Efficacy of growth factors compared to other therapies for low-risk myelodysplastic syndromes

Abstract

The myelodysplastic syndromes (MDS) represent a heterogeneous group of disorders. Low-risk MDS represent a subgroup with a relatively good prognosis, but with few trials evaluating outcomes. A pooled analysis based upon a MEDLINE search identified 162 original articles describing patient characteristics and effect of therapy on 2592 individuals with pathologically confirmed refractory anaemia or refractory anaemia with ringed sideroblasts with <5% bone marrow blasts. Treatments were categorised as growth factors (GF) or non-growth factors (NGF). International Prognostic Scoring System (IPSS) score was documented or calculated when possible. Responses and outcomes were standardised according to the International Working Group MDS criteria. Growth factors produced higher overall response rates (39.5% vs. 31.4% for NGF, P = 0.019), while NGF yielded better CR/PR rates (25.6% vs. 9.1% for GF, P = 0.03). Over 2 years of follow-up, those receiving GF demonstrated greater overall and progression-free survival than NGF, after controlling for baseline patient characteristics. Decision tools need to be developed to determine which therapy to choose for patients with low-risk MDS.