The MDS Beacon™

Recombinant erythropoietin (r-EPO) without granulyte-colony stimulating factor (G-CSF) may lead to longer survival times in myelodysplastic syndromes (MDS) patients according to an analysis conducted by Italian researchers.

Recombinant erythropoietin alpha is an erythroid stimulating agent that stimulates red blood cell production. It is beneficial for MDS patients with anemia (low red blood cell count) and can reduce transfusion-dependence and improve quality of life. Recombinant means that the erythropoietin originates from an external organism and not from the patients’ own bodies.

Past studies reported patients benefited from r-EPO administered in combination with G-CSF. G-CSF is a growth factor that stimulates the production of white blood cells and red blood cell-producing stem cells. The analysis showed that r-EPO is effective when used without G-CSF.

Researchers analyzed the responses and survival times of 192 transfusion-dependent MDS patients.  Eighty-three patients received r-EPO in doses of either 10,000 units three times per week or 40,000 units once a week for at least 12 weeks, while the other 109 patients were not treated with r-EPO.  All patients were followed for at least five years after diagnosis, or until death.

The median length of treatment was 19 months for responders, compared to 14 months for patients who received r-EPO but did not respond.

Researchers found that median overall survival was not statistically different between patients who had received r-EPO (36 months) and those who had not received the treatment (38 months).

However, researchers observed differences in survival among the patients who had received r-EPO. Patients who responded to r-EPO had significantly longer survival times (52 months) than non-responders (31 months). The survival time of non-responders was similar to patients who never received r-EPO, according to researchers.

In addition, 28.9 percent of patients who received r-EPO experienced improved red blood cell production for a median of 17 months. 

Some patients had long-lasting responses that continued even after r-EPO treatment was stopped, evidence that r-EPO can produce a prolonged improvement in red blood cell production in some patients. 

The study suggests there are certain MDS patients who respond to r-EPO treatment while otherwise similar patients do not. This analysis did not specify if the responsive MDS patients had a naturally better prognosis which led to their response, or if r-EPO modified their disease, producing a response.

The authors are currently conducting further related research using r-EPO and another erythroid stimulating agent called darbepoetin.  They are observing long-term effects on recently diagnosed MDS patients who are being treated with high doses of both drugs.

For more information, please see the study in the journal Leukemia Research (abstract).