Pediatric Polycythemia Medication

Updated: Jun 13, 2023
  • Author: May C Chien, MD; Chief Editor: Lawrence C Wolfe, MD  more...
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Medication Summary

Current recommendations for treatment of young patients with polycythemia primarily rely on phlebotomy.


Antineoplastic agents

Class Summary

The following medications are not approved for pediatric polycythemia but are extrapolated from other pediatric treatment regimens, including leukemia and myelodysplastic syndrome.

Interferon alfa 2a and 2b (Roferon-A [alfa-2a], Intron A [alfa-2b])

A recombinant purified protein used IV for CML, hairy cell leukemia, and Kaposi sarcoma. Inhibits cellular growth and alters cell differentiation.

Chlorambucil (Leukeran)

Antineoplastic alkylating agent of nitrogen mustard type used for CLL, giant follicular lymphoma, Hodgkin lymphoma, and lymphosarcoma.

Hydroxyurea (Hydrea)

Inhibitor of deoxynucleotide synthesis. PO antineoplastic agent used in CML, melanoma, ovarian carcinoma, and some head and neck carcinomas.

Busulfan (Myleran)

Potent cytotoxic drug that, at recommended dosage, causes profound myelosuppression. As alkylating agent, mechanism of action of active metabolites may involve cross-linking of DNA, which may interfere with growth of normal and neoplastic cells.