Managing MPNs

Managing Myelofibrosis (MF)

The following key factors will help your specialist to work out your best treatment options:

  • The number of abnormal red blood cells and white blood cells
  • The number of blast cells in the blood (blast cells are cells which are in the early stages of development and do not yet carry out any function)
  • Your age
  • Certain genetic changes
  • Symptoms such as fever, night sweats or weight loss
  • Health complications such as spleen and liver enlargement, gastrointestinal bleeding, infections and malnutrition.

What are your options?

Watch and wait

If you have no symptoms when you are first diagnosed with MF, your specialist may suggest a “watch and wait” approach. This usually involves regular check-ups and blood counts, as well as your specialist advising you on ways to live a healthy lifestyle. If your symptoms develop or the disease progresses, you talk to your doctor about what options would be right for you.

Managing an enlarged spleen

An enlarged spleen is a common symptom of MF, often leading to pain, discomfort and a feeling of fullness or a loss of appetite. Treatment options include:

  • Splenectomy (surgical removal of the spleen): This is considered if your enlarged spleen is painful and causing complications or if you have an increased need for blood transfusions. While it is generally safe, as with any surgery, splenectomy carries the potential risk of complications, including bleeding, blood clots, infection, injury to nearby organs, including the stomach, pancreas and colon.
  • Radiotherapy: Radiotherapy or radiation of the spleen is an option if splenectomy is ruled out. Performed in hospital, radiotherapy helps to reduce the size of the spleen. It usually provides temporary relief that lasts for between three and six months. The way radiation is now delivered is very precise and advancements in how it is delivered have minimized side effects of the procedure. In some patients white blood cell, platelet and red blood cell counts can drop, and this is one side effect is to watch for.
  • Jakavi® (ruxolitinib): is a drug option for the treatment of an enlarged spleen and/or associated symptoms in adult patients with MF. Jakavi® can cause blood-related side effects such as anemia (low levels of red blood cells), thrombocytopenia (low platelet levels in the blood) and neutropenia (low level of white blood cells). The most common other side effects in studies were bruising, dizziness, headache and fever.

Managing your anemia

Anemia is common in people with MF. Symptoms include excessive tiredness, weakness and shortness of breath. If these symptoms are causing you difficulties, your specialist may suggest a blood transfusion.
Blood transfusions are usually done in an out-patient clinic and involves the transfer of red blood cells from a compatible donor into your body. This can increase red blood cell count and quickly reduce symptoms of anemia, often within 24 hours. Blood transfusions are a relatively safe procedure and don’t usually cause serious complications, however there is an increased risk of iron overload if you receive a series of transfusions. This usually happens over a number of years, or after a total of 10-20 transfusions have been given. Feeling hot, cold, shivery or in any way unwell during or after the procedure might be a sign that you are having an adverse reaction.

Managing thrombocythemia
Agrylin® (anagrelide) is a drug option for lowering platelet counts in people who have thrombocythemia (too many platelets in the blood). In some cases it can cause serious side effects including trouble breathing or shortness of breath, chest pain, palpitations and swelling in the feet and ankles. Most common side effects are headache, diarrhea, weakness, nausea, abdominal pain and dizziness

Bone marrow or stem cell transplant

A bone marrow or stem cell transplant offers the only curative treatment for MF. Patients are individually assessed as transplants are not commonly recommended. This is due to a high risk of life-threatening side effects, as well as a risk that the new stem cells will react against your body’s healthy tissues. Transplants are often only considered an option for fit patients with advanced disease. The first step involves very high levels of chemotherapy or radiation therapy.

The decision to begin any treatment should take place only after the risks and benefits are discussed with your treating physician.

Managing Polycythemia Vera (PV)

Management of PV is aimed at reducing the number of cells in your blood to control symptoms and reduce the risk of complications.

It is vital to monitor and control your hematocrit level as this is an important indicator of the severity of your condition. Keeping your hematocrit levels below 45% and normalizing your white blood cell and platelet counts can help to reduce the risk of major thrombosis and cardiovascular complications.

The effective management of your PV can also help alleviate some of the symptoms that these blood problems can cause. Avoiding situations that make symptoms worse can also help – for instance, hot baths can make itching worse.

Reducing the hematocrit

  • Phlebotomy: If you have no symptoms when you are diagnosed but your hematocrit levels (the percentage of red blood cells in your blood) are too high, your doctor may recommend using a procedure called phlebotomy to help reduce the volume percentage of red blood cells in your blood. Phlebotomy is a simple procedure that involves the removal of some of your blood to help reduce your hematocrit level. The procedure is similar to donating blood and carries the same risks.
  • Jakavi® (ruxolitinib): An option that controls the hematocrit in adult PV patients who are unable to use or do not have their hematocrit controlled with a cytoreductive agent. Jakavi® can cause blood-related side effects such as anemia (low levels of red blood cells), thrombocytopenia (low platelet levels in the blood) and neutropenia (low levels of white blood cells). The most common other side effects in studies were bruising, diarrhea, muscle spasms and dizziness.

Managing thrombocythemia
Agrylin® (anagrelide) is a drug option for lowering platelet counts in people who have thrombocythemia (too many platelets in the blood). In some cases it can cause serious side effects including trouble breathing or shortness of breath, chest pain, palpitations and swelling in the feet and ankles. Most common side effects are headache, diarrhea, weakness, nausea, abdominal pain and dizziness.

The decision to begin any treatment should take place only after the risks and benefits are discussed with your treating physician.

What you can expect

Your individual situation and health history, as well as the ways you respond to treatment, can all affect your prognosis. Your specialist will be able to provide you with a more accurate picture.

In general, patients with PV who do not progress to myelofibrosis (MF) or leukemia can expect to have a normal to slightly reduced life expectancy if properly monitored and treated as necessary.

In some patients the disease may remain stable or gradually progress over time. This means that you may never experience many of the symptoms and outcomes that you read about – or if you do, it may not be for many years. Many people with PV live normal lives for years without experiencing any complications.However, even if you are experiencing no symptoms, it is still important that your hematocrit level is controlled in order to reduce the risk of serious problems such as major thrombosis and cardiovascular complications.

In a minority of cases, PV can progress into myelofibrosis (MF) or acute myeloid leukemia (AML).Myelofibrosis is also an MPN and it occurs when the bone marrow is replaced by scar tissue, which affects the bone marrow’s ability to produce normal blood cells.AML is a malignant disease that develops when there are too many immature blood-forming cells in the blood and bone marrow, which in turn affects the development of your infection-fighting white blood cells.If you have any concerns, talk to your specialist.

Managing Essential Thrombocythemia (ET)

Management of ET is aimed at managing symptoms and reducing complications, to help improve quality of life.

If you have no symptoms when you are first diagnosed, a “watch and wait” approach may be recommended if your platelet counts are at an acceptable level and if you are not in any other high-risk groups for developing blood clots. Your doctor may advise you on ways to live a healthy lifestyle, including giving you help to stop smoking.

Some ET patients may require various therapies, based on the symptoms and the results of routine monitoring by a physician. Treatment choice is generally based on a variety of risk factors including age, history of thrombotic events, and drug tolerance.

Reducing the risk of blood clots

Lowering blood platelet counts in people who have thrombocythemia (too many platelets in the blood) can reduce the risk of clotting and improve symptoms, including serious complications related to blockages in blood vessels and bleeding.

Managing thrombocythemia
Agrylin® (anagrelide) is a drug option for lowering platelet counts in people who have thrombocythemia (too many platelets in the blood). In some cases it can cause serious side effects including trouble breathing or shortness of breath, chest pain, palpitations and swelling in the feet and ankles. Most common side effects are headache, diarrhea, weakness, nausea, abdominal pain and dizziness.

Your treatment plan will depend on your own unique situation and take into account the symptoms you are experiencing as well as any lifestyle risk factors like high blood pressure and whether or not you smoke.

The decision to begin treatment should take place only after the risks and benefits are discussed with your treating physician.

What you can expect

Your individual situation and health history, as well as the ways you respond to treatment, can all affect your prognosis (the predicted outcome of the disease). Your hematologist will be able to provide you with a more accurate picture.

In general, patients with ET without severe clotting or bleeding complications have an excellent chance of living out a near-normal life span if properly monitored.

In some patients the disease may remain stable or gradually progress over time. This means that you may never experience many of the symptoms and outcomes that you read about – or if you do, it may not be for many years. Many people with ET live normal lives for years at a time without experiencing complications.

In a minority of cases, ET can progress into myelofibrosis (MF) or acute myeloid leukaemia (AML). MF is also an MPN and it occurs when the bone marrow is replaced by scar tissue, which affects the bone marrow’s ability to produce normal blood cells. AML is a malignant disease that develops when there are too many immature blood-forming cells in the blood and bone marrow, which in turn affects the development of your infection-fighting white blood cells. If you have any concerns, talk to your specialist.