The MDS Beacon™

Aranesp (darbepoetin alfa, or DPO) relieves the symptoms of anemia for myelodysplastic syndromes (MDS) patients and improves patients’ quality of life, according to a recent study published in the journal Leukemia & Lymphoma. 

For the majority of low- or intermediate-1 risk MDS patients, anemia, or low red blood cell count, is the most common cause of symptoms that significantly alters their quality of life.  Although regular red blood cell transfusions can relieve symptoms and keep the body adequately supplied with blood, transfusions are associated with certain side effects, such as excess iron (see related Beacon article). Previous research has shown that MDS patients who are dependent on transfusions typically have significantly lower overall survival than those who do not require regular transfusions. 

Aranesp is a growth factor that stimulates red blood cell development.  As a type of supportive care, it does not stop or reverse MDS progression, but it is still an important treatment option. 

The study’s results showed that the majority of MDS patients had improved red blood cell development after receiving Aranesp.  In addition, their short-term quality of life improved, with better physical function, increased emotional well-being, and decreased fatigue.

In the study, 40 low-risk MDS patients received 150 µg Aranesp by subcutaneous injection once a week for 24 weeks.  Throughout the study period, the dose was increased to 300 µg for transfusion-independent patients who did not respond after four weeks or transfusion-dependent patients who did not improve after eight weeks.  After three months, 80 percent of MDS patients received the 300 µg dose.

Seventy-three percent of all patients responded to Aranesp during the 24 weeks, with the most significant responses occurring by 8 weeks of treatment.

Fifty-six percent of transfusion-independent MDS patients responded to the treatment for an average of 22 weeks.  Sixty-five percent responded after a median of six weeks.

Fifty-nine percent of patients receiving regular blood transfusions responded to Aranesp.  Transfusion dependence was defined as requiring at least two red blood cell transfusions every 16 weeks.  Although their response was shorter (average duration of 15.1 weeks), 82 percent achieved a response after a median of 8.4 weeks.  Almost 24 percent became transfusion-independent during the study period.

As there was no observed disease progression through the 24-week period, study authors concluded that Aranesp did not decrease survival.

Patients did not experience any significant side effects, such as blood clotting, after receiving Aranesp, wrote Dr. Esther Oliva, lead scientist in the study, in an e-mail to the MDS Beacon.  A few patients reported pain at the injection site.

In addition to measuring responses, researchers evaluated changes in patients’ quality of life.  The MDS patients experienced improved physical function, emotional well-being, and decreased fatigue as a result of anemia symptom relief.  Most improvements were observed in the first eight weeks of the study.

Quality of life increased more significantly for transfusion-independent patients than transfusion-dependent patients. Researchers speculated that this was likely due to the inferior prognosis for patients who require regular transfusions. 

In addition, transfusion-dependent patients needed to travel regularly to the treatment center to receive transfusions which may have produced a poorer perception of their illness. These study participants “may have been disturbed by dependence on hospital and staff and by the ‘waste’ of personal time,” added Dr. Oliva.

Improved quality of life was only sustained as long as improved blood cell production was observed.  The study authors speculated this was due to the study protocol requiring reduced Aranesp doses after patients reached the target goal of 12 g/dL hemoglobin.

The researchers recommend further studies investigate what doses and dosing schedules of Aranesp can produce sustained improvement for MDS patients with anemia, as well as how to treat higher-risk patients.

For more information, please see Leukemia & Lymphoma (abstract).