Myeloproliferative neoplasms
A group of slow-growing diseases in which the bone marrow makes too many red blood cells, white blood cells or platelets.1 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.1 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.2 Polycythaemia vera, one of the main types, usually starts later in life, on average at 60.1
Read more about Myeloproliferative neoplasmsTrial-stage treatments and research studies you can join
Filters
- Tests pacritinib in people with myelodysplastic syndromes
Tests the capsule pacritinib in people 12 or older with myelodysplastic syndromes.
Bethesda, United StatesAge 12 to 120 - Tests LY5830966 in adults with myelofibrosis after a prior JAK2 inhibitor
Tests a new pill, LY5830966, in adults with myelofibrosis, a bone marrow disease.
Ann Arbor, United StatesAlso Boston, Charlotte, Cincinnati, Columbus, Houston, Kansas City, New York, Palo Alto, …Age 18+ - Tests imetelstat in people with myelodysplastic and myeloproliferative diseases
Tests imetelstat, a drug, in adults with blood disorders called MDS/MPN.
Houston, United StatesAge 18+ - Testing dexrazoxane to protect the heart during chemotherapy for blood cancers
Tests dexrazoxane to reduce heart side effects of chemotherapy in people with blood cancers.
Houston, United StatesAge 12+ - Long-term bomedemstat use in people with blood disorders who took it before
Follows people with essential thrombocythemia or polycythemia vera who continue bomedemstat.
Ann Arbor, United StatesAlso Columbus, Durham, PittsburghAge 18+ - Tests pelabresib with ruxolitinib in adults with myelofibrosis
Tests pelabresib plus ruxolitinib in adults with myelofibrosis not yet treated with JAK inhibitors.
Albany, United StatesAlso Atlanta, Austin, Berkeley Heights, Bethesda, Chicago, Columbus, Dallas, Denver, …Age 18+ - 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.
Ann Arbor, United StatesAlso Cincinnati, Dallas, Houston, Milwaukee, New York, Portland, Salt Lake City, St LouisAge 7 to 100 - Testing New Treatment Combinations for Newly Diagnosed High-Risk Acute Myeloid Leukemia
Compares newer drug combinations with standard chemotherapy in adults with high-risk acute myeloid leukemia.
‘Aiea, United StatesAlso Albany, Albuquerque, Allentown, Anaconda, Ann Arbor, Appleton, Ashland, Augusta, …Age 18 to 59 - Axatilimab With or Without Azacitidine for Advanced Blood Cancers of the Bone Marrow
Tests axatilimab, alone or with azacitidine, in adults with advanced blood and bone marrow cancers.
Columbus, United StatesAlso PortlandAge 18+ - Tests elritercept against placebo for anemia in myelofibrosis
Tests elritercept against placebo in adults with myelofibrosis and anemia taking ruxolitinib.
Albuquerque, United StatesAlso Ann Arbor, Atlanta, Baytown, Billings, Boston, Chapel Hill, Charleston, Cleveland, …Age 18+ - Ropeginterferon alfa-2b for chronic myelomonocytic leukemia and related overlap blood cancers
Tests ropeginterferon alfa-2b in adults with chronic myelomonocytic leukemia or related blood cancers.
Los Angeles, United StatesAge 18+ - Tests INCA033989 Against Standard Care in Essential Thrombocythemia
Tests INCA033989 against standard treatment in adults with essential thrombocythemia and a CALR mutation.
Ann Arbor, United StatesAlso Baltimore, Baton Rouge, Birmingham, Boston, Chicago, Cleveland, Columbia, Dallas, …Age 18+ - Tests bomedemstat against usual treatments for polycythemia vera
Compares bomedemstat with usual treatments in adults with polycythemia vera not helped by hydroxyurea.
Charlottesville, United StatesAlso Eugene, NewarkAge 18+ - Tests JNJ-88549968 in people with CALR-mutated myeloproliferative neoplasms
Tests JNJ-88549968, a new antibody drug, in adults with CALR-mutated blood disorders.
Ann Arbor, United StatesAlso Atlanta, Aurora, Charlotte, Duarte, East Brunswick, Goodyear, Grand Rapids, Greenville, …Age 18+ - Testing Momelotinib with Luspatercept in Transfusion-Dependent Myelofibrosis
Tests momelotinib with luspatercept in adults with myelofibrosis who need regular blood transfusions.
Ann Arbor, United StatesAlso Houston, Nashville, New York, SeattleAge 18+ - Tests stopping ropeginterferon treatment in polycythemia vera with a JAK2 change
Tests whether stopping ropeginterferon treatment is practical for adults with JAK2-mutant polycythemia vera.
Basking Ridge, United StatesAlso Commack, East White Plains, Middletown, Montvale, New York, UniondaleAge 18+ - Tests natural killer cell therapy after cord blood transplant in blood cancers
Tests natural killer cells after chemotherapy and cord blood transplant in people with leukemia.
Houston, United StatesAge 15 to 80 - Imetelstat Maintenance After Stem Cell Transplant for High-Risk Blood Cancers
Tests whether imetelstat is safe in adults with high-risk myeloid cancers after transplant.
Madison, United StatesAge 18 to 80 - Tests INCA036978 for safety in people with myeloproliferative neoplasms
Tests INCA036978, alone or with standard therapy, in adults with myeloproliferative neoplasms.
Atlanta, United StatesAlso Baltimore, Birmingham, Boston, Chapel Hill, Charlotte, Cleveland, Columbus, Durham, …Age 18+ - Tests a two-step ATG dosing plan to prevent graft versus host disease after transplant
Tests a two-step ATG dosing plan in adults with blood cancers after stem cell transplant.
Birmingham, United StatesAge 18 to 75
Where treatment stands
Where research stands now
On 11 November 2026, 237 trials and studies of myeloproliferative neoplasms were open. At that time, 256 treatments were being tested in people; 42 had reached phase 3, the last, largest trials before approval, and 131 phase 2. From 5 November 2025 to 11 November 2026, 35 articles reported treatment studies in people, 15 in animals and 3 in the lab. On 11 November 2026, 9 medicines had US approval and 7 had EU approval for this disease. One EU-listed medicine, momelotinib, got its first EU authorisation in January 2024.
We found 12 medicines approved for myeloproliferative neoplasms in the EU, the US, Switzerland or France, and 14 being tested in people.
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.
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.
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.
Support organisations
Patient organisations and helplines in United States, for you and your family.
Myeloproliferative neoplasms organisations in United States
- National Cancer Institute – Cancer Information Service (CIS)
NCI's contact center: information specialists answer cancer questions from patients, families and the public by phone, chat and email.
- American Cancer Society
Nationwide US nonprofit helping people who face cancer: research, patient services, early detection, treatment information and education, with a cancer helpline on its site.
- Cactus Cancer Society
Young adult cancer resource and community: support programs for people diagnosed with cancer as young adults.
- Cancer Hope Network
Nonprofit providing free one-on-one support to cancer patients and families by matching them with trained volunteers who have had a similar experience.
- Cancer Nation
Survivor-led US cancer advocacy organization (formerly the National Coalition for Cancer Survivorship) working on quality cancer care and survivorship resources.
- Genetic and Rare Diseases Information Center
NIH information center (GARD) providing reliable information on genetic and rare diseases to patients and families via website and contact center.
- Imerman Angels
Nonprofit offering free one-on-one cancer support and personalized mentoring that connects cancer fighters, survivors and caregivers with someone who has been there.
- MPN Advocacy & Education International
Nonprofit providing education and advocacy for people living with myeloproliferative neoplasms.
- MPN Cancer Connection
Non-profit charity for people with myeloproliferative neoplasms (PV, ET, MF), offering patient guides, an MPN specialist finder, news and advocacy resources.
- MPN Education Foundation
Volunteer-run nonprofit for people with myeloproliferative neoplasms, offering disease information, the MPN-NET online support group, and conferences.
- MPN Research Foundation
Foundation funding research into myeloproliferative neoplasms and supporting the MPN patient community.
- Triage Cancer
National nonprofit providing free education on legal and practical issues (insurance, employment, finances) for people with cancer and caregivers.
General support in United States
- 988 Suicide & Crisis Lifeline
US national 24/7 free and confidential lifeline for anyone in suicidal crisis or emotional distress, reachable by calling or texting 988 or by chat.
- MedlinePlus
Free, easy-to-read health information service of the US National Library of Medicine (NIH) on diseases, conditions, drugs and wellness.
International organisations
Offer tissue or data
There are three ways to help research move faster.
Treatments approved outside the EU
Medicines for Myeloproliferative neoplasms approved in the US or Japan that are not approved in the EU.
Checked on 11 Nov 2026 in the official lists of the US (FDA), Japan (PMDA, approvals since April 2004) and the EU (EMA). 1 uncertain match is left out.
Your health insurer may pay part of the cost, sometimes all of it. This depends on your insurer, your plan and the country, so always ask your insurer before you go. For planned care in the EU, the EEA and Switzerland, ask about form S2.
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About Myeloproliferative neoplasms
A group of slow-growing diseases in which the bone marrow makes too many red blood cells, white blood cells or platelets.1 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.1 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.2 Polycythaemia vera, one of the main types, usually starts later in life, on average at 60.1
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.1 Usually one kind of cell is affected most.1 The main types are polycythaemia vera (too many red cells), essential thrombocythaemia (too many platelets) and primary myelofibrosis (the marrow becomes scarred).1 Chronic myeloid leukaemia also belongs to this group but is described on its own page.1
Many people have a change in a gene such as JAK2, CALR or MPL in their marrow cells.1 In polycythaemia vera, a JAK2 change usually causes the disease, and it is not usually inherited.2 Polycythaemia vera mostly starts later in life: the average age at diagnosis is 60.2
The first signs
Some people have no signs at first, for example with polycythaemia vera, essential thrombocythaemia or myelofibrosis.1
How doctors find out
A blood test counts your blood cells.1,2 Your family doctor may then send you to a blood specialist (haematologist).2 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.1
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.Source: NCI PDQ
Will it get worse?
Usually slowly, and some cases, particularly polycythaemia vera, need long-term treatment.1,2 People with polycythaemia vera may live slightly less long than average, because of the higher risk of heart attack and stroke.2 Over time, polycythaemia vera can lead to scarring of the marrow (myelofibrosis), and sometimes an MPN can turn into acute leukaemia.2,1 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.3,1
Medicines: what helps, and what to watch for
Treatment aims to prevent symptoms and problems such as blood clots.2,1 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.2 Low-dose aspirin helps prevent clots.2,1 Medicines such as hydroxycarbamide or interferon can lower the number of red blood cells.2 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.3,1 Some people under 60 with essential thrombocythaemia and no symptoms only need regular checks.1
Good to know:
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.2
Surgery, radiotherapy or a transplant, for some people
In myelofibrosis, an enlarged spleen can be removed with surgery or treated with radiotherapy.1 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.1
Is there a cure?
Treatment usually aims to control the symptoms, not to cure the condition.4 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.4
Median survival
About 20 years in essential thrombocythemia, 13.5 years in polycythemia vera and 6 years in primary myelofibrosisSource: Blood, 2014
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.
Sources
Written in plain words by Frontlight from these pages: