Therapeutic targets
- Symptom relief
- Preservation and improvement of the quality of life
- Prolongation of survival time
Therapy recommendations
Therapy of low-risk myelodysplastic syndrome.
In the presence of low-grade cytopenia (decrease in cell count) and depending on age and comorbidities (concomitant diseases), it is sufficient to initially observe or wait (“watch and wait”) in these patients. Usually, a proven anemia is the trigger for starting therapy.
- Supportive therapy
- Transfusions (erythrocytes, platelets) symptom-oriented – see “Further therapy”
- For infections: early administration of broad-spectrum antibiotics (prophylactic administration is not recommended).
- Cave: do not give steroids and preferably no non-steroidal anti-inflammatory drugs (NSAIDs)!
- Use of growth factors – erythropoietin therapy (EPO therapy).
- To correct the misdirected hematopoiesis (blood formation).
- Indications:
- 5q anomaly (del(5q)) and/or.
- SEPO < 500 mU/mL and/or
- < 2 ECs (red blood cell concentrates) per month.
- Use of: rHuEpo (recombinant human erythropoietin) (40-60,000 U/wk. s. c.) or darbepoetin (500 µg every 2-3 weeks).
- In neutropenic febrile episodes (febrile episodes associated with a decrease in neutrophil granulocytes/belong to the white blood cell group in the blood): Granulocyte colony-stimulating factors (G-CSF).
- In thrombocytopenia (reduction in the number of platelets / platelets): thrombopoietin receptor agonists romiplostim and eltrombopag.
- Immunomodulatory therapy
- Indications:
- MDS patients in the “low” or “intermediate” risk group and 5q abnormality (del(5q)); and
- SEPO ≥ 500 mU/mL and
- < 2 ECs (red blood cell concentrates) per month.
- Use of: Lenalidomide (Red Hand Letter: new important note on reactivation of viral infections).
- The frequency of required transfusions may be reduced. It may also be that no further transfusions are needed.
- Other therapeutic measures have been found to be insufficiently effective or inappropriate for these patients.
- Indications:
- Immunosuppressive therapy
- Indications:
- Age < 60 years
- Blasts in bone marrow <5
- Normal cytogenetics
- Transfusion-dependent
- Use of: Antithymocyte globulin (ATG) or CsA.
- Indications:
Therapy of high-risk myelodysplastic syndrome.
- Chemotherapy – if no suitable stem cell donor available.
- To reduce the excess of blasts (young, not yet finally differentiated cells).
- Indications:
- Patients who are not eligible for stem cell transplantation (at intermediate risk II or high risk).
- As a bridge to allogeneic stem cell transplantation.
- Use of: 5-azacitidine (Dosing Instructions: As long as 5-azacitidine is effective and no serious toxicity (toxicity) occurs).
- Significant prolongation of the overall survival rate!
- Intensive polychemotherapy (AML induction protocols) – not an established treatment option for high-risk MDS patients!
- Indication:
- High-risk patients <70 years of age without comorbidities (concomitant diseases), taking into account the risk-benefit ratio.
- Approximately 60% full remissions
- Indication:
Other active ingredients
- Luspatercept (mode of action: Ligand trap: attracts and traps hormones that suppress erythropoiesis (process of formation and development of erythrocytes/red blood cells)): a Phase III study demonstrated that approximately 38% of participants taking luspatercept did not require a blood transfusion for at least 8 weeks (placebo group: 13%); 28% of patients taking the drug were even transfusion-free for more than 12 weeks (placebo group: 8%).
