STEP 4: Treatment

All patients should receive supportive therapies, including education, active surveillance and monitoring, transfusions as appropriate and prompt infection control. Some lower risk MDS patients (not on any active interventions) may be suitable for monitoring and surveillance with their GP. The range of disease- specific/directed additional treatment options varies across different risk groups depending on age and comorbidities.

Establish intent of treatment

  • Curative
  • Anti-cancer therapy to improve quality of life and/or longevity without expectation of cure
  • Symptom palliation.

Supportive therapies will be required by all MDS patients. It may be the only long-term treatment needed for those with lower risk disease or those who are older, unfit or don’t respond to other treatments. This can include red blood cell transfusions, platelet transfusions or tranexamic acid, infection management and iron chelation therapy.

Systemic therapy options may vary for lower or higher risk MDS patients. In patients with lower risk MDS, available treatment options include lenalidomide, hypomethylating agents and

immunosuppressive therapy. Anaemia is the most common cytopenia for lower risk MDS and erythropoiesis-stimulating agents may be used as first-line therapy.

In patients with higher risk MDS, standard treatment options include hypomethylating agents (azacitidine and decitabine), AML induction chemotherapy (in those with a high blast count who are eligible for intensive therapy) and haematopoietic stem cell transplantation.

Allogeneic stem cell transplantation (allo-SCT) is the only potentially curative treatment for MDS. Patients with higher risk MDS may benefit from allo-SCT close to the time of diagnosis. Depending on the patient’s goals of therapy, consider proceeding to transplantation as soon as feasible after an optimal donor is found.

Palliative care

Early referral to palliative care can improve quality of life and in some cases survival. Referral should be based on need, not prognosis. For more information, visit the Palliative Care Australia website <>.


The lead clinician and team’s responsibilities include:

  • discussing treatment options with the patient and/or carer including the intent of treatment as well as risks and benefits
  • discussing advance care planning with the patient and/or carer where appropriate
  • communicating the treatment plan to the patient’s GP
  • helping patients to find appropriate support for exercise programs where appropriate to improve treatment outcomes.


  • Intent, risk and benefits of treatment discussed with the patient and/or carer
  • Treatment plan discussed with the patient and/or carer and provided to GP
  • Supportive care needs assessed and referrals to allied health services actioned as required
  • Early referral to palliative care considered and advance care planning discussed with the patient and/or carer



For symptomatic patients with higher risk disease, disease- specific therapy should begin within the first 6 weeks of initial specialist consultation.


Potential stem cell transplantation candidates should be referred to a bone marrow transplant specialist as soon as transplantation is considered a potential option.


For lower risk MDS, the timing of treatment is guided by clinical presentation and urgency.