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Myelodysplastic syndromes

Current status: Treatable, not yet curable

A group of blood cancers in which the bone marrow makes blood cells that do not grow into healthy ones. Too few healthy blood cells can lead to infection, anaemia or easy bleeding. Treatment ranges from blood transfusions and medicines to strong chemotherapy with a stem cell transplant, which can cure some people. It is rare and more likely after age 60, or after chemotherapy or radiotherapy for another cancer.

Read more about myelodysplastic syndromes
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
Your ageShows only trials that take people your age.
Phase
  1. Trial-stage treatmentPhase 2Soon in Poland
    Bexmarilimab With Azacitidine for Higher-Risk Myelodysplastic Syndromes

    Tests bexmarilimab with azacitidine in adults newly diagnosed with higher-risk myelodysplastic syndromes.

    Katowice, PolandAlso Skórzewo, WarsawAge 18+
  2. Trial-stage treatmentPhase 3Taking people
    Tests elritercept in adults with myelodysplastic syndromes needing regular blood transfusions

    Tests elritercept against placebo in adults with low-risk myelodysplastic syndromes needing transfusions.

    Katowice, PolandAlso Lodz, Olsztyn, PoznanAge 18+
  3. Trial-stage treatmentPhase 3Taking people
    Elritercept Compared With Epoetin Alfa for Anemia in Lower Risk Myelodysplastic Syndromes

    Compares elritercept with epoetin alfa for anemia in adults with myelodysplastic syndrome needing transfusions.

    Katowice, PolandAlso Skorzewo, WarsawAge 18+
  4. Trial-stage treatmentPhase 1Taking people
    Continued Treatment and Safety Monitoring for People With Certain Cancers

    Follows people with blood cancers and solid tumors who continue study drugs long term.

    Gdansk, PolandAlso Katowice, SkorzewoAge 18+
  5. Trial-stage treatmentPhase 3Taking people
    Tests lisaftoclax with azacitidine for newly diagnosed higher-risk myelodysplastic syndrome

    Tests lisaftoclax plus azacitidine against placebo in adults with newly diagnosed higher-risk myelodysplastic syndrome.

    Gdansk, PolandAlso Legnica, Lodz, Olsztyn, WarsawAge 18+
  6. Trial-stage treatmentPhase 2/3Taking people
    Tests oral ASTX030 alone or with venetoclax in myeloid blood cancers

    Tests an oral form of azacitidine, alone or with venetoclax, in adults with blood cancers.

    Gdynia, PolandAlso Lodz, LublinAge 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 2Taking people
    Continued ASTX727 treatment for people from earlier studies with blood cancers

    Gives continued ASTX727 to adults with blood cancers who benefited from earlier studies.

    Gdansk, PolandAlso WroclawAge 18+
  10. Trial-stage treatmentPhase 2Taking people
    Testing momelotinib for anemia caused by low-risk myelodysplastic syndrome

    Tests momelotinib, at different doses, in adults with low-risk myelodysplastic syndrome and anemia.

    Katowice, PolandAlso WarsawAge 18+
  11. Trial-stage treatmentPhase 1Taking people
    Tests a pill for cancer in people with severe kidney problems

    Checks how the body handles oral decitabine and cedazuridine in adults with cancer.

    Wroclaw, PolandAge 18+
  12. Trial-stage treatmentPhase 1Taking people
    Tests an oral drug combination in cancer patients with liver problems

    Checks how the body handles oral decitabine and cedazuridine in adults with liver damage.

    Wroclaw, PolandAge 18+
  13. Trial-stage treatmentPhase 2Taking people
    Tests SP-420 for removing iron from organs in certain blood disorders

    Tests SP-420 to remove excess iron in adults with thalassemia or myelodysplastic syndrome.

    Gdansk, PolandAlso Olsztyn, Skorzewo, WarsawAge 18 to 64
  14. Trial-stage treatmentPhase 1/2
    Tests tuspetinib in adults with relapsed or refractory acute myeloid leukemia

    Tests tuspetinib, alone or with other drugs, in adults with acute myeloid leukemia.

    Gdansk, PolandAlso Gdynia, WarsawAge 18+
  15. Trial-stage treatmentPhase 4Soon in Poland
    Comparing two treatment intensities after leukemia or lymphoma shows no remaining disease

    Compares standard and reduced intensity follow-up treatment in adults with AML or CLL.

    Gdansk, PolandAlso Gliwice, Katowice, Lodz, Lublin, Warsaw, WroclawAge 18+

Where treatment stands

Some patients can be cured with strong chemotherapy and a stem cell transplant from a donor. For others, treatment may slow the disease from getting worse.

In the EU, imetelstat and efbemalenograstim alfa were first approved in 2025 and 2024, not always for this disease.

Approved: 13

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

Human testing stage: 11

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

Animal testing stage: 3

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 128 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 myelodysplastic syndromes. 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.

Myelodysplastic syndromes 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.

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

  • Aplastic Anemia and MDS International Foundation

    Foundation supporting patients and families with aplastic anemia, MDS and related bone marrow failure diseases.

  • DKMS

    Finds blood stem cell donors worldwide so that people with blood cancer can have a transplant.

  • MDS Foundation

    US-based global nonprofit supporting patients with MDS and related diseases such as AML, their families and providers, with education, resources and support.

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

17 medicines are approved for myelodysplastic syndromes 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. 6 uncertain matches are left out. Other countries' own approvals were not checked.

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About myelodysplastic syndromes

Sources: NCI PDQ, MedlinePlus, The Lancet Oncology

A group of blood cancers in which the bone marrow makes blood cells that do not grow into healthy ones. Too few healthy blood cells can lead to infection, anaemia or easy bleeding. Treatment ranges from blood transfusions and medicines to strong chemotherapy with a stem cell transplant, which can cure some people. It is rare and more likely after age 60, or after chemotherapy or radiotherapy for another cancer.

What is it?

Myelodysplastic syndromes (MDS) are a group of blood cancers. Normally, stem cells in the bone marrow grow into healthy red cells, white cells and platelets. In MDS many of them do not mature and die in the marrow. This leaves too few healthy blood cells, which can lead to anaemia (too few red cells), infections and bleeding.

MDS is rare and mostly affects older people: half are about 70 or older when they find out. In most people the cause is not known. The risk is higher after chemotherapy or radiotherapy for another cancer, and after contact with tobacco smoke, some chemicals (such as benzene, pesticides or fertilisers) or heavy metals such as lead and mercury.

The first signs

MDS often causes no early signs and is sometimes found by a routine blood test.

  • feeling weak or tired;
  • shortness of breath;
  • skin that is paler than usual;
  • bruising or bleeding easily, or tiny spots under the skin caused by bleeding;
  • fever or infections that keep coming back.

How doctors find out

Blood tests count the red blood cells, white blood cells and platelets. A bone marrow test takes a small sample of marrow with a needle, and a lab checks it for abnormal cells and chromosome changes. Some of these tests, such as checking the chromosomes, help plan treatment.

Current treatment

Treatment depends on several factors. It can include transfusions, medicines that help make red blood cells, and antibiotics for symptoms; lenalidomide or azacitidine and decitabine for some people; and chemotherapy with a donor stem cell transplant for certain patients.

Will it get worse?

Prognosis (chance of recovery) and treatment options depend on certain factors. How it goes depends on how many immature cells (blasts) are in the marrow, how many kinds of blood cells are low, certain chromosome changes, whether it followed earlier cancer treatment, and your age and general health. Some types of MDS can turn into acute myeloid leukaemia (AML).

Medicines: what helps, and what to watch for

Supportive care eases the problems caused by the disease. It can include red blood cell or platelet transfusions, medicines that help the body make more red blood cells, and antibiotics to fight infection. Azacitidine or decitabine can slow the change to AML. Lenalidomide helps some people with a certain chromosome change (del(5q)) need fewer transfusions. People at high risk of AML may get the same chemotherapy as for AML.

Good to know:

  • Many transfusions can build up too much iron in the body and harm organs; medicines can remove the extra iron;
  • You can join a clinical trial before, during or after treatment, and some trials take only people who have not started treatment.

A transplant, for some people

Some people can be cured with strong chemotherapy followed by a stem cell transplant from a donor, which gives them healthy blood-forming cells. It may not work as well when MDS was caused by earlier cancer treatment.

Is there a cure?

For some people, yes: strong chemotherapy with a donor stem cell transplant can cure certain patients. There is no standard treatment when MDS comes back or stops responding. Clinical trials are testing new treatments.

See the 397 trials you can join

Median survival

About 2 years with azacitidine, in higher-risk MDS

Measured in: Higher-risk myelodysplastic syndromes: a phase III trial of azacitidine against conventional care (358 patients)

The figures behind it

Phase III trial in higher-risk myelodysplastic syndromes (358 patients): median overall survival 24.5 months with azacitidine vs 15.0 months with conventional care regimens. Lower-risk MDS is not part of this figure.