From the Guidelines
Epogen (epoetin alfa) can be effective for some patients with anemia related to myelodysplastic syndrome (MDS), with a response rate of approximately 36.8% in patients with low or intermediate-1 risk and moderate to severe anemia. The effectiveness of Epogen in MDS patients has been evaluated in several studies, including a randomized controlled phase 3 trial that showed a significant response rate in patients treated with Epogen compared to placebo 1. The typical starting dose of Epogen is 30,000 to 60,000 units per week, administered as subcutaneous injections, with treatment response usually evaluated after 8-12 weeks.
Patient Selection
Patients most likely to benefit from Epogen have:
- Lower-risk MDS
- Serum erythropoietin levels below 500 mU/mL
- Lower transfusion requirements (less than 2 units of red blood cells per month) These patients should be considered for therapy with Epogen at an initial dose ranging from 30,000 to 60,000 IU per week, as recommended by the European LeukemiaNet 1.
Treatment Response and Side Effects
The medication works by stimulating red blood cell production in the bone marrow, compensating for the ineffective erythropoiesis characteristic of MDS. However, many patients will eventually become refractory to treatment over time. Common side effects include hypertension, headache, and potential increased risk of thrombotic events. For patients who don't respond to Epogen alone, adding granulocyte colony-stimulating factor (G-CSF) may improve response rates in some cases, as suggested by the European LeukemiaNet guidelines 1.
Alternative Treatments
Alternative treatments should be considered if no response is seen after 8-12 weeks of adequate dosing. Thrombopoiesis-stimulating agents, such as romiplostim and eltrombopag, have been tested in clinical trials, but their use is currently restricted to clinical trials due to concerns about safety and disease progression 1.
From the Research
Effectiveness of Epogen in Anemia of Mylodysplasia
- The effectiveness of Epogen (erythropoietin) in treating anemia associated with myelodysplastic syndrome (MDS) has been studied in various clinical trials and case reports 2, 3, 4, 5, 6.
- A meta-analysis of 205 patients from 17 studies found that 16% of patients with MDS showed a significant response to epo treatment, with patients having refractory anemia with ring sideroblasts (RAS) showing a lower response rate than other patients 5.
- Another study found that patients with no transfusion requirement and MDS other than RAS showed a response rate of > or = 50%, irrespective of their serum level of epo 5.
- A case report described a patient with low-risk MDS whose erythropoiesis-stimulating agent treatment was switched from darbepoetin α to epoetin β pegol (continuous erythropoietin receptor activator) to treat transfusion-dependent anemia, resulting in an increased hemoglobin concentration and no requirement for transfusion 2.
- However, other studies have found that only a minority of patients benefit from treatment with recombinant human erythropoietin, and that serum erythropoietin levels are extremely variable in anemia patients with MDS 6.
Predictive Factors of Response
- Baseline EPO levels <100 IU/L were found to be a predictive factor of response to darbepoetin alfa treatment in patients with low or intermediate-risk MDS 3.
- Patients with MDS other than RAS and no transfusion requirement were found to have a higher response rate to epo treatment 5.
- Serum erythropoietin levels were found to be significantly lower in responding patients, but this parameter alone could not be used to identify patients with a favorable response 5.
Safety and Tolerability
- Darbepoetin alfa was found to be well tolerated in patients with low or intermediate-risk MDS, with few adverse events reported 3.
- Epoetin β pegol (continuous erythropoietin receptor activator) was also found to be effective and safe in a case report, with no adverse events reported 2.
- However, other studies have found that erythropoietin treatment can affect cells of the myeloid and megakaryocytic lineage in a small proportion of patients, resulting in changes in non-erythroid cells 6.