Current use of recombinant human erythropoietin (r-huEPO) in the management of symptomatic anaemia in patients with myelodysplastic syndromes (MDS).

PURPOSE

To evaluate the efficacy of subcutaneous erythropoietin (r-huEPO) in patients with myelodysplastic syndrome (MDS) requiring periodical blood transfusion.

PATIENTS AND METHODS

The study was performed on 9 MDS patients distributed as follows: 4 with refractory anaemia, 2 with refractory anaemia with ringed sideroblasts, 2 with chronic myelomonocytic leukaemia and 1 with refractory anaemia with excess blasts in transformation. All the patients had been diagnosed at least three months earlier and had a mean monthly transfusion requirement of 3.5 blood units (range, 2-5.3). Baseline serum EPO levels were assessed by ELISA, subcutaneous r-huEPO was given at a doses of 10,000 IU three times a week. A positive response was defined as increase of haemoglobin rates in 1 g/dL or decrease of transfusion requirements in 50%. A negative response was registered when the haemoglobin rates or transfusion requirements were maintained in the patient. The results were evaluated 4,6 and 12 weeks after the beginning of the treatment.

RESULTS

Response to treatment was seen in five patients (56%), who showed increased haemoglobin rates and in four of them (45%) no transfusions were needed since the onset of this therapy. The positive response to r-huEPO appeared within the 4th week of treatment. The four unresponsive patients (44%) showed no changes in spite of prolonged treatment. Positive responses correlated with increased red-cell progenitors in the bone marrow and decreased myelo/erythroid ratio. No severe untoward effects were seen with this treatment, and mild local pain at the sites of injection was alleviated by warming the r-huEPO solution before administration.

CONCLUSIONS

MDS with symptomatic anaemia can be initially treated with r-huEPO for 4 weeks, although it seems advisable to maintain this treatment in respondents until the maximal response has been attained. On the contrary, when no response is seen in that period treatment must be discontinued.