The MDS Beacon™

Immunosuppressant agents may increase responsiveness of anemic, low-risk myelodysplastic syndromes (MDS) patients to treatment with erythropoietin (EPO). Additionally, immunosuppressant agents may decrease transfusion dependency, according to a study published in Haematologica.

Anemia is common in low-risk MDS and is usually treated with transfusions, but MDS patients who are transfusion dependent typically have poor survival. EPO is a hormone used to treat anemia in MDS by promoting the production of red blood cells. EPO and other growth factors have been shown in previous studies to be successful in approximately one third of patients.

Researchers concluded that although the study was small, consisting of only 10 patients, the rate of response was good and the treatment was well-tolerated.

The study examined the use of immunosuppressants followed by treatment with EPO to increase red blood cell counts. The treatment regimen was tested in low-risk MDS patients who were resistant to EPO treatments and patients who had a low probability of response to EPO alone.

In the study, 10 patients were given daily, oral doses of the immunosuppressant agents CellCept (mycophenolate mofetil) and prednisone.

The treatment response of the patients was evaluated at 12 weeks.

For patients who still showed no red blood cell response at evaluation, the growth factor EPO was administered via subcutaneous injection for six weeks. If no response was observed, the EPO dose was increased.

Of the 10 patients treated, eight had received EPO before but had become nonresponsive and seven were receiving transfusions. One patient was hospitalized for pneumonia and removed from the study.

Of the remaining nine patients in the study, five experienced a response. Three of the five responsive patients experienced a major response, while the other two patients experienced a minor response.

Researchers reported that the treatment was well tolerated, with the most common side effect during the study being low-grade diarrhea. Also during the study, one patient who was diabetic required an increase in diabetes treatment and another patient was hospitalized for pneumonia.

The researchers highlighted that the patients included in the study had few alternative treatment options, with most of them being nonresponsive to EPO.

Researchers concluded that the study results were positive, but recommended a larger study to corroborate the findings.

For more information, please see the full study in the journal Haematologica. For more information on EPO treatment for anemia in MDS, please see previous related Beacon articles.