Aberrant immunophenotype of blasts in myelodysplastic syndromes is a clinically relevant biomarker in predicting response to growth factor treatment

作者：Westers Theresia M; Alhan Canan; Chamuleau Martine E D; van der Vorst Maurice J D L; Eeltink Corien; Ossenkoppele Gert J; van de Loosdrecht Arjan A^*

来源：Blood, 2010, 115(9): 1779-1784.

DOI：10.1182/blood-2009-08-239749

Myelodysplastic syndromes (MDS) are a group of clonal disorders of the bone marrow characterized by peripheral cytopenias. Standard treatment in low-and intermediate-I-risk MDS is supportive therapy consisting of regular transfusions and growth factors, that is, erythropoietin (Epo) and granulocyte-colony-stimulating factor (G-CSF). Because flow cytometric analysis of MDS bone marrow samples can identify clinically relevant subgroups regarding transfusion dependency and disease progression, we ad-dressed the question whether flow cytometry (FCM) was instrumental in predicting response. In 46 patients with low-and intermediate-I-risk MDS that were treated with Epo/G-CSF, low Epo level and low transfusion need were associated with response to Epo/G-CSF. Interestingly, aberrant phenotype of myeloblasts identified nonresponders among patients with the greatest response probability according to the predictive model of Hellstrom-Lindberg et al. Moreover, aberrant FCM of myeloblasts acted as a significant biomarker for treatment failure in multivariate analysis. A new predictive model based on the basis FCM combined with previously validated Epo levels is proposed defining 3 subgroups with 94%, 17%, and 11% response probability. In conclusion, FCM may add significantly to well-known predictive parameters in selecting MDS patients eligible for Epo/G-CSF treatment. This is of relevance regarding prevention of treatment failure. (Blood. 2010;115:1779-1784)

• 出版日期2010-3-4