FAQs

7. How will my MPN be treated?

Therapy for your MPN will be determined by your haematologist and their recommendation will take into account your age, comorbidities (other health issues) and the presence of disease-related complications. Treatment aims to reduce burdensome symptoms and the risk of life-threatening complications, and where possible to minimise the risk of progression to acute leukaemia. Cardiovascular risk factors should be aggressively managed for all patients, including smoking cessation. More detailed information on many of the treatment modalities below is available in the “Treatments” Section of this website.

(i) Polycythaemia vera

The management of PV is focused on controlling symptoms and reducing the risk of thrombotic and bleeding complications. The treatment, or combination of treatments, recommended for you by your haematologist will depend on several factors, including the duration and severity of your PV, whether you have a history of blood clots, your age and your general health.

Treatment for all patients includes low-dose aspirin and venesections to reduce the risk of vascular and thrombotic events. Aspirin reduces the risk of thrombotic complications by reducing the stickiness of platelets. If you are allergic or intolerant of aspirin an alternative anti-platelet medication is usually recommended.

Venesection (or phlebotomy) is a procedure where blood is removed from your bloodstream. This procedure is commonly used when PV is initially diagnosed because it rapidly reduces the high red cell count and the risk of thrombotic complications. Usually 450 ml of blood is removed from a large vein at the elbow bend by nursing staff at your local hospital. This procedure is repeated frequently until your haematocrit (proportion of blood made up of cells) is reduced to the desired level of <45% in both men and women.  While the evidence has only been tested at <45%, some MPN specialists prefer that haematocrit in women is <42% as women always have a lower haematocrit than men.  Venesections are repeated as required to maintain the haematocrit at the target level. For many people, particularly younger patients without any complications from their disease, regular venesections (every few months) may be all that is needed to control their disease for many years. PV patients who are aged >60 years or who have a prior history of thrombosis usually receive therapy with drugs to slow bone marrow production of blood cells (cytoreductive therapy) in addition to aspirin and venesections. Hydroxycarbamide (hydroxyurea) or interferon alpha are standard firstline therapies for PV patients requiring cytoreductive therapy. Hydroxycarbamide is well tolerated with infrequent side-effects which can include low blood counts, macrocytosis (increased red cell size), leg ulcers and rarely drug fever and hepatitis. Interferon alpha is also very well tolerated if started at a low dose. It is the first line therapy for younger people and also for women during pregnancy.

Busulfan (usually for older, high-risk patients), melphalan (infrequently used) or JAK inhibitors (not readily available in Australia for PV) may also be used in certain circumstances. If available, participation in clinical trials may also be considered.

(ii) Essential thrombocythaemia

The role of aspirin is less clear in low-risk ET patients outside of the presence of microvascular symptoms although most haematologists in Australia recommend its use except in patients with a very high platelet count (>1500 x 109/L) or with a bleeding tendency.

Cytoreductive therapy is used in high-risk ET patients to reduce the occurrence of thrombotic and haemorrhagic events. Frontline cytoreductive treatments may include hydroxycarbamide (hydroxyurea), or interferon alpha, both of which have been shown to lower thrombotic complications.

Anagrelide is a viable second-line therapy in ET although it is not currently PBS-funded in Australia. Other second-line options include busulfan or participation in clinical trials.

(iii) Primary myelofibrosis

The management of patients with myelofibrosis requires evaluation of the risk score using one of the prognostic scores (IPSS, DIPSS or DIPSS-Plus) and the symptom burden of the disease. Low-risk patients are often only observed with regularly monitoring of their blood count and symptom burden.

Patients with symptomatic anaemia may benefit from therapy with androgens (male hormones), low-dose corticosteroids, immunomodulatory agents or erythropoiesis-stimulating agents. Not all of these agents are readily available in Australia.

Cytoreductive treatments including hydroxycarbamide (hydroxyurea), cladribine, melphalan, and busulfan have been shown to be of only modest benefit for splenomegaly but may be used to control high white cell and platelet counts. Interferon alpha has also been increasingly used in recent years for blood count control and reducing splenomegaly.

Ruxolitinib is approved for patients with myelofibrosis (risk groups: intermediate-1 risk with severe symptoms, intermediate-2 risk or high-risk) and has been shown to reduce splenomegaly, improve symptoms, and prolong survival.

Patients with symptoms due to splenomegaly (enlarged spleen) who have failed ruxolitinib and other pharmacological therapies may occasionally be considered for radiotherapy or splenectomy used to control symptoms. There have been several clinical trials for myelofibrosis patients internationally testing a variety of  different drug types and combinations with promising results.  If available, participation in clinical trials may also be considered.

For some myelofibrosis patients with progressive or advanced disease a stem cell transplant using blood stem cells from a family member or unrelated donor may be considered.