3.3 Effect of erythropoiesis- stimulating agents and iron

3.3.6 Myelodysplastic syndrome

Evidence Statements for Myelodysplastic syndrome

Evidence Statements – myelodysplastic syndrome
ES3.35	In anaemic patients with MDS, the effect of ESAs on mortality is uncertain.	X		X
ES3.36	In anaemic patients with MDS receiving GM- CSF, ESAs may reduce transfusion incidence compared with no ESAs.	X	NA
ES3.37	In anaemic patients with MDS, the effect of ESAs on thromboembolic events is uncertain.	X		NA
ES3.38	In anaemic patients with MDS, the effect of ESAs on functional or performance status is uncertain.	X	NA	NA

ES, evidence statement; ESA, erythropoiesis-stimulating agent; GM-CSF, granulocyte/macrophage colony-stimulating factor; MDS, myelodysplastic syndrome

=A; =B; =C; X=D; NA,not applicable (see Table 2.1)

Erythropoiesis-stimulating agents – myelodysplastic syndrome

The review identified three RCTs (Level II)^125-127 evaluating the use of ESAs in patients with myelodysplastic syndrome (MDS).

MDS is a heterogeneous group of disorders characterised by varying degrees of dyserythropoiesis and marrow infiltration by abnormal haemopoietic cells. There is RCT (Level II) evidence of a favourable impact on mortality among patients with refractory anaemia with ringed sideroblasts (RARS), but not in other MDS subgroups treated with ESAs.¹²⁵

Fewer RBC transfusions were required among patients with MDS when ESAs were added to granulocyte/macrophage colony-stimulating factor (GM-CSF) therapy, provided the baseline endogenous erythropoietin level was <500 mU/ml.¹²⁶ No significant differences in the incidence of thromboembolic complications, including stroke, were seen. Patients in whom an erythroid response had been seen at 4 months showed improvements in physical, emotional and functional well-being, fatigue and overall quality of life.¹²⁵