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Myeloproliferative neoplasms

Current status: Treatable, not yet curable

A group of slow-growing diseases in which the bone marrow makes too many red blood cells, white blood cells or platelets. Too many red blood cells make the blood thick, and extra blood cells can make clots form, which can raise the risk of stroke or heart attack. For polycythaemia vera, treatment can include removing some blood, daily low-dose aspirin, or medicines such as hydroxycarbamide that lower the number of red blood cells. Polycythaemia vera, one of the main types, usually starts later in life, on average at 60.

Read more about myeloproliferative neoplasms
Where research stands todayUpdated 15 Nov 2026

Trial-stage treatments and research studies you can join

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Get notified of new trialsPoland, all cities, all phases
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Phase
  1. Trial-stage treatmentPhase 3Taking people
    Tests pelabresib with ruxolitinib in adults with myelofibrosis

    Tests pelabresib plus ruxolitinib in adults with myelofibrosis not yet treated with JAK inhibitors.

    Gdansk, PolandAlso Katowice, Lublin, Skorzewo, Torun, WarsawAge 18+
  2. Trial-stage treatmentPhase 3Soon in Poland
    Tests INCA033989 Against Standard Care in Essential Thrombocythemia

    Tests INCA033989 against standard treatment in adults with essential thrombocythemia and a CALR mutation.

    Gdansk, PolandAlso Katowice, Krakow, Lodz, Wałbrzych, WarsawAge 18+
  3. Trial-stage treatmentPhase 4Taking people
    Long-term asciminib treatment for people with chronic myeloid leukemia

    Tests asciminib long term in people with chronic myeloid leukemia who finished an earlier study.

    Katowice, PolandAlso Krakow, Warsaw, WroclawAge 7 to 100
  4. Trial-stage treatmentPhase 3Soon in Poland
    Tests elritercept against placebo for anemia in myelofibrosis

    Tests elritercept against placebo in adults with myelofibrosis and anemia taking ruxolitinib.

    Chorzów, PolandAlso Katowice, Lodz, Poznan, Torun, WarsawAge 18+
  5. Trial-stage treatmentPhase 3Taking people
    Tests sapablursen with standard care in polycythemia vera

    Tests sapablursen added to usual care in adults with polycythemia vera.

    Gdansk, PolandAlso Katowice, Lodz, Lublin, Skorzewo, TorunAge 18+
  6. Trial-stage treatmentPhase 1/2Soon in Poland
    Tests oral nuvisertib in people with myelofibrosis

    Tests oral nuvisertib, its safety and how the body handles it, in adults with myelofibrosis.

    Lublin, PolandAge 18+
  7. Trial-stage treatmentPhase 2Taking people
    Continued RVU120 Treatment for Patients From an Earlier Study

    Gives continued RVU120 treatment to adults from an earlier RVU120 study.

    Chorzow, PolandAlso Gdansk, Katowice, Lublin, WroclawAge 18+
  8. Trial-stage treatmentPhase 2
    Tests bezuclastinib in adults with advanced systemic mastocytosis

    Tests bezuclastinib in adults with advanced systemic mastocytosis, a rare blood disease.

    Lublin, PolandAge 18+
  9. Trial-stage treatmentPhase 3
    Compares givinostat with hydroxyurea in high-risk polycythemia vera

    Compares givinostat and hydroxyurea in adults with high-risk polycythemia vera.

    Poznan, PolandAlso WroclawAge 18+
  10. Trial-stage treatmentPhase 2Taking people
    Tests selinexor in people with myelofibrosis and low platelet counts

    Tests selinexor in adults with myelofibrosis who have not had JAK inhibitor treatment.

    Lublin, PolandAlso Skorzewo, TorunAge 18+
  11. Trial-stage treatmentPhase 3Taking people
    Tests navtemadlin added to ruxolitinib in myelofibrosis patients with a weak response

    Tests navtemadlin with ruxolitinib in adults with myelofibrosis responding poorly to ruxolitinib.

    Bialystok, PolandAlso Gdansk, Krakow, Lodz, Nowy Sącz, Olsztyn, Poznan, Słupsk, Szczecin, …Age 18+
  12. Trial-stage treatmentPhase 1/2Taking people
    Tests TL-895 with ruxolitinib in adults with myelofibrosis

    Tests TL-895 combined with ruxolitinib in adults with myelofibrosis, a bone marrow disease.

    Katowice, PolandAge 18+
  13. Trial-stage treatmentPhase 2
    Tests selinexor alone in people with myelofibrosis and low platelet counts

    Tests selinexor alone in adults with myelofibrosis who have not had JAK inhibitors.

    Lublin, PolandAlso TorunAge 18+
  14. Trial-stage treatmentPhase 3Taking people
    Testing Navtemadlin Added to Ruxolitinib for Myelofibrosis Patients

    Tests navtemadlin with ruxolitinib in adults with myelofibrosis responding poorly to ruxolitinib.

    Bydgoszcz, PolandAlso Gdansk, Katowice, Lodz, Olsztyn, Skorzewo, Torun, Warsaw, WroclawAge 18+
  15. Research studyTaking people
    Observational Study of Ropeginterferon Alfa-2b in Adults With Polycythemia Vera

    Follows adults with polycythemia vera treated with ropeginterferon alfa-2b in routine clinical care.

    Gdansk, PolandAlso Katowice, Krakow, Lodz, WroclawAge 18 to 100
  16. Research studyTaking people
    Following people with myelofibrosis over time in Europe

    Observes adults with myelofibrosis across European centres through regular follow-up visits.

    Krakow, PolandAlso WroclawAge 18 to 100

Where treatment stands

One EU-listed medicine, momelotinib, got its first EU authorisation in January 2024.

Approved: 12

Officially approved for this disease in the EU, the US, Switzerland or France.

Human testing stage: 14

Being tested in people. Many treatments at this stage are never approved.

Animal testing stage: 2

Worked in animals in a study published in the last 12 months. Most treatments at this stage never reach people.

Words used here

Placebo: a dummy treatment without the medicine, used for comparison.

Phase 1, 2, 3: the steps of testing in people. Phase 1 checks safety, phase 3 is the last step before approval.

Where this comes from

The EU medicines agency (EMA) and its list of medicines in the EU, US prescribing labels (DailyMed, openFDA), the Swiss medicines register (Swissmedic), the French medicines database (ANSM) and the Health Canada drug database, checked on 11 November 2026. Every medicine links to its own official text.

A computer made this list from the official texts: it kept only medicines whose own approval text names the disease, wrote "Who it is for" from that text and checked it against the text a second time. People have not reviewed it yet.

The trials: ClinicalTrials.gov and the EU trials register, as copied into our trial database, checked on 12 November 2026. Every treatment in trials links to its own trial records; the texts are written by AI from them and checked against them by a second AI. People have not reviewed them yet. Placebo, standard medicines used for comparison and procedures are left out.

Phase 3 trials are listed from every country; only some phase 2 trials are listed, so 66 other open phase 2 trials are not listed.

Animal and lab research: studies listed in PubMed and published in the 12 months before 12 November 2026, found by a search for myeloproliferative neoplasms. AI chose the treatments from the study summaries and wrote the texts; a second AI checked each one against its study summary. People have not reviewed them yet. Treatments already in the approved or human testing lists are left out. A result in animals or cells does not show that a treatment works in people.

Not medical advice. Talk to your doctor before you change any treatment.

The approval records under a medicine come from Drugs@FDA and the FDA labels (openFDA) and from the EU medicines agency’s medicine data, with its orphan designations, checked on 13 Nov 2026. A date is the medicine’s first approval, for any use; the quoted text is its approved use as written today.

Support organisations

Patient organisations and helplines in Poland, for you and your family.

Myeloproliferative neoplasms organisations in Poland

  • Polska Liga Walki z Rakiem

    Polish cancer league (ECL member) building cooperation between health care, education, science and social policy to fight cancer and support patients.

  • Stowarzyszenie Wspierania Osób z Nowotworami Mieloproliferacyjnymi "MPN Polska"

    Polish association supporting people with myeloproliferative neoplasms. Member of MPNAN.

General support in Poland

  • Centrum Wsparcia dla osób dorosłych w kryzysie psychicznym

    Free 24/7 support line (800 70 2222) and chat for adults in psychological crisis in Poland, funded by the Ministry of Health.

  • pacjent.gov.pl

    Patient portal of the Polish Ministry of Health and National Health Fund with reliable, verified information on health and the care system.

  • Rzecznik Praw Pacjenta

    Poland's central government Patient Ombudsman protecting patient rights, with a free patient helpline (800 190 590).

International organisations

  • MPN Advocacy & Education International

    Nonprofit providing education and advocacy for people living with myeloproliferative neoplasms.

  • MPN Research Foundation

    Foundation funding research into myeloproliferative neoplasms and supporting the MPN patient community.

Patient organisation umbrella bodies

Offer tissue or data

There are three ways to help research move faster.

Give tissue or bloodA biobank stores your sample so many research teams can use it.No open programme yet
Share your health dataA patient registry collects your health data, without your name, for research.How to join
Take part as a healthy volunteerStudies also need people without the disease, for comparison. Family and friends can help.Volunteer

Approved treatments

13 medicines are approved for myeloproliferative neoplasms in at least one of the places we checked. An approval in one place does not mean the medicine is offered or paid for where you live.

Checked on 11 Nov 2026 in the official lists of the US (FDA), the EU (EMA), Japan (PMDA, approvals since April 2004), Switzerland (Swissmedic), France (ANSM, medicines approved in France only) and Taiwan (TFDA). For Canada (Health Canada) and Singapore (HSA) we checked only whether a medicine is sold there, not what for. 1 uncertain match is left out. Other countries' own approvals were not checked.

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About myeloproliferative neoplasms

Sources: NCI PDQ, NHS, Cancer Research UK, Blood

A group of slow-growing diseases in which the bone marrow makes too many red blood cells, white blood cells or platelets. Too many red blood cells make the blood thick, and extra blood cells can make clots form, which can raise the risk of stroke or heart attack. For polycythaemia vera, treatment can include removing some blood, daily low-dose aspirin, or medicines such as hydroxycarbamide that lower the number of red blood cells. Polycythaemia vera, one of the main types, usually starts later in life, on average at 60.

What is it?

Myeloproliferative neoplasms (MPNs) are a group of diseases in which the bone marrow makes too many red blood cells, white blood cells or platelets. Usually one kind of cell is affected most. The main types are polycythaemia vera (too many red cells), essential thrombocythaemia (too many platelets) and primary myelofibrosis (the marrow becomes scarred). Chronic myeloid leukaemia also belongs to this group but is described on its own page.

Many people have a change in a gene such as JAK2, CALR or MPL in their marrow cells. In polycythaemia vera, a JAK2 change usually causes the disease, and it is not usually inherited. Polycythaemia vera mostly starts later in life: the average age at diagnosis is 60.

The first signs

Some people have no signs at first, for example with polycythaemia vera, essential thrombocythaemia or myelofibrosis.

  • headaches, dizziness, or changes in vision;
  • itching, especially after a warm bath or shower;
  • a red face, or red, warm, burning or tingling hands and feet;
  • fullness or pain under the left ribs;
  • tiredness, night sweats, or losing weight without trying.

How doctors find out

A blood test counts your blood cells. Your family doctor may then send you to a blood specialist (haematologist). More blood tests look for the changed JAK2, CALR or MPL gene, and a bone marrow sample, taken with a needle, is checked in a lab.

Current treatment

Treatment depends on the type: for polycythaemia vera, removing blood, low-dose aspirin and hydroxyurea; for essential thrombocythaemia, hydroxyurea or interferon; for myelofibrosis, ruxolitinib or a donor stem cell transplant for some people.

Will it get worse?

Usually slowly, and some cases, particularly polycythaemia vera, need long-term treatment. People with polycythaemia vera may live slightly less long than average, because of the higher risk of heart attack and stroke. Over time, polycythaemia vera can lead to scarring of the marrow (myelofibrosis), and sometimes an MPN can turn into acute leukaemia. In myelofibrosis, how it goes depends on age, blood counts, immature cells in the blood, chromosome changes and symptoms such as fever or weight loss.

Medicines: what helps, and what to watch for

Treatment aims to prevent symptoms and problems such as blood clots. In polycythaemia vera, blood is removed regularly (venesection), about half a litre at a time, like giving blood: often weekly at first, then less often. Low-dose aspirin helps prevent clots. Medicines such as hydroxycarbamide or interferon can lower the number of red blood cells. Ruxolitinib, a targeted medicine, can ease symptoms such as tiredness, night sweats and weight loss in myelofibrosis, and is used in polycythaemia vera if chemotherapy does not work. Some people under 60 with essential thrombocythaemia and no symptoms only need regular checks.

Good to know:

  • Interferon is an injection 1 to 3 times a week that you can learn to give yourself, and it can be used in pregnancy; it can cause flu-like symptoms and hair loss;
  • In myelofibrosis, anaemia may need blood transfusions or other medicines.

A healthy lifestyle helps too

Healthy lifestyle changes can lower the risk of blood clots. Being overweight or smoking raises the risk, so losing weight and stopping smoking can help.

Surgery, radiotherapy or a transplant, for some people

In myelofibrosis, an enlarged spleen can be removed with surgery or treated with radiotherapy. For some people with myelofibrosis, a stem cell or bone marrow transplant from a donor is an option: strong chemotherapy is followed by healthy blood-forming cells.

Is there a cure?

Treatment usually aims to control the symptoms, not to cure the condition. A bone marrow or stem cell transplant from a donor aims to cure it. It is not suitable for everyone, because it is an intensive treatment with many risks.

See the 238 trials you can join

Median survival

About 20 years in essential thrombocythemia, 13.5 years in polycythemia vera and 6 years in primary myelofibrosis

Measured in: Patients with essential thrombocythemia, polycythemia vera or primary myelofibrosis from the Mayo Clinic (826 patients)

The figures behind it

Mayo Clinic patients (826, 58% followed until death): median survival 19.8 years in essential thrombocythemia (292 patients), 13.5 years in polycythemia vera (267) and 5.9 years in primary myelofibrosis (267). In primary myelofibrosis (428 Mayo and Italian patients) the median was 16 years with a CALR mutation and 2.3 years in triple-negative patients.