those who have experience or from having their doctor contact people who have experience with this. Some of the things we are used to thinking about in older adults may not always apply to children and younger patients. I think younger patients probably have some different concerns and things that might need to be focused on when caring for them as opposed to caring for somebody who is 60 to 70 years old.

TARGETED ONCOLOGY: What are the next steps for research in this population?

Kucine:There is unfortunately not a lot of clinical trials for children. I am hoping over the next 5 years that will change and we will be able to open up some trials as we get more data in adults. It is challenging because the numbers are low, and people always want to be cautious with children, but I think it is important that children are able to have new therapies available to them. I think that prospective studies are the next step, so following these patients for longer periods of time going forward in future studies. Most of the studies we have done to date are retrospective, which is when somebody at 1 time point looks back over the medical records where they might lose a lot of information and they do not tend to have a lot of follow-up. Prospective studies are really important for these groups of patients, and that is going to require collaborative groups because the number [of patients] is so low. To make valuable insights, it is going to take international, collaborative trials, especially in the field of pediatrics where the number of children with these diagnoses are low compared to older adults.

We need to get people together, both in the United States and abroad, to track these patients. It is a big challenge to do that. I know there are some people and groups that are interested in that, so hopefully, that is something that can develop over the next year or so to really think about how we can put all the patient data together to learn from it.

TARGETED ONCOLOGY: Is there anything else you would like to highlight?

Kucine: