Recombinant Human Erythropoietin Is Effective in Cancer-Related Anaemia, but Is it Cost Effective?

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Various Parameters Improved...

rhEPO is a recombinant form of the hormone erythropoietin, a hormone which increases endogenous red blood cell production. rhEPO produces higher haematocrit values and haemoglobin levels than transfusion alone in patients with cancer-related anaemia. This drug also significantly reduces or eliminates the need for transfusion.[1] For example, in a randomised trial of 121 patients with transfusion-dependent anaemia associated with haematological malignancy, 58 to 64% of those who received epoetin beta 2000 to 10 000 IU/day subcutaneously for 6 months required transfusions compared with 82% of those in the control group (p < 0.05) during months 2 to 6.[6]

In addition, rhEPO prevents the occurrence of anaemia in many nonanaemic patients with cancer who are receiving chemotherapy. In these patients, rhEPO also increases haemoglobin levels and reduces or eliminates the need for transfusion.[1] In one of the more recent trials 130 nonanaemic patients treated with platinum-based chemotherapy were randomised to receive no further treatment or epoetin alfa 150 or 300 IU/kg three times a week.[7] Anaemia, defined as haemoglobin <10 g/dl, developed in 48 and 39% of patients receiving the lower and higher dosages of rhEPO, respectively, compared with 66% of patients in the untreated group. The respective transfusions rates were 45, 20 and 59%. All differences between rhEPO and no further treatment were statistically significant.

Although rhEPO can be administered subcutaneously or intravenously for other indications, it is approved for subcutaneous administration in the management of anaemia associated with cancer/chemotherapy,[5] and therefore can potentially be self administered.[8]

Two large studies in patients receiving chemotherapy have evaluated the effects of epoetin alfa on mean energy, activity and overall quality of life.[2,9] One of the studies used a Functional Assessment of Cancer Therapy-Anaemia (FACT-An) questionnaire and linear analogue scale assessment.[2] In general, results of the studies showed significant improvements from baseline in these parameters when assessed 4 months after starting therapy.[2,9]

Limited data on the effect of transfusion on quality of life indicate that improvements in ratings of strength, dyspnoea and overall sense of well-being are significantly improved after transfusion, but the effect is not sustained beyond 2 weeks.[10]

rhEPO is effective in about 60% of patients with cancer-related anaemia, although in some studies in patients with solid tumour the rate has been as high as 80%.[1] Response to rhEPO is defined as ≥2g/dl increase in haemoglobin level, ≥6% increase in haematocrit, and/or no requirement for blood transfusion.

Patients respond to rhEPO after several weeks. In contrast, anaemia symptoms improve almost immediately with transfusion.[11] Therefore, rhEPO is not appropriate for patients with severe or life-threatening anaemia. However, since there may be reluctance in the use of transfusion by some physicians and patients (because of associated risks) until more severe anaemia develops, it may be useful to target rhEPO for use in patients at risk of developing more severe anaemia.