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.
There are limited guidelines available for the management of MPN in children.
Treatment of Polycythemia Vera may involve phlebotomy, aspirin, cytoreductive therapy, or a combination of these. Recommendations can be found in https://www.ncbi.nlm.nih.gov/pubmed/22262773
Treatment of Essential thrombocytosis may involve monitoring, aspirin, or cytoreductive therapy. Our groups recommendations can be found athttps://www.ncbi.nlm.nih.gov/pubmed/24688110
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 essential and should include aspirate and biopsy, assessment of bone marrow iron stores, and evaluation for fibrosis
- Erythropoietin levels should be done in children with thrombocytosis to help distingusih between PV and ET
- vonWillebrand (vW) testing in children with thrombocytosis should be performed because of the potential for acquired vW disease
2) Management practices
- Not every child with an MPN requires therapy - we often monitor our asymptomatic patients without intervention
- Severe bleeding or thrombotic events are usually indications to start cytoreductive therapy
- Hydroxyurea and Interferon are both appropriate options for children with MPN
- When treating children with ET, we often do not aim for a normal platelet count, but rather normalization of the symptom (resolution of headache, normalization of vW levels, etc.)
- If treating due to thrombosis, then normalization of counts is more appropriate