Growth Factors

Following the Food and Drug Administration (FDA) approval of recombinant human erythropoietin (rHuEPO) therapy for treating anemia in hemodialysis patients, rHuEPO was given to patients with anemia, including those with MDS, in a variety of clinical trials.

These studies are summarized in Table 40.1. A metaanalysis of 17 early, small studies in anemic MDS patients revealed an overall erythroid response rate of 16%, generally at rHuEPO doses >150 U/kg three times per week.11 Predictors for a response (a rise in hemoglobin >2.0 g/dL or becoming PRBC transfusion independent) included a low serum-erythropoietin level, nontransfusion-dependent anemia, and a diagnosis of refractory anemia (RA) or refractory anemia with excess blasts (RAEB). Patients with low erythropoi-etin levels and few or no transfusion requirements had a response rate >50%. High doses of erythropoietin are

Larger trials of rHuEPO (alone or with rHuG-CSF or rHuGM-CSF) to treat anemia in MDS patients


Trial design

Number of patients

Overall erythroid response rate

Erythroid response Major Minor

Response correlates

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