# Treatment options for myelodysplastic syndrome
How your MDS is treated depends on several factors: your risk score (based on cytogenetic abnormalities, blast percentage and other characteristics), your general health, your age and what symptoms you have. In recent years, new medicines and combinations have been added, and the treatment landscape continues to evolve. This overview describes the standard approaches per risk grade and the supportive care that is important in all phases.
Supportive treatments
Even without directly targeting MDS cells, supportive measures are crucial for quality of life and sometimes for delaying progression.
**Blood transfusions**
ProveniIncluded in official guidelines, or approved by EMA or FDA
When your hemoglobin drops too low (usually below 7–8 g/dL), red cell concentrates can be given. This helps combat fatigue and prevents heart problems from lack of oxygen. With low platelet counts (usually below 10,000–20,000), platelet transfusions can be given to prevent bleeding. Transfusions offer direct, short-term relief, but repeated transfusions can lead to iron accumulation over time (see iron chelation below). According to recent research, transfusion dependency is an independent predictor of mortality in MDS patients, regardless of risk score.
**Iron chelation**
ProveniIncluded in official guidelines, or approved by EMA or FDA
People who receive many red cell concentrates accumulate iron in the heart, liver and hormone glands. Iron-chelating agents bind this iron so it can be removed from the body. These are given by injection or orally and can cause side effects such as abdominal complaints, nausea and sometimes kidney or hearing problems. Regular monitoring of liver and kidney function is necessary.
**Antibiotic prophylaxis**
ProveniIncluded in official guidelines, or approved by EMA or FDA
(selective)
People with severely low white blood cell counts (neutropenia) are at risk of serious infections. In certain situations — especially around chemotherapy or stem cell transplantation — this is prevented with antibiotics. This does not happen routinely in all MDS patients, but only if neutropenia is severe and infection risk is high.
**Growth factors (G-CSF)**
ResearchediPositive results in clinical studies, not yet standard treatment
Agents that stimulate the production of white blood cells (granulocyte colony-stimulating factors) can reduce infection risk. They are mainly used when neutropenia is severe. The evidence that they improve disease progression or survival is limited, so they are used cautiously and usually for short periods.
---
Low-risk MDS
In low-risk MDS, there are few abnormalities in bone marrow cells and the percentage of blasts grows slowly. Many patients go years without further treatment, although symptoms such as anemia may require attention.
**Low-dose chemotherapy (hypomethylating agents)**
ProveniIncluded in official guidelines, or approved by EMA or FDA
Azacitidine and decitabine — agents that affect DNA methylation — are given in low doses (much lower than in acute leukaemia). They can inhibit cell division and sometimes keep the blast percentage lower. They are given in cycles, for example intravenously or as subcutaneous injections. Side effects include blood cell decreases, infections, nausea and fatigue. They slow progression in some patients, but do not cure the disease.
**Lenalidomide**
ProveniIncluded in official guidelines, or approved by EMA or FDA
This immunomodulatory agent is mainly given in MDS with isolated 5q deletion (a specific chromosomal abnormality). It can improve anaemia and can provide years without further progression. Side effects include thrombosis (blood clots), loss of sensation in hands and feet, and increased infection risk. Regular monitoring is necessary.
**Lutetium-177-PSMA-617 and other targeted agents**
ResearchediPositive results in clinical studies, not yet standard treatment
Newer medicines target specific genetic abnormalities in MDS cells (for example, mutations in SF3B1). Some are in the research phase and are being tested in clinical trials. Laboratory tests (sequencing of your MDS cells) can show which mutations you have, which can provide insight into which targeted medicine would be most suitable.
---
Intermediate-risk MDS
At intermediate risk, the blast percentage increases more rapidly. Treatment is usually needed to slow progression.
**Hypomethylating agents (azacitidine, decitabine)**
ProveniIncluded in official guidelines, or approved by EMA or FDA
These are the cornerstone of treatment at this risk level. They are given according to fixed cycles (usually monthly or regularly), just as with low-risk, but used more frequently. They can extend the time to progression to acute leukemia. In approximately 40–50% of patients, some improvement occurs. Here too, cytopenias (low blood cell counts), infections and gastrointestinal side effects are possible.
**Venetoclax in combination**
ResearchediPositive results in clinical studies, not yet standard treatment
(in combination with hypomethylating agents)
Venetoclax is an agent that can cause certain cancer cells to die. In combination with azacitidine, it is being studied for intermediate- to high-risk MDS. This may potentially increase response rates. Side effects include severe cytopenias and infections. Recent research (2026) is looking at venetoclax also in the maintenance phase after stem cell transplantation.
**Physiological preparation for stem cell transplantation**
ProveniIncluded in official guidelines, or approved by EMA or FDA
For some patients, an allogeneic (from donor bone marrow) stem cell transplantation is prepared. This is an invasive treatment; not everyone is eligible (age, overall health and availability of a suitable donor play a role). Preparation is done with chemotherapy and/or radiation to make room for donor cells. We set this apart because it is a core treatment for severely progressive MDS.
---
High-risk MDS (and MDS in transition to acute leukemia)
At high risk — many cytogenetic abnormalities, high blast percentage — rapid progression is likely and acute leukemia may develop.
**Hypomethylating agents (first choice)**
ProveniIncluded in official guidelines, or approved by EMA or FDA
Azacitidine and decitabine are the standard treatment for many patients who are not suitable for stem cell transplantation. They can cause remission (normalization of blood cell counts) in approximately 30–40% of patients and can stabilize the disease. Duration and effect vary greatly from person to person. The same side effects as mentioned earlier.
**Venetoclax with azacitidine**
ResearchediPositive results in clinical studies, not yet standard treatment
The combination of venetoclax and azacitidine has been studied and shows higher complete remission rates than azacitidine alone in high-risk MDS. This is mentioned as an option in some guidelines, especially for patients not eligible for stem cell transplantation. Side effects can be significant (severe infections and cytopenias).
**Allogeneic stem cell transplantation**
ProveniIncluded in official guidelines, or approved by EMA or FDA
This is the only potentially curative treatment. The donor bone marrow cells engraft and can displace the MDS cells. Preparation is done with chemotherapy and/or radiation; intensive preparation is appropriate for younger, fitter patients, while older or less fit patients receive less intensive preparation. Major risks are rejection (graft failure) and graft-versus-host disease (GVHD), where donor cells attack the recipient's body. Supportive care after transplantation is intensive. Survival after transplantation depends on many factors: donor matching, age, disease stage and comorbidity. Recent research (2026) is investigating new preparation schedules (low-dose melphalan with thiotepa and fludarabine) and maintenance combinations (e.g. venetoclax) after transplantation.
**Kinase inhibitors**
ResearchediPositive results in clinical studies, not yet standard treatment
New selective kinase inhibitors (e.g. pacritinib, targeting CSF1R, IRAK1, JAK2 and FLT3) are being tested in MDS. They can suppress inflammatory pathways and inhibit cell division in certain MDS types. These are still in research phases; side effects and effectiveness are currently being determined.
**CAR-T and engineered immunotherapy**
ExperimentaliOngoing in study setting, outcome still unknown
Modified T-cells (CAR-T) and other immune-engineered cells (e.g. NK-cells) are being used in research stages against MDS. This occurs in clinical trials with strict selection criteria. Side effects can be serious (cytokine release syndrome, immune activation).
---
Molecularly-guided approaches (emerging)
ResearchediPositive results in clinical studies, not yet standard treatment
/ **Experimental**
As more genetic characteristics of MDS become known, opportunities arise to target specific mutations. Examples:
- **SF3B1 mutations**: Small studies suggest that certain drugs (currently in research phase) are more effective when this mutation is present.
- **TP53 mutations**: Very difficult type of MDS with poor prognosis. New combinations are being investigated, especially after stem cell transplantation (hypomethylating agents as maintenance).
This is still largely research territory; genetic testing can be done at specialist centres, but is not yet routine everywhere.
---
Supportive preparation for all phases
Regardless of which active treatment is chosen:
- **Psychological support**: Counselling helps to cope with the diagnosis.
- **Diet and nutrition**: Healthy eating can support energy levels and overall health.
- **Physical activity**: Moderate exercise, to the extent you feel well enough.
- **Infection prevention**: Hand hygiene, caution in busy environments with low immune function.
---
_This information never replaces a doctor's judgment. Always discuss your situation with your own healthcare provider._