Myeloproliferative neoplasm medical therapy

Myeloproliferative Neoplasm Microchapters
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Editor-In-Chief: C. Michael Gibson, M.S., M.D. [1] Associate Editor(s)-in-Chief: Mohamad Alkateb, MBBCh [2] Shyam Patel [3]

Overview

The mainstay of therapy for myeloproliferative neoplasm is chemotherapy, aspirin, and palliative care. Treatment is directed at reducing the excessive numbers of blood cells.

Medical Therapy

Medical therapy for myeloproliferative neoplasm is based on the specific subtype of myeloproliferative neoplasm.

Polycythemia vera

Therapy	Mechanism of Action	Dosing	Adverse Effects
Aspirin	Irreversibly inhibits cyclooxygenase-1 and -2 (COX-1 and COX-2)	81mg PO daily	Mucosal bleeding Gastrointestinal bleeding
Hydroxyurea	Inhibits ribonucleotide reductase	20mg/kg PO daily	Anemia, thrombocytopenia, ulcerations, secondary cancers
Ruxolitinib	Inhibits JAK2 (tyrosine kinase inhibitor)	10mg PO twice daily	Weight gain, zoster, non-melanoma skin cancers, cytopenias
Phlebotomy	Mechanically removes red blood cells from circulation	Induction: 450cc blood removal daily until hematocrit < 45% Maintenance: One session every 2 months, with goal hematocrit < 45%	Iron deficiency anemia, fatigue, vasovagal episodes, pain at phlebotomy site

Essential thrombocythemia

Therapy	Mechanism of Action	Dosing	Adverse Effects
Anagrelide	Inhibits phosphodiesterase 3 (PDE-3) Inhibits release of arachidonic acid from phospholipase A2 Disrupts maturation of megakaryocytes	0.5mg PO every 6 hours or 1mg every 12 hours	Headache, palpitations, diarrhea, edema, nausea
Hydroxyurea	Inhibits ribonucleotide reductase	20mg/kg PO daily	Anemia, thrombocytopenia, ulcerations, secondary cancers
Aspirin	Irreversibly inhibits cyclooxygenase-1 and -2 (COX-1 and COX-2)	81mg PO twice daily	Mucosal bleeding Gastrointestinal bleeding
Ruxolitinib	Inhibits JAK2 (tyrosine kinase inhibitor)	10mg PO twice daily	Weight gain, zoster, non-melanoma skin cancers, cytopenias
Plateletpheresis	Mechanically removes platelets from circulation	Daily until platelet count returns to normal range	Hypotension, thrombocytopenia
Pegylated interferon alpha 2a	Immunomodulatory agent; anti-angiogenic agent	45mcg/week	Hypotension, infusion reaction

Primary myelofibrosis=

Therapy	Mechanism of Action	Dosing	Adverse Effects
Hydroxyurea	Inhibits ribonucleotide reductase	20mg/kg PO daily	Anemia, thrombocytopenia, ulcerations, secondary cancers
Ruxolitinib	Inhibits JAK2 (tyrosine kinase inhibitor)	10mg PO twice daily	Weight gain, zoster, non-melanoma skin cancers, cytopenias
Radiation therapy	Unknown mechanism	0.5 Gy to spleen 5 days weekly	Fatigue, secondary cancers, nausea, cytopenias