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208 MANAGEMENT OF MYELODYSPLASTIC SYNDROMES WITH ERYTHROPOIESIS STIMULATING AGENTS: EVALUATION OF ERYTHROPOIETIC ASPECTS AND ANALYSIS OF RESPONSE
S104
Poster Presentations – 13th International Symposium on Myelodyspastic Syndromes / Leukemia Research 39 S1 (2015) S1–S166
Conclusions: Our study reveals that patients treated with AMLlike chemotherapy reach more responses and more infections. OS is better among patients who reach transplat and is similar in both subgroups, aza or chemo as cytorreductors.
Table 1.
208 MANAGEMENT OF MYELODYSPLASTIC SYNDROMES WITH ERYTHROPOIESIS STIMULATING AGENTS: EVALUATION OF ERYTHROPOIETIC ASPECTS AND ANALYSIS OF RESPONSE C. Cerchione1, O. Vitagliano1, L. Simeone1, A.E. Pareto1, I. Soriente2, A.M. D’arco2, P. Danise2, F. Alfinito1, F. Pane1 1 Hematology, University of Naples Federico II, Naples, Italy; 2 Hematology, Ospedale Umberto I, Nocera Inferiore (SA), Italy Erythropoiesis stimulating agents (ESAs) are the frontline treatment in low risk anemic MDS patients and an employment of this therapy in the earlier stage of the disease can delay the need for RBC transfusion, hypothetically by slowing the disease course. It is matter of debate whether the clinical response is a result of proliferation and maturation of the dysplastic clone or stimulation of residual normal erythropoiesis by ESAs. Macrocytosis is one of the cytological hallmarks of dyserithropoiesis in MDS: an analysis of the erythropoietic response to ESAs therapy in a cohort of anemic not trasfusion-dependent MDS patients, enrolled in a retrospective register, RECAMDS, subgroup of Italian MDS register, was performed. 137 patients, treated with standard-dose ESAs, have been retrospectively analyzed (Table 1). Data analysis was performed, according to IWG 2006 criteria, at the baseline, after three and six month of continuous treatment, with a subanalysis of the patients according to WHO and R-IPSS risk stratification (Figures 1 and 2). ESAs treatment was started at mean Hb concentration of 9.5 g/dl, mean serum EPO concentration:
41 mU/L, after a mean time from diagnosis of six months (r.1-118). Overall response rate (ORR) was 83% (114/137) and no difference among WHO and IPSS subgroups was found: 76% achieved response after three months of treatment, while other 7% after six months. 2 patients with stable disease (non responders IWG), in which treatment was continued, achieved response after 9 months. In the macrocytic-responders group 87% exhibits again macrocytosis after 3 months, while 13% become normocytic. In the normocytic-responders group 92% exhibits again normocytosis, while 4/52 (8%) become macrocytic: in these 4 patients after three months there was a contemporary worsening in neutropenia and thrombocytopenia, with transfusion-dependence, regarded as first signs of progression of disease. Non responders were 23/137 (17%): in the macrocytic-non responders group 89% exhibit again macrocytosis after 3 months, while 11% become normocytic; in the normocytic group 80% exhibits again macrocytosis, while 20% become normocytic: of these 74% become transfusion-dependent at 6 months (median time to transfusion 12 months : range 1-23). These preliminary data can suggest that, in the majority of MDS patients responsive to ESA treatment, the increase of hemoglobin concentration occurs mainly stimulating erythroid production in MDS clones; in the minority of patients probably it happens recruiting residual polyclonal erythropoiesis. It is interesting to note that stimulating effects of ESAs last even when the expression of dysplasia progresses.
Fig. 1.
209 PROGNOSTIC SIGNIFICANCE OF WT1 EXPRESSION IN MYELODYSPLASTIC SYNDROME T. Zhang1, J. Cen1, L. Yao1, Y. Chen1, J. Xie1, C. Xu1, D. Wu1, A. Sun1, S. Chen1 1 Hemotalogy, 1st Affiliated Hospital Soochow University, Suzhou, China
Fig. 2.
Wilms’ tumor gene (WT1) has been recognized as a new prognostic factor and marker for the detection of minimal residual disease (MRD) in acute myeloid leukemia (AML). To reveal the expression pattern and clinical significance of WT1 gene in MDS, we measured the transcript levels of the WT1 gene in 113 newly diagnosed MDS patients by real-time quantitative RT-PCR. Fortyfour percent (50/113) of the patients were detected with high WT1 transcript levels (>300 copies/104 ABL) and it was highly
Poster Presentations – 13th International Symposium on Myelodyspastic Syndromes / Leukemia Research 39 S1 (2015) S1–S166
Conclusions: Our study reveals that patients treated with AMLlike chemotherapy reach more responses and more infections. OS is better among patients who reach transplat and is similar in both subgroups, aza or chemo as cytorreductors.
Table 1.
208 MANAGEMENT OF MYELODYSPLASTIC SYNDROMES WITH ERYTHROPOIESIS STIMULATING AGENTS: EVALUATION OF ERYTHROPOIETIC ASPECTS AND ANALYSIS OF RESPONSE C. Cerchione1, O. Vitagliano1, L. Simeone1, A.E. Pareto1, I. Soriente2, A.M. D’arco2, P. Danise2, F. Alfinito1, F. Pane1 1 Hematology, University of Naples Federico II, Naples, Italy; 2 Hematology, Ospedale Umberto I, Nocera Inferiore (SA), Italy Erythropoiesis stimulating agents (ESAs) are the frontline treatment in low risk anemic MDS patients and an employment of this therapy in the earlier stage of the disease can delay the need for RBC transfusion, hypothetically by slowing the disease course. It is matter of debate whether the clinical response is a result of proliferation and maturation of the dysplastic clone or stimulation of residual normal erythropoiesis by ESAs. Macrocytosis is one of the cytological hallmarks of dyserithropoiesis in MDS: an analysis of the erythropoietic response to ESAs therapy in a cohort of anemic not trasfusion-dependent MDS patients, enrolled in a retrospective register, RECAMDS, subgroup of Italian MDS register, was performed. 137 patients, treated with standard-dose ESAs, have been retrospectively analyzed (Table 1). Data analysis was performed, according to IWG 2006 criteria, at the baseline, after three and six month of continuous treatment, with a subanalysis of the patients according to WHO and R-IPSS risk stratification (Figures 1 and 2). ESAs treatment was started at mean Hb concentration of 9.5 g/dl, mean serum EPO concentration:
41 mU/L, after a mean time from diagnosis of six months (r.1-118). Overall response rate (ORR) was 83% (114/137) and no difference among WHO and IPSS subgroups was found: 76% achieved response after three months of treatment, while other 7% after six months. 2 patients with stable disease (non responders IWG), in which treatment was continued, achieved response after 9 months. In the macrocytic-responders group 87% exhibits again macrocytosis after 3 months, while 13% become normocytic. In the normocytic-responders group 92% exhibits again normocytosis, while 4/52 (8%) become macrocytic: in these 4 patients after three months there was a contemporary worsening in neutropenia and thrombocytopenia, with transfusion-dependence, regarded as first signs of progression of disease. Non responders were 23/137 (17%): in the macrocytic-non responders group 89% exhibit again macrocytosis after 3 months, while 11% become normocytic; in the normocytic group 80% exhibits again macrocytosis, while 20% become normocytic: of these 74% become transfusion-dependent at 6 months (median time to transfusion 12 months : range 1-23). These preliminary data can suggest that, in the majority of MDS patients responsive to ESA treatment, the increase of hemoglobin concentration occurs mainly stimulating erythroid production in MDS clones; in the minority of patients probably it happens recruiting residual polyclonal erythropoiesis. It is interesting to note that stimulating effects of ESAs last even when the expression of dysplasia progresses.
Fig. 1.
209 PROGNOSTIC SIGNIFICANCE OF WT1 EXPRESSION IN MYELODYSPLASTIC SYNDROME T. Zhang1, J. Cen1, L. Yao1, Y. Chen1, J. Xie1, C. Xu1, D. Wu1, A. Sun1, S. Chen1 1 Hemotalogy, 1st Affiliated Hospital Soochow University, Suzhou, China
Fig. 2.
Wilms’ tumor gene (WT1) has been recognized as a new prognostic factor and marker for the detection of minimal residual disease (MRD) in acute myeloid leukemia (AML). To reveal the expression pattern and clinical significance of WT1 gene in MDS, we measured the transcript levels of the WT1 gene in 113 newly diagnosed MDS patients by real-time quantitative RT-PCR. Fortyfour percent (50/113) of the patients were detected with high WT1 transcript levels (>300 copies/104 ABL) and it was highly