Myeloproliferative Disease Treatment & Management
- Author: Haleem J Rasool, MD, FACP; Chief Editor: Emmanuel C Besa, MD more...
Treatment of myeloproliferative neoplasms varies by disease subtype. For full discussion, see the following Medscape articles:
Treatment of Chronic Myelogenous Leukemia
Imatinib mesylate (Gleevec), a bcr-abl –specific tyrosine kinase inhibitor (TKI), is approved for use in Philadelphia chromosome–positive chronic myelogenous leukemia (CML) in chronic phase. In one study at 18 months, the complete response rate was 76.2% and the major cytogenetic response rate was 87.1%. Imatinib is also indicated for CML in blast crisis, accelerated phase, or in chronic phase after interferon alfa therapy failure. This is the treatment of choice for most patients.
Interferon alfa, usually administered as a subcutaneous daily injection in a dose of 5 million U, produces hematologic and molecular remissions in some patients with CML. In these patients, evidence shows that it prolongs survival. Several patients who achieved molecular remissions have survived for more than 10 years. Addition of low-dose cytosine arabinoside to interferon alfa has been reported to achieve higher remission rates. Patients with CML who are intolerant of interferon alfa therapy can be treated with hydroxyurea.
Dasatinib (Sprycel) is a TKI indicated for the treatment of adults patients with CML in chronic, accelerated, or myeloid or lymphoid blast phase who are resistant or intolerant to prior therapy including imatinib. Nilotinib (Tasigna) is a TKI indicated for the treatment of chronic phase and accelerated phase Philadelphia chromosome-positive CML in adult patients resistant to or intolerant to prior therapy including imatinib.
Hematopoietic stem cell transplantation can be considered in young patients with CML in chronic phase, if a human leukocyte antigen (HLA)-matched donor is available.
When the disease progresses to the blast phase (see the image below), it is treated as acute leukemia, though the outcome is usually grave.
Treatment of Polycythemia Vera
Treatment of polycythemia vera (PV) is palliative. Young (<40 y), asymptomatic patients with PV can be considered for therapeutic phlebotomies alone to maintain hematocrit level below 45%.
High-risk patients with systemic symptoms, history of thrombosis or bleeding, or high rate of phlebotomies or patients older than 69 years are best treated with myelosuppressive therapy in the form of hydroxyurea. In December 2014, the US Food and Drug Administration (FDA) expanded the approval of the JAK1/JAK2 inhibitor, ruxolitinib (Jakafi) for the treatment of PV in patients who have an inadequate response to or cannot tolerate hydroxyureaAn alternative therapy.
in older patients, radioactive phosphorus (32P) can be used for treatment of PV. However, this is unsuitable for younger patients because of the potential for causing secondary leukemia.
Treatment of Essential Thrombocythemia
Treatment of essential thrombocythemia (ET) is meant to relieve symptoms and to prevent complications because no curative modality is available at present. The aim of treatment is to maintain the platelet count within the reference range. This usually can be achieved with hydroxyurea or anagrelide.
Treatment of Primary Myelofibrosis
No curative treatment of primary myelofibrosis (MF) is available at the present time. Asymptomatic patients can be monitored clinically until symptoms develop. Treatment options for symptomatic MF include the following:
Anemia is treated with correction of reversible contributing factors, followed by erythropoiesis-stimulating agents, androgens, immunomodulating drugs, corticosteroids, and splenectomy; however, most patients eventually become transfusion dependent 
Hydroxyurea is useful to suppress the number of circulating cells
Patients with painful, massively enlarged spleens refractory to myelosuppressive therapy are occasionally treated with radiation therapy, but they may ultimately require splenectomy
Two case reports suggest that oral bisphosphonates may be beneficial in decreasing bone marrow fibrosis associated with this illness
Allogeneic hematopoietic stem cell transplantation is curative and may be an alternative for selected patients whose disease is refractory to conventional therapies 
In November 2011, the JAK1/JAK2 inhibitor, ruxolitinib (Jakafi), became the first US Food and Drug Administration (FDA)–approved drug for patients with intermediate- or high-risk myelofibrosis, including primary myelofibrosis, post-polycythemia vera myelofibrosis, and post-essential thrombocythemia myelofibrosis. Approval was expedited in accordance with the US Orphan Drug Act and based on US and international data from the COMFORT-1 and COMFORT-2 trials. Results from the COMFORT-1 trial showed patients (n=309) who received ruxolitinib had a significant reduction in spleen volume (at least 35%) at 24 weeks when assessed by MRI or CT compared with placebo (41.9% vs 0.7%).
See the list below:
Surgical consultation for permanent central venous access device placement may be required for patients in whom repeated blood draws, blood transfusions, and/or chemotherapy is anticipated.
A radiation oncologist may need to be involved in selected cases, when splenic radiation is considered appropriate.
Diet and Activity
Massive splenomegaly is usually associated with epigastric and left upper quadrant discomfort and early satiety. Patients with these symptoms are encouraged to eat frequent, small meals rather than 3 large meals.
Individuals with myeloproliferative diseases are not encouraged to restrict their daily activities, but they are encouraged to refrain from physical activities that might expose them to abdominal trauma because massively enlarged spleens are likely to rupture, sometimes in response to minimal trauma.
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|Chronic myelogenous leukemia||Chronic myelogenous leukemia, BCR/ABL1 positive|
|Polycythemia vera||Polycythemia vera|
|Essential thrombocythemia||Essential thrombocythemia|
|Agnogenic myeloid metaplasia/myelofibrosis||Primary myelofibrosis|
|...||Chronic neutrophilic leukemia, not otherwise specified|
|...||Myeloproliferative neoplasms, unclassifiable|