As one of the only comprehensive centers for children with MPN in the nation, we have experience in both diagnosing and treating children with MPN. We are available to provide guidance and support to both pediatric hematologists and primary care providers. We are actively pursuing research aimed at identifying the causes and outcomes in pediatric MPN, and hope to contribute to improved management of these children.
Practice Recommendations
There are limited guidelines available for the management of MPN in children. A consensus statement from adult and pediatric MPN providers is shared in this article:
https://pubmed.ncbi.nlm.nih.gov/40038897/
Some key points we follow for diagnosis and management in our young patients with MPN:
1) Diagnostic work-up
- Secondary causes of polycythemia or thrombocytosis should be ruled out
- Bone marrow evaluation is important and should include aspirate and biopsy, assessment of bone marrow iron stores, and evaluation for fibrosis
- vonWillebrand (vW) testing in children with extreme thrombocytosis should be performed because of the potential for acquired vW disease
2) Management practices
- Not every child with an MPN requires therapy - it is a balance of risks and benefits for each patient
- Severe bleeding or thrombotic events are usually indications to start cytoreductive therapy
- Discussing the potential of interferon to provide disease modifying therapy for young patients is appropriate
- Interferon and hydroxyurea are both appropriate options for children with MPNs who need cytoreduction